Skip to content
EU Parl Watch

amendment list, 27 March 2026

EU rare disease action plan

Document SANT-AM-786805 · (2025/2130(INL))

Committee on Public Health

On Parliament’s site PDF Word

Full text

Jump to an amendment (360)
Text 2,178 paragraphs

Amendment 361

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part A – Recommendation 2 – paragraph 2 – indent 9

Motion for a resolutionAmendment
– define measurable Union-level targets, including objectives relating to maximum average diagnostic delay, rare disease registry interoperability coverage and equitable availability of authorised therapies across Member States.– define measurable Union-level targets, including objectives relating to maximum average diagnostic delay, rare disease registry interoperability coverage and the full implementation of Directives 2011/24/EU and (EU) 2023/970, contributing to equitable availability of authorised therapies across Member States.

Or. en

Amendment 362

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part A – Recommendation 2 – paragraph 2 – indent 9

Motion for a resolutionAmendment
– define measurable Union-level targets, including objectives relating to maximum average diagnostic delay, rare disease registry interoperability coverage and equitable availability of authorised therapies across Member States.– define measurable Union-level targets, including objectives relating to maximum average diagnostic delay, rare disease registry interoperability coverage and equitable availability of authorised therapies across Member States, participation in ERNs, and indicators monitoring the socio-economic wellbeing of caregivers.
Read the rest (2,166 paragraphs)

Or. en

Amendment 363

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 2 – paragraph 2 – indent 9

Motion for a resolutionAmendment
– define measurable Union-level targets, including objectives relating to maximum average diagnostic delay, rare disease registry interoperability coverage and equitable availability of authorised therapies across Member States.– define realistic and measurable Union-level targets, taking into account national healthcare system capacities, including objectives relating to maximum average diagnostic delay, rare disease registry interoperability coverage and equitable availability of authorised therapies across Member States.

Or. en

Amendment 364

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 2 – paragraph 3

Motion for a resolutionAmendment
The European Parliament considers that the structural design of the framework should be outcome-oriented, measurable and aligned with existing Union instruments, including the European Health Data Space, the Pharmaceutical Strategy for Europe and the objectives of the European Health Union.The European Parliament considers that the structural design of the framework should be outcome-oriented, measurable and aligned with existing Union instruments, including the European Health Data Space, the Pharmaceutical Strategy for Europe, Directive 2011/24/EU and the objectives of the European Health Union, as well as the proposal for a Regulation of the European Parliament and of the Council laying a framework for strengthening the availability and security of supply of critical medicinal products as well as the availability of, and accessibility of, medicinal products of common interest, and amending Regulation (EU) 2024/795 and the proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union's biotechnology and biomanufacturing sectors particular in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act).

Or. en

Amendment 365

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 1

Motion for a resolutionAmendment
The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families within Union health policy and ensure meaningful participation in governance structures.The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families within Union health policy, ensure their meaningful and effective participation, including where appropriate involvement in decision-making processes, in governance structures.

Or. en

Amendment 366

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 1

Motion for a resolutionAmendment
The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families within Union health policy and ensure meaningful participation in governance structures.The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families within Union health policy and ensure meaningful participation in governance structures and the development of Union - level benchmarks and scoreboards.

Or. en

Amendment 367

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 1

Motion for a resolutionAmendment
The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families within Union health policy and ensure meaningful participation in governance structures.The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families and caregivers within Union health policy and ensure meaningful participation in governance structures.

Or. en

Amendment 368

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 1

Motion for a resolutionAmendment
The European Parliament considers that the legislative act to be adopted should strengthen the position of rare disease patients and their families within Union health policy and ensure meaningful participation in governance structures.The European Parliament considers that the recommendation to be adopted should strengthen the position of rare disease patients and their families within Union health policy and ensure meaningful participation in governance structures.

Or. en

Amendment 369

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 1

Motion for a resolutionAmendment
– guarantee structured involvement of patient organisations, healthcare professionals and civil society in advisory bodies and evaluation processes at Union level;– guarantee structured involvement of patient organisations, healthcare professionals and civil society, representatives of the industry and industry associations in advisory bodies and evaluation processes at Union level;

Or. en

Amendment 370

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 1

Motion for a resolutionAmendment
– guarantee structured involvement of patient organisations, healthcare professionals and civil society in advisory bodies and evaluation processes at Union level;– guarantee structured, inclusive, and renumerated involvement of patient organisations, healthcare professionals and civil society in advisory bodies and evaluation processes at Union level;

Or. en

Amendment 371

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 1

Motion for a resolutionAmendment
– guarantee structured involvement of patient organisations, healthcare professionals and civil society in advisory bodies and evaluation processes at Union level;– guarantee structured involvement of patient organisations, healthcare professionals and civil society in advisory bodies and evaluation processes;

Or. en

Amendment 372

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 2

Motion for a resolutionAmendment
– ensure transparency of decision-making, public availability of monitoring data and periodic reporting to the European Parliament and to the Council;– ensure transparency of decision-making, public availability of monitoring data and periodic reporting to the European Parliament and to the Council, including an evaluation of how input from patients, carers, and other stakeholders has been taken into account, alongside mechanisms to assess the impact of the framework from a patient perspective, including through patient-reported experience and empowerment measures to identify gaps in care and support systems;

Or. en

Amendment 373

Kateřina Konečná

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 2 a (new)

Motion for a resolutionAmendment
– ensure meaningful and sustainable involvement of patients organisations through adequate and transparent funding;

Or. en

Amendment 374

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 3

Motion for a resolutionAmendment
– promote cross-border recognition of expert centres and facilitate patient mobility where appropriate, without undermining national competence in healthcare organisation;– promote cross-border recognition of expert centres, facilitate their integration, collaboration, real-time data exchange, and facilitate patient mobility where appropriate, without undermining national competence in healthcare organisation;

Or. en

Amendment 375

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 4

Motion for a resolutionAmendment
– provide safeguards to ensure that the generation and sharing of health data fully respect fundamental rights, including data protection and privacy;– provide safeguards to ensure that the generation and sharing of health data fully respect fundamental rights, including strict data protection safeguards, explicit patient consent and guarantees against unauthorised access or transfer to third countries, data protection and privacy;

Or. en

Amendment 376

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 4

Motion for a resolutionAmendment
– provide safeguards to ensure that the generation and sharing of health data fully respect fundamental rights, including data protection and privacy;– provide safeguards to ensure that the generation and sharing of health data fully respect fundamental rights, including the highest standards of data protection and privacy;

Or. en

Amendment 377

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 6

Motion for a resolutionAmendment
– address the socio-economic impact of rare diseases, including effects on employment, education and gender equality, through coordinated policy approaches at Union level.– address the socio-economic impact of rare diseases on patients and caregivers, including effects on employment, education, disability status, poverty risk and gender equality, through coordinated policy approaches at Union level.

Or. en

Amendment 378

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 6

Motion for a resolutionAmendment
– address the socio-economic impact of rare diseases, including effects on employment, education and gender equality, through coordinated policy approaches at Union level.– address the socio-economic impact of rare diseases on both patients and caregivers, including effects on employment, education and gender equality, through coordinated policy approaches at Union level.

Or. en

Amendment 379

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 6 a (new)

Motion for a resolutionAmendment
– promote integration with other non-health services, specially educational, with the objective of improving the integration of services both synchronously and diachronically, with special attention to the transition of patients from childhood and adolescence to adulthood, which in turn involves changes in care services from a relational, pedagogical, and health perspective.

Or. en

Amendment 380

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 6 a (new)

Motion for a resolutionAmendment
– promote training and guidance for healthcare professionals on the management of lifelong rare diseases beyond the age of childhood, including adherence support, shared decision-making, comorbidities linked to ageing, and communication on sensitive issues such as dietary restrictions, fertility, pregnancy, and family planning.

Or. en

Amendment 381

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 2 – indent 6 a (new)

Motion for a resolutionAmendment
– support the information and continuous training of primary care practioners;

Or. en

Amendment 382

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 3

Motion for a resolutionAmendment
The European Parliament considers that individuals affected by rare diseases should benefit from equitable access to timely diagnosis, high-quality treatment and integrated care irrespective of their Member State of residence, and that this objective cannot be sufficiently achieved by Member States acting alone.The European Parliament considers that individuals affected by rare diseases should benefit from equitable access to timely diagnosis, high-quality treatment and integrated care irrespective of their Member State of residence, and that this objective cannot be sufficiently achieved by Member States acting alone, thus, the rare diseases action plan should:

Or. en

Amendment 383

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 3 – indent 1 (new)

Motion for a resolutionAmendment
– establish life-course, patient-journey integrated care pathways as a core deliverable of European Reference Networks (ERNs), mandating the co-creation of implementable, end-to-end pathways structured around paediatric patient journeys, covering early suspicion and diagnosis, initial management, coordinated specialist care, rehabilitation and psychosocial support, as well as transition to adult services;

Or. en

Amendment 384

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 3 – paragraph 3 – indent 2 (new)

Motion for a resolutionAmendment
– stress the importance of patient-centred, digitally enabled coordination as the backbone of integrated care pathways, by promoting multidisciplinary care teams, active involvement of patients, families, and caregivers, and the use of interoperable digital tools—such as tele-expertise, shared care plans, and registries—to support the continuous monitoring of the paediatric journey, track quality indicators, and ensure seamless transition into adult care systems.

Or. en

Amendment 385

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 1

Motion for a resolutionAmendment
The European Parliament considers that the legislative act to be adopted should ensure the effective and measurable implementation of the European rare disease action framework through clear operational instruments, financial alignment and enforceable coordination mechanisms.The European Parliament considers that the recommendation to be adopted should ensure the effective and measurable implementation of the European rare disease action framework through clear operational instruments, financial alignment and enforceable coordination mechanisms.

Or. en

Amendment 386

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 1

Motion for a resolutionAmendment
– establish binding implementation tools, including delegated and implementing acts, to operationalise the European diagnostic coordination mechanism, the genetic diagnosis roadmap, the rare disease research agenda and the Union framework on artificial intelligence in rare diseases;– establish binding implementation tools, including delegated and implementing acts, to operationalise the European diagnostic coordination mechanism, the genetic diagnosis roadmap, the rare disease research agenda and the Union framework on artificial intelligence in rare diseases without prejudice to Member States’ competences in the organisation and delivery of health services;

Or. en

Amendment 387

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 1

Motion for a resolutionAmendment
– establish binding implementation tools, including delegated and implementing acts, to operationalise the European diagnostic coordination mechanism, the genetic diagnosis roadmap, the rare disease research agenda and the Union framework on artificial intelligence in rare diseases;– establish binding implementation tools, including delegated and implementing acts, to operationalise the European diagnostic coordination mechanism, the genetic diagnosis roadmap, the rare disease research agenda and the Union framework on responsible deployment of artificial intelligence in rare diseases, in compliance with Regulation (EU) 2024/1689;

Or. en

Amendment 388

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 1

Motion for a resolutionAmendment
– establish binding implementation tools, including delegated and implementing acts, to operationalise the European diagnostic coordination mechanism, the genetic diagnosis roadmap, the rare disease research agenda and the Union framework on artificial intelligence in rare diseases;– establish implementation tools, including delegated and implementing acts, to operationalise the European diagnostic coordination mechanism, the genetic diagnosis roadmap, the rare disease research agenda and the Union framework on artificial intelligence in rare diseases;

Or. en

Amendment 389

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 1 a (new)

Motion for a resolutionAmendment
– emphasise that he Coordination Board shall comprise representatives from relevant bodies within the Member States, European Reference Networks (ERNs), patient organisations, researchers, and the Commission, and shall serve as a steering body with effective decision-making capacity to address the needs related to rare diseases, bringing together patients, relevant healthcare authorities and institutions, as well as pricing and reimbursement, regulatory authorities and other public bodies from the Union and Member States, with the lead of the Commission and the cooperation of the Member States.

Or. en

Amendment 390

Elena Nevado del Campo, Dolors Montserrat, Tomislav Sokol, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 2

Motion for a resolutionAmendment
– ensure that Union financial instruments, including the national regional partnership plan (NRPP), are strategically aligned with the objectives of the Regulation and linked to measurable progress in reducing diagnostic delay, strengthening genomic capacity and improving equitable access to therapies;– ensure that Union financial instruments, including the upcoming European Competitiveness Fund, Horizon Europe and the national regional partnership plan (NRPP), are strategically aligned with the objectives of the Regulation and linked to measurable progress in reducing diagnostic delay, strengthening genomic capacity and improving equitable access to orphan medicinal products and advanced therapies;

Or. en

Amendment 391

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 2

Motion for a resolutionAmendment
– ensure that Union financial instruments, including the national regional partnership plan (NRPP), are strategically aligned with the objectives of the Regulation and linked to measurable progress in reducing diagnostic delay, strengthening genomic capacity and improving equitable access to therapies;– ensure that Union financial instruments, including the national regional partnership plan (NRPP), provide support for and are strategically aligned with the objectives of the Regulation and linked to measurable progress in reducing diagnostic delay, strengthening genomic capacity and improving equitable access to therapies;

Or. en

Amendment 392

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 2

Motion for a resolutionAmendment
– ensure that Union financial instruments, including the national regional partnership plan (NRPP), are strategically aligned with the objectives of the Regulation and linked to measurable progress in reducing diagnostic delay, strengthening genomic capacity and improving equitable access to therapies;– ensure that Union financial instruments, including the national regional partnership plan (NRPP), are strategically aligned with the objectives of the Regulation and support measurable progress in reducing diagnostic delay, strengthening genomic capacity and improving equitable access to therapies;

Or. en

Amendment 393

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 2 a (new)

Motion for a resolutionAmendment
– calls for a clear articulation of the post-2027 Multiannual Financial Framework (2028-2034) for rare diseases needs by ensuring that funding for rare diseases is addressed in its transnational dimension, systematically integrating the European Competitiveness Fund (ECF) and Horizon Europe, which are well suited to support cross-border research, innovation, and coordination;

Or. en

Amendment 394

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 2 b (new)

Motion for a resolutionAmendment
– stresses the need to explicitly reference these funding instruments and to allocate to them those components—particularly in research, data sharing, infrastructure, and cross-border collaboration shall ensure a coherent, balanced, and effective financing architecture at Union level.

Or. en

Amendment 395

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 3

Motion for a resolutionAmendment
– introduce conditionality mechanisms whereby access to specific Union funding is linked to compliance with agreed screening standards, interoperability requirements and reporting obligations;deleted

Or. en

Amendment 396

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 3

Motion for a resolutionAmendment
– introduce conditionality mechanisms whereby access to specific Union funding is linked to compliance with agreed screening standards, interoperability requirements and reporting obligations;deleted

Or. en

Amendment 397

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 3

Motion for a resolutionAmendment
– introduce conditionality mechanisms whereby access to specific Union funding is linked to compliance with agreed screening standards, interoperability requirements and reporting obligations;deleted

Or. en

Amendment 398

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 3

Motion for a resolutionAmendment
– introduce conditionality mechanisms whereby access to specific Union funding is linked to compliance with agreed screening standards, interoperability requirements and reporting obligations;– introduce conditionality mechanisms whereby access to specific Union funding is linked to compliance with agreed screening standards, interoperability requirements and reporting obligations, while ensuring targeted technical and financial support for Member States that need to build capacity in order to achieve such standards ;

Or. en

Amendment 399

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 3 a (new)

Motion for a resolutionAmendment
– highlight the importance of implementing population-wide preconception screening programmes, enabling the identification of carriers of genetic conditions and supporting informed reproductive decision-making, as a key measure for the prevention of certain rare diseases where feasible;

Or. en

Amendment 400

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 4

Motion for a resolutionAmendment
– require the Commission to develop a performance scoreboard based on the European rare disease benchmark, enabling objective comparison of Member States and identification of structural gaps;– require the Commission to develop - in close cooperation with patient organisations - a performance scoreboard based on the European rare disease benchmark, enabling objective comparison of Member States and identification of structural gaps;

Or. en

Amendment 401

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 4

Motion for a resolutionAmendment
– require the Commission to develop a performance scoreboard based on the European rare disease benchmark, enabling objective comparison of Member States and identification of structural gaps;– support the development of a performance scoreboard based on the European rare disease benchmark, enabling objective comparison of Member States and identification of structural gaps;

Or. en

Amendment 402

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 5

Motion for a resolutionAmendment
– provide for systematic data collection and independent evaluation of outcomes, including progress towards the one-year diagnosis objective where medically feasible;– provide for systematic and interoperable data collection and independent evaluation of outcomes, including progress in reducing diagnostic delay to a reasonable time where medically feasible;

Or. en

Amendment 403

Elena Nevado del Campo, Dolors Montserrat, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6

Motion for a resolutionAmendment
– strengthen cross-border implementation capacity by supporting joint clinical trials, coordinated regulatory pathways for advanced therapies and shared digital infrastructures.– strengthen cross-border implementation capacity by supporting joint clinical trials, coordinated regulatory pathways for advanced therapies and shared digital infrastructures while building on existing initiatives such as the ERNs and forthcoming Union legislation, such as the proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union's biotechnology and biomanufacturing sectors particular in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act).

Or. en

Amendment 404

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6

Motion for a resolutionAmendment
– strengthen cross-border implementation capacity by supporting joint clinical trials, coordinated regulatory pathways for advanced therapies and shared digital infrastructures.– strengthen cross-border implementation capacity by promoting transnational ecosystems to attract and support joint clinical trials, coordinated regulatory pathways for advanced therapies and shared digital infrastructures.

Or. en

Amendment 405

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6

Motion for a resolutionAmendment
– strengthen cross-border implementation capacity by supporting joint clinical trials, coordinated regulatory pathways for advanced therapies and shared digital infrastructures.– strengthen cross-border implementation capacity by supporting joint clinical trials, coordinated regulatory and access pathways for advanced therapies and shared digital infrastructures.

Or. en

Amendment 406

Nicolás González Casares, Vytenis Povilas Andriukaitis

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6 a (new)

Motion for a resolutionAmendment
– calls for the establishment of a sustainable financing system for academic clinical trials, based on public-private investment models complemented by appropriate incentives, specially pulled-oriented ones, in order to de-risk innovation and ensure the continued development and commercial viability of new therapies for rare diseases.

Or. en

Amendment 407

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6 a (new)

Motion for a resolutionAmendment
– promote simplification of administrative procedures for clinical trials on rare diseases in order to accelerate innovation and improve patient access to new therapies;

Or. en

Amendment 408

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6 b (new)

Motion for a resolutionAmendment
– urge the introduction and implementation, of accelerated regulatory pathways and sandboxes ('fast-track' procedures) for advanced therapy medicinal products (ATMPs), with the aim of expediting patient access while maintaining high standards of safety, quality, and efficacy.

Or. en

Amendment 409

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6 b (new)

Motion for a resolutionAmendment
– support the development of European production capacities for orphan medicinal products and advanced therapies in order to reduce strategic dependencies on third countries;

Or. en

Amendment 410

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 2 – indent 6 c (new)

Motion for a resolutionAmendment
– in accordance with the Regulation, the Commission is to adopt a Communication setting out a public health action plan specifically focused on rare diseases.

Or. en

Amendment 411

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part A – Recommendation 4 – paragraph 3

Motion for a resolutionAmendment
The European Parliament considers that the success of the framework will depend not only on policy design but on enforceable monitoring, transparent reporting and sustained financial commitment, ensuring that convergence in standards and outcomes is achieved across the Union while fully respecting Member States’ competences in healthcare organisation.deleted

Or. en

Amendment 412

Michele Picaro

Motion for a resolution

Annex I – Part B

Motion for a resolutionAmendment
[...]deleted

Or. en

Amendment 413

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 1

Motion for a resolutionAmendment
Regulation (EU) 2026/... of the European Parliament and of the Council on the Rare diseases action frameworkCouncil Recommendation on the Rare diseases action framework

Or. en

Amendment 414

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 3

Motion for a resolutionAmendment
THE EUROPEAN PARLIAMENT AND THE COUNCIL OF THE EUROPEAN UNION,THE COUNCIL OF THE EUROPEAN UNION,

Or. en

Amendment 415

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 4

Motion for a resolutionAmendment
Having regard to the Treaty on the Functioning of the European Union, and in particular Article 114(1) thereof,Having regard to the Treaty on the Functioning of the European Union, and in particular Article 168 thereof,

Or. en

Amendment 416

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 7

Motion for a resolutionAmendment
After transmission of the draft legislative act to the national parliaments,deleted

Or. en

Amendment 417

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 8

Motion for a resolutionAmendment
Having regard to the opinion of the European Economic and Social Committee1 ,deleted
1 OJ...

Or. en

Amendment 418

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 9

Motion for a resolutionAmendment
Acting in accordance with the ordinary legislative procedure2 ,deleted
2 OJ...

Or. en

Amendment 419

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 10 – point 1

Motion for a resolutionAmendment
(1) Although individually rare, they collectively affect an estimated 27 to 36 million people in the Union and therefore constitute a significant public health challenge.(1) Although individually rare, rare diseases collectively affect an estimated 27 to 36 million people in the Union and therefore constitute a significant public health challenge.

Or. en

Amendment 420

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 2

Motion for a resolutionAmendment
(2) More than 6 000 rare diseases have been identified, the majority of which are genetic in origin, often manifesting in childhood and requiring lifelong, multidisciplinary care.(2) More than 6 000 rare diseases have been identified and the number of conditions continues to grow as advances in genomics and clinical research enhance rare diseases identification, the majority of which are genetic in origin, often manifesting in childhood and requiring lifelong, multidisciplinary care.

Or. en

Amendment 421

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 3

Motion for a resolutionAmendment
(3) Approximately 95 % of rare diseases lack an authorised therapeutic option and, where treatments exist, access across Member States remains uneven, resulting in disparities in patient outcomes.(3) Approximately 95 % of rare diseases lack an authorised therapeutic option and, where treatments exist, access across Member States remains uneven, resulting in disparities in patient outcomes which reaffirms the need to improve access to specialised expertise, advanced diagnostic technologies and cross-border collaboration across the Union to address the needs of undiagnosed patients.

Or. en

Amendment 422

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 3 a (new)

Motion for a resolutionAmendment
(3 a) A significant proportion of rare diseases affect children and require early diagnosis, adapted care pathways and targeted research efforts.

Or. en

Amendment 423

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 3 b (new)

Motion for a resolutionAmendment
(3 b) Dedicated research streams for paediatric rare diseases should be established.

Or. en

Amendment 424

Elena Nevado del Campo, Dolors Montserrat, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 4

Motion for a resolutionAmendment
(4) Patients affected by rare diseases frequently experience prolonged diagnostic delays, misdiagnoses and inappropriate treatments, leading to avoidable health deterioration, psychological distress and increased long-term healthcare and social costs.(4) Patients affected by rare diseases frequently experience prolonged diagnostic delays, misdiagnoses and inappropriate treatments, leading to avoidable health deterioration, psychological distress and increased long-term healthcare and social costs for both them and their caregivers.

Or. en

Amendment 425

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part B – paragraph 10 – point 5

Motion for a resolutionAmendment
(5) Rare diseases are characterised by small and geographically dispersed patient populations, limiting the ability of individual Member States to independently generate sufficient clinical evidence, research capacity, specialised expertise and negotiating leverage.(5) Rare diseases are characterised by small and geographically dispersed patient populations, limiting the ability of individual Member States to independently generate sufficient clinical evidence and expertise, research capacity, specialised expertise and negotiating leverage.

Or. en

Amendment 426

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 5 a (new)

Motion for a resolutionAmendment
(5 a) Ultra rare conditions generate extra challenges in diagnostics, clinical evidence and treatment due to their low prevalence - affecting fewer than 1 in 50 000 persons - and even wider geographical dispersion.

Or. en

Amendment 427

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 5 a (new)

Motion for a resolutionAmendment
(5 a) Primary care practitioners play a key role in the early identification of rare diseases and should thus be properly trained, informed and supported.

Or. en

Amendment 428

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part B – paragraph 10 – point 6

Motion for a resolutionAmendment
(6) Cross-border pooling of expertise, data and resources generates European added value by overcoming structural scale limitations and enabling efficient research, diagnosis and treatment pathways.(6) Cross-border pooling of expertise, data and resources generates European added value by overcoming structural scale limitations and enabling efficient research, the development of expertise, diagnosis and treatment pathways.

Or. en

Amendment 429

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 6 a (new)

Motion for a resolutionAmendment
(6 a) The public consultation carried out by the European Parliament in March 2025 underlined the need to strengthen the ERNs and to improve communication of Union initiatives and funded-tools in the area of rare diseases, in order to increase awareness among patients and ensure that existing measures translate in tangible benefits.

Or. en

Amendment 430

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 8 a (new)

Motion for a resolutionAmendment
(8 a) Among the defining challenges of rare disease therapies is the lack of knowledge about the conditions, biological origins, and the general scientific understanding. In recognition of the various ongoing initiatives and to maximise the impact of ongoing research and resources, the European rare disease research agenda should consider and build on existing initiatives, such as the European Rare Diseases Research Alliance (ERDERA).

Or. en

Amendment 431

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 9 a (new)

Motion for a resolutionAmendment
(9 a) The Commission communication of 2 July 2025 Choose Europe for life sciences - A strategy to position the EU as the world´s most attractive place for life sciences by 2030 highlights the importance of strengthening the competitiveness of the Union´s life sciences and biotechnology ecosystem, which is particularly relevant for rare diseases.

Or. en

Amendment 432

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 9 a (new)

Motion for a resolutionAmendment
(9 a) The organisation, financing and delivery of healthcare services remain the exclusive competence of the Member States. Union action should therefore support, coordinate and complement national policies without leading to harmonisation.

Or. en

Amendment 433

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 10

Motion for a resolutionAmendment
(10) The establishment of European Reference Networks (ERNs) under Directive 2011/24/EU of the European Parliament and of the Council3 has demonstrated the benefits of Union-level cooperation in pooling expertise for rare and complex conditions. However, enhanced governance, sustainable financing, structured integration into national health systems and strengthened referral pathways are required to fully realise their potential.(10) The establishment of European Reference Networks (ERNs) under Directive 2011/24/EU of the European Parliament and of the Council3 has demonstrated the benefits of Union-level cooperation in pooling expertise for rare and complex conditions. However, further support to ERNs as complementary tools to national centres of expertise, and strengthened referral pathways are required to fully realise their potential.
3 Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare (OJ L 88, 4.4.2011, p. 45, ELI: http://data.europa.eu/eli/dir/2011/24/oj).3 Directive 2011/24/EU of the European Parliament and of the Council of 9 March 2011 on the application of patients’ rights in cross-border healthcare (OJ L 88, 4.4.2011, p. 45, ELI: http://data.europa.eu/eli/dir/2011/24/oj).

Or. en

Amendment 434

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 10 a (new)

Motion for a resolutionAmendment
(10 a) The reform of the Union pharmaceutical legislation updates the regulatory and incentive framework applicable to orphan medicinal products, including market exclusivity, with a view to supporting innovation and competitiveness while ensuring that the interests of patients, in terms of the availability and accessibility of treatments for rare diseases, remain central.

Or. en

Amendment 435

Elena Nevado del Campo, Dolors Montserrat, Tomislav Sokol, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 10 b (new)

Motion for a resolutionAmendment
(10 b) The proposal for a Regulation of the European Parliament and of the Council laying a framework for strengthening the availability and security of supply of critical medicinal products as well as the availability of, and accessibility of, medicinal products of common interest, and amending Regulation (EU) 2024/795 recognises the importance of collaborative procurement and voluntary joint procurement mechanisms to improve the availability of critical medicinal products across the Union. Those initiatives could contribute to more equitable access to orphan medicinal products, in particular in smaller markets or where patient populations are geographically dispersed.

Or. en

Amendment 436

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part B – paragraph 10 – point 11

Motion for a resolutionAmendment
(11) Significant disparities persist across Member States in the screening programmes of newborns, genomic testing capacity and early detection strategies, resulting in unequal opportunities for timely diagnosis. Voluntary alignment of screening principles and the establishment of benchmark standards can contribute to convergence while respecting national competences.(11) Significant disparities persist across Member States in the screening programmes of newborns, genomic testing capacity and early detection strategies, resulting in unequal opportunities for timely diagnosis. Voluntary alignment of screening principles and the establishment of benchmark standards can contribute to convergence while respecting national competences, when combined with targeted support for Member States facing structural capacity gaps.

Or. en

Amendment 437

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 11

Motion for a resolutionAmendment
(11) Significant disparities persist across Member States in the screening programmes of newborns, genomic testing capacity and early detection strategies, resulting in unequal opportunities for timely diagnosis. Voluntary alignment of screening principles and the establishment of benchmark standards can contribute to convergence while respecting national competences.(11) Significant disparities persist across Member States in the screening programmes of newborns, genomic testing capacity and early detection strategies, resulting in unequal opportunities for timely diagnosis. The development of a harmonised best- in-class Union newborn screening panel by which all newborns are to be screened, and the establishment of benchmark standards can contribute to convergence while respecting national competences.

Or. en

Amendment 438

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 11

Motion for a resolutionAmendment
(11) Significant disparities persist across Member States in the screening programmes of newborns, genomic testing capacity and early detection strategies, resulting in unequal opportunities for timely diagnosis. Voluntary alignment of screening principles and the establishment of benchmark standards can contribute to convergence while respecting national competences.(11) Significant disparities persist across and within Member States in the screening programmes of newborns, genomic testing capacity and early detection strategies, resulting in unequal opportunities for timely diagnosis. Voluntary alignment of screening principles and the establishment of benchmark standards can contribute to convergence while respecting national competences.

Or. en

Amendment 439

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 11 a (new)

Motion for a resolutionAmendment
(11 a) Whereas patients in island and geographically isolated Member States face additional structural barriers to accessing specialised rare disease care, including higher costs of cross-border travel, absence of national ERN reference centres and limited domestic genomic sequencing capacity; whereas those patients are disproportionately dependent on cross-border healthcare pathways and require targeted Union-level support to ensure equitable access.

Or. en

Amendment 440

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 10 – point 11 a (new)

Motion for a resolutionAmendment
(11 a) Significant disparities also persist between men and women and across ages, further contributing to unequal access to timely diagnosis. Further measures by Member States and the Commission to improve diagnosis of rare diseases in women and children are necessary to provide equal care.

Or. en

Amendment 441

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 12

Motion for a resolutionAmendment
(12) Earlier and more accurate diagnosis reduces avoidable healthcare expenditure, improves patient outcomes and enhances labour market participation. The establishment of measurable objectives, including a one-year diagnostic target where medically feasible, can guide coordinated action and enhance accountability.(12) Earlier and more accurate diagnosis, including through newborn screening, which plays a key role in enabling pre-symptomatic diagnosis, reduces avoidable healthcare expenditure, improves patient outcomes and enhances labour market participation. The establishment of measurable objectives, including a one-year diagnostic target where medically feasible, can guide coordinated action and enhance accountability.
In the case of rare diseases for which a delay in diagnosis is likely to result in irreversible harm or loss of treatment opportunity, the Commission and Member States should promote shorter target timelines. For many rare diseases, early diagnosis and timely initiation of pharmacological and non-pharmacological treatment can prevent irreversible damage or disability. Coordinated Union action to ensure timely access to pharmacological and non-pharmacological treatments can significantly contribute to improved patient outcomes and long-term health system resilience.

Or. en

Amendment 442

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 12

Motion for a resolutionAmendment
(12) Earlier and more accurate diagnosis reduces avoidable healthcare expenditure, improves patient outcomes and enhances labour market participation. The establishment of measurable objectives, including a one-year diagnostic target where medically feasible, can guide coordinated action and enhance accountability.(12) Earlier and more accurate diagnosis reduces avoidable healthcare expenditure, improves patient outcomes and enhances labour market participation. The establishment of measurable objectives, including a one-year diagnostic target where medically feasible considering national capacities, can guide coordinated action and enhance accountability.

Or. en

Amendment 443

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 12

Motion for a resolutionAmendment
(12) Earlier and more accurate diagnosis reduces avoidable healthcare expenditure, improves patient outcomes and enhances labour market participation. The establishment of measurable objectives, including a one-year diagnostic target where medically feasible, can guide coordinated action and enhance accountability.(12) Earlier and more accurate diagnosis reduces avoidable healthcare expenditure, improves patient outcomes and enhances labour market participation. The establishment of objectives to reduce diagnostic delay to a reasonable time, where medically feasible, can guide coordinated action and enhance accountability.

Or. en

Amendment 444

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 12 a (new)

Motion for a resolutionAmendment
(12 a) Whereas certain rare genetic conditions, including beta-thalassaemia and sickle cell disease, exhibit significantly higher prevalence in specific geographic regions of the Union, therefore targeted Union support should acknowledge this regional epidemiological reality and promote the development of regional centres of expertise.

Or. en

Amendment 445

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 13

Motion for a resolutionAmendment
(13) The progressive integration of genomic technologies into healthcare systems requires structured coordination at Union level to promote quality standards, interoperability and equitable access. A Union roadmap can define common objectives and measurable targets without harmonising national healthcare delivery.(13) The progressive integration of genomic technologies into healthcare systems requires structured coordination at Union level to promote quality standards, interoperability, equitable access as well as targeted support to Member States in their necessary investments in such technologies and in the training of the relevant workforce. A Union roadmap can define common objectives and measurable targets without harmonising national healthcare delivery.

Or. en

Amendment 446

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14

Motion for a resolutionAmendment
(14) Market fragmentation within the Union contributes to disparities in access to orphan medicinal products and advanced therapies, with patients in certain Member States experiencing substantial delays in availability and reimbursement.(14) Multi-factorial reasons, including evidential uncertainty, delays in diagnosis, treatment expertise or infrastructure, divergencies between national plans, priorities or strategies for rare diseases, as well as different requirements in national HTA and P&R processes, within the Union contributes to disparities in access to orphan medicinal products and advanced therapies, with patients in certain Member States experiencing substantial delays in availability and reimbursement.

Or. en

Amendment 447

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14

Motion for a resolutionAmendment
(14) Market fragmentation within the Union contributes to disparities in access to orphan medicinal products and advanced therapies, with patients in certain Member States experiencing substantial delays in availability and reimbursement.(14) Market fragmentation within the Union contributes to disparities in access to orphan medicinal products and advanced therapies, with patients in certain Member States experiencing substantial delays in availability and reimbursement. The reform of the Union pharmaceutical legislation and the proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union's biotechnology and biomanufacturing sectors particular in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act) are expected to contribute to a more supportive framework for innovation, competitiveness and availability in the field of rare diseases.

Or. en

Amendment 448

András Tivadar Kulja

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14

Motion for a resolutionAmendment
(14) Market fragmentation within the Union contributes to disparities in access to orphan medicinal products and advanced therapies, with patients in certain Member States experiencing substantial delays in availability and reimbursement.(14) Market fragmentation, lack of diagnosis, treatment expertise or infrastructure, divergence between national plans and priorities or strategies for rare diseases within the Union contributes to disparities in access to orphan medicinal products and advanced therapies, with patients in certain Member States experiencing substantial delays in availability and reimbursement.

Or. en

Amendment 449

Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14 a (new)

Motion for a resolutionAmendment
(14 a) The protection of intellectual property rights and regulatory exclusivities plays a crucial role in fostering innovation in the development of advanced therapies and medicines for rare diseases; ensuring that innovators can secure adequate protection for proprietary technologies, data, and know-how is essential to incentivise investment in research and development, facilitate the translation of scientific discoveries into clinical applications, and maintain the sustainability of the European pharmaceutical ecosystem. Appropriate regulatory safeguards, including market authorisation exclusivities, data protection periods, and patent rights, should be maintained in a manner that balances the need for access and affordability with the necessity to preserve incentives for continuous innovation and technological advancement.

Or. en

Amendment 450

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14 a (new)

Motion for a resolutionAmendment
(14 a) The Union’s life science sector is a major driver of European competitiveness and a key asset in developing new treatments and therapies for rare diseases, and needs to be supported by a clear, flexible and efficient regulatory framework.

Or. en

Amendment 451

Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14 b (new)

Motion for a resolutionAmendment
(14 b) It is essential to further strengthen the global regulatory leadership of the European Medicines Agency (EMA), reinforcing its role as a key reference authority to ensure high scientific and ethical standards and to support, in a proactive manner, the development of new pharmaceutical products from the earliest stages, including study design and the assessment of the treatment landscape.

Or. en

Amendment 452

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 10 – point 14 b (new)

Motion for a resolutionAmendment
(14 b) The Commission has proposed new general pharmaceutical legislation that aims to increase incentives for investments in therapies for rare diseases, as well as also the proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union's biotechnology and biomanufacturing sectors particular in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act) to further strengthen the Union’s life science sector.

Or. en

Amendment 453

Billy Kelleher, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 15

Motion for a resolutionAmendment
(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.(15) Coordinated clinical evidence gathering and collation, structured exploitation obligations linked to Union funding, the full implementation of Directives 2011/24/EU and (EU) 2023/970, cross-border cooperation initiatives, and strengthened, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equitable patient access to treatment, while fully respecting Member States’ competence in pricing and reimbursement decisions. In addition, action to enable harmonised packaging for OMPs could help facilitate availability across Member States, particularly in smaller markets.

Or. en

Amendment 454

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 15

Motion for a resolutionAmendment
(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, the full implementation of Directives 2011/24/EU and (EU) 2023/970, cross-border cooperation initiatives, and strengthened collaboration in health technology assessment can enhance patient access to treatment while fully respecting Member States’ competence in pricing and reimbursement decisions. In addition, action to enable harmonised packaging for OMPs could help facilitate availability across Member States, particularly in smaller markets.

Or. en

Amendment 455

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 15

Motion for a resolutionAmendment
(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, the full implementation of Directives 2011/24/EU and (EU) 2023/970, cross-border cooperation initiatives, and strengthened voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.

Or. en

Amendment 456

Emmanouil Fragkos, Galato Alexandraki, Geadis Geadi

Motion for a resolution

Annex I – Part Β – paragraph 10 – point 15

Motion for a resolutionAmendment
(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.(15) Coordinated clinical evidence generation, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.

Or. el

Amendment 457

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 15

Motion for a resolutionAmendment
(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, voluntary cooperation in procurement and strengthened collaboration in health technology assessment can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.(15) Coordinated clinical evidence generation, structured exploitation obligations linked to Union funding, voluntary cooperation in procurement and strengthened collaboration in health technology assessment, including under Regulation (EU) 2021/2282, can enhance equity and efficiency while fully respecting Member States’ competence in pricing and reimbursement decisions.

Or. en

Amendment 458

Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part B – paragraph 10 – point 16

Motion for a resolutionAmendment
(16) Platform technology master files made available under transparent and non-discriminatory conditions can facilitate the development and scaling-up of advanced therapy medicinal products, particularly for rare diseases with limited commercial incentives, while ensuring high scientific standards and regulatory efficiency.(16) deleted

Or. en

Amendment 459

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 16 a (new)

Motion for a resolutionAmendment
(16 a) The existing rare disease ecosystem has helped develop over 260 orphan medicinal products. To build on this success, an overarching and holistic European policy approach to rare disease policy making is needed.

Or. en

Amendment 460

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 16 b (new)

Motion for a resolutionAmendment
(16 b) To improve access to innovative therapies and meet the needs of people living with a rare disease, the Union and its Member States should ensure that the access environment remains attractive for the life sciences industry to launch novel therapies.

Or. en

Amendment 461

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 17

Motion for a resolutionAmendment
(17) A structured cross-border exchange framework for hospital exemption advanced therapy medicinal products can facilitate access to treatments addressing unmet needs, particularly for patients who are clinically unfit to travel or affected by ultra-rare conditions, while preserving patient safety, regulatory responsibility and the non-commercial character of the hospital exemption framework.deleted

Or. en

Amendment 462

Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part B – paragraph 10 – point 17

Motion for a resolutionAmendment
(17) A structured cross-border exchange framework for hospital exemption advanced therapy medicinal products can facilitate access to treatments addressing unmet needs, particularly for patients who are clinically unfit to travel or affected by ultra-rare conditions, while preserving patient safety, regulatory responsibility and the non-commercial character of the hospital exemption framework.(17) deleted

Or. en

Amendment 463

Elena Nevado del Campo, Dolors Montserrat, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 18

Motion for a resolutionAmendment
(18) Rare disease research requires critical mass in patient recruitment, data pooling and biobank coordination, which can only be achieved through structured cross-border collaboration and strategic alignment of Union funding instruments.(18) Rare disease research requires critical mass in patient recruitment, data pooling and biobank coordination, which can only be achieved through structured cross-border collaboration and strategic alignment of Union funding instruments. Existing initiatives, including the European Rare Diseases Research Alliance (ERDERA), the ERNs, the Innovative Health Initiative and the International Rare Diseases Research Consortium (IRDiRC), provide an important basis for avoiding duplication and accelerating scientific progress.

Or. en

Amendment 464

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 18 a (new)

Motion for a resolutionAmendment
(18 a) Strengthened investments in European biomedical research capacities, including public laboratories, hospitals and universities, would reduce dependency on third countries for the development of innovative therapies and increase availability for patients.

Or. en

Amendment 465

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 10 – point 19

Motion for a resolutionAmendment
(19) Artificial intelligence systems have the potential to improve diagnostic accuracy, optimise clinical trial recruitment and enhance research efficiency in rare diseases. A Union-level framework is necessary to ensure that such systems are developed and deployed in accordance with Union legislation on data protection, medical devices and high-risk artificial intelligence, while safeguarding transparency and human oversight.(19) Artificial intelligence systems have the potential to improve diagnostic accuracy, optimise clinical trial recruitment and enhance research efficiency in rare diseases. Clear guidance and a Union-level framework is necessary to ensure that such systems are developed and deployed in accordance with Union legislation on data protection, medical devices and high-risk artificial intelligence, while safeguarding transparency and human oversight, creating opportunities for researchers, medical professionals and the life science sector to improve health outcomes.

Or. en

Amendment 466

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 19

Motion for a resolutionAmendment
(19) Artificial intelligence systems have the potential to improve diagnostic accuracy, optimise clinical trial recruitment and enhance research efficiency in rare diseases. A Union-level framework is necessary to ensure that such systems are developed and deployed in accordance with Union legislation on data protection, medical devices and high-risk artificial intelligence, while safeguarding transparency and human oversight.(19) Artificial intelligence systems have the potential to improve diagnostic accuracy, reduce diagnostic delay, optimise clinical trial recruitment, enhance research efficiency and facilitate the identification of complex and undiagnosed rare diseases. A Union-level framework is necessary to ensure that such systems are developed and deployed in accordance with Union legislation on data protection, medical devices and high-risk artificial intelligence, while safeguarding transparency and human oversight.

Or. en

Amendment 467

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part B – paragraph 10 – point 19

Motion for a resolutionAmendment
(19) Artificial intelligence systems have the potential to improve diagnostic accuracy, optimise clinical trial recruitment and enhance research efficiency in rare diseases. A Union-level framework is necessary to ensure that such systems are developed and deployed in accordance with Union legislation on data protection, medical devices and high-risk artificial intelligence, while safeguarding transparency and human oversight.(19) Artificial intelligence systems have the potential to improve diagnostic accuracy, optimise clinical trial recruitment and enhance research efficiency in rare diseases, and should be developed and deployed interoperably, in accordance with Union legislation on data protection, medical devices and high-risk artificial intelligence, while safeguarding transparency, non-discrimination and human oversight.

Or. en

Amendment 468

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 20

Motion for a resolutionAmendment
(20) Fragmented rare disease registry systems and inconsistent coding standards limit the capacity to conduct epidemiological surveillance, generate real-world evidence and support regulatory and health technology assessment processes.(20) Fragmented rare disease registry systems and inconsistent coding standards, including insufficient use of recognised rare disease nomenclatures such as ORPHAcodes maintained by Orphanet, limit the capacity to conduct epidemiological surveillance, generate real-world evidence and support regulatory and health technology assessment processes. The European Platform on Rare Disease Registration provides a basis for improving the comparability and interoperability of rare disease data across the Union.

Or. en

Amendment 469

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Annex I – Part B – paragraph 10 – point 20

Motion for a resolutionAmendment
(20) Fragmented rare disease registry systems and inconsistent coding standards limit the capacity to conduct epidemiological surveillance, generate real-world evidence and support regulatory and health technology assessment processes.(20) Fragmented rare disease registry systems, limited real-time data exchange among clinical expert centres and inconsistent coding standards limit the capacity to conduct epidemiological surveillance, generate real-world evidence, clinical expertise and support regulatory and health technology assessment processes.

Or. en

Amendment 470

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 20 a (new)

Motion for a resolutionAmendment
(20 a) The integration of Orphacodes across registries and healthcare systems would improve the identification, visibility and comparability of rare diseases across Member States

Or. en

Amendment 471

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 21

Motion for a resolutionAmendment
(21) The integration of rare disease data infrastructures into the European Health Data Space established by Regulation (EU) 2025/327 of the European Parliament and of the Council4 presents an opportunity to enhance interoperability, facilitate secure secondary use of data and support evidence-based policymaking in compliance with Union data protection rules.(21) The integration of rare disease data infrastructures into the European Health Data Space established by Regulation (EU) 2025/327 of the European Parliament and of the Council4 presents an opportunity to enhance interoperability, facilitate secure secondary use of data, secure cross-border data sharing, use of health data for research and innovation, and support evidence-based policymaking in compliance with Union data protection rules.
4 Regulation (EU) 2025/327 of the European Parliament and of the Council of 11 February 2025 on the European Health Data Space and amending Directive 2011/24/EU and Regulation (EU) 2024/2847 (OJ L, 2025/327, 5.3.2025, ELI: http://data.europa.eu/eli/reg/2025/327/oj).4 Regulation (EU) 2025/327 of the European Parliament and of the Council of 11 February 2025 on the European Health Data Space and amending Directive 2011/24/EU and Regulation (EU) 2024/2847 (OJ L, 2025/327, 5.3.2025, ELI: http://data.europa.eu/eli/reg/2025/327/oj).

Or. en

Amendment 472

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 21 a (new)

Motion for a resolutionAmendment
(21 a) The sharing and use of health data must fully respect patients’ rights, including explicit, informed and freely given consent, and ensure that Member States retain control over sensitive health data generated within their territory.

Or. en

Amendment 473

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 22 a (new)

Motion for a resolutionAmendment
(22 a) Rare diseases have a significant gender dimension as women often bear an unequal burden of care as well as gender related differences may persist in diagnosis, access to care and participation in research, requiring a more inclusive and gender-sensitive approach to rare disease policies.

Or. en

Amendment 474

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 23

Motion for a resolutionAmendment
(23) Integrated, multidisciplinary care pathways and cross-border continuity of care are necessary to address the complex and lifelong needs of rare disease patients and their families.(23) Integrated, multidisciplinary holistic patient - centred care pathways and cross-border continuity of care are necessary to address the complex and lifelong needs of rare disease patients and their families.

Or. en

Amendment 475

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Annex I – Part B – paragraph 10 – point 24 a (new)

Motion for a resolutionAmendment
(24 a) The development of advanced therapies, including genome-editing technologies, should be strictly regulated to prevent ethical abuses and ensure that applications remain limited to therapeutic purposes

Or. en

Amendment 476

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 25

Motion for a resolutionAmendment
(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection and reduction of diagnostic delay, access to certain Union funding instruments, including the national regional partnership plan (NRPP), should be linked to the existence or progressive implementation of screening frameworks aligned with Union benchmark standards.deleted

Or. en

Amendment 477

Elena Nevado del Campo, Dolors Montserrat, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 25

Motion for a resolutionAmendment
(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection and reduction of diagnostic delay, access to certain Union funding instruments, including the national regional partnership plan (NRPP), should be linked to the existence or progressive implementation of screening frameworks aligned with Union benchmark standards.(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection, the reduction of diagnostic delay and long- term care, Member States should be encouraged to include strategic projects, reforms, investments and other interventions supporting screening, coordination programmes, and long-term care services in their national and regional partnership plans (NRPPs), leaving their design and implementation to the discretion of Member States;

Or. en

Amendment 478

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Annex I – Part B – paragraph 10 – point 25

Motion for a resolutionAmendment
(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection and reduction of diagnostic delay, access to certain Union funding instruments, including the national regional partnership plan (NRPP), should be linked to the existence or progressive implementation of screening frameworks aligned with Union benchmark standards.(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection and reduction of diagnostic delay, certain Union funding instruments, including the national regional partnership plan (NRPP), should support the implementation of screening frameworks aligned with Union benchmark standards.

Or. en

Amendment 479

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek

Motion for a resolution

Annex I – Part B – paragraph 10 – point 25

Motion for a resolutionAmendment
(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection and reduction of diagnostic delay, access to certain Union funding instruments, including the national regional partnership plan (NRPP), should be linked to the existence or progressive implementation of screening frameworks aligned with Union benchmark standards.(25) In order to ensure that Union financial support in the field of rare diseases effectively contributes to early detection and reduction of diagnostic delay, Union financial support, including the national regional partnership plan (NRPP), should aim to support Member States’ efforts without imposing conditionality affecting the organisation of national healthcare systems.

Or. en

Amendment 480

Elena Nevado del Campo, Dolors Montserrat, Tomislav Sokol, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Part B – paragraph 10 – point 25 a (new)

Motion for a resolutionAmendment
(25 a) In the context of the post-2027 Multiannual Financial Framework (2028-2034), Union programmes and instruments, including Horizon Europe and the future European Competitiveness Fund, should contribute to supporting research, innovation, data infrastructure and strategic capacities in the field of rare diseases.

Or. en

Amendment 481

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Annex I – Part B – paragraph 10 – point 26 a (new)

Motion for a resolutionAmendment
(26 a) The socio-economic impact of rare diseases extends beyond healthcare, affecting education, employment, gender equality and social inclusion and placing a disproportionate burden on caregivers, whose financial and social wellbeing should be monitored as part of the framework’s implementation;

Or. en

Amendment 482

Billy Kelleher, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 27

Motion for a resolutionAmendment
(27) A permanent coordination board bringing together Member States, ERNs, patient organisations, researchers and the Commission can strengthen strategic alignment and ensure effective implementation of this Regulation.(27) A permanent coordination board bringing together Member States, ERNs, patient organisations, representatives of the industry and industry associations, researchers and the Commission can strengthen strategic alignment and ensure effective implementation of this Regulation.

Or. en

Amendment 483

Michalis Hadjipantela

Motion for a resolution

Annex I – Part B – paragraph 10 – point 27

Motion for a resolutionAmendment
(27) A permanent coordination board bringing together Member States, ERNs, patient organisations, researchers and the Commission can strengthen strategic alignment and ensure effective implementation of this Regulation.(27) A permanent coordination board bringing together Member States, ERNs, patient organisations, representatives of the industry, industry associations, researchers and the Commission can strengthen strategic alignment and ensure effective implementation of this Regulation.

Or. en

Amendment 484

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 28

Motion for a resolutionAmendment
(28) Since the objectives of this Regulation — namely reducing fragmentation, enhancing coordination, ensuring interoperable data exchange, facilitating cross-border cooperation in advanced therapies and generating European added value in the field of rare diseases — cannot be sufficiently achieved by the Member States acting alone but can rather, by reason of the cross-border nature and scale of the challenge, be better achieved at Union level, the Union may adopt measures, in accordance with the principle of subsidiarity as set out in Article 5 of the Treaty on European Union.(28) Since the objectives of this legislative act — namely reducing fragmentation, enhancing coordination, ensuring interoperable data exchange, facilitating cross-border cooperation in advanced therapies and generating European added value in the field of rare diseases — cannot be sufficiently achieved by the Member States acting alone but can rather, by reason of the cross-border nature and scale of the challenge, be better achieved at Union level, the Union may adopt measures, in accordance with the principle of subsidiarity as set out in Article 5 of the Treaty on European Union.

Or. en

Amendment 485

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 30

Motion for a resolutionAmendment
(30) A Regulation is the appropriate legal instrument to ensure uniform application of coordination standards, data interoperability requirements and governance mechanisms across the Union, thereby preventing further fragmentation that could result from divergent transposition measures. In order to ensure uniform conditions for the implementation of this Regulation, implementing powers should be conferred on the Commission. Those powers should be exercised in accordance with Regulation (EU) No 182/2011 of the European Parliament and of the Council5 .deleted
5 Regulation (EU) No 182/2011 of the European Parliament and of the Council of 16 February 2011 laying down the rules and general principles concerning mechanisms for control by the Member States of the Commission's exercise of implementing powers (OJ L 55, 28.2.2011, p. 13, ELI: http://data.europa.eu/eli/reg/2011/182/oj).

Or. en

Amendment 486

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 31

Motion for a resolutionAmendment
(31) In order to supplement or amend certain technical non-essential elements of this Regulation, the power to adopt acts in accordance with Article 290 TFEU should be delegated to the Commission in respect of the establishment and coordination of a Union Roadmap for the improvement of genetic diagnosis in rare diseases, the establishment of interoperability criteria and common data standards for the rare disease data infrastructures, the establishment of the structure and functionality of an European rare disease coordination board, and the establishment of an EU rare diseases benchmark. It is of particular importance that the Commission carry out appropriate consultations during its preparatory work, including at expert level, and that those consultations be conducted in accordance with the principles laid down in the Interinstitutional Agreement of 13 April 2016 on Better Law-Making6 . In particular, to ensure equal participation in the preparation of delegated acts, the European Parliament and the Council receive all documents at the same time as Member States' experts, and their experts systematically have access to meetings of Commission expert groups dealing with the preparation of delegated acts.deleted
6 OJ L 123, 12.5.2016, p. 1, ELI: http://data.europa.eu/eli/agree_interinstit/2016/512/oj.

Or. en

Amendment 487

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 32

Motion for a resolutionAmendment
(32) The Commission should periodically evaluate the implementation, effectiveness and proportionality of this Regulation, including progress towards diagnostic targets, performance convergence and equitable access to therapies.(32) The Commission should periodically evaluate the implementation, effectiveness and proportionality of this legislative act, including progress towards diagnostic targets, performance convergence and equitable access to therapies.

Or. en

Amendment 488

Emmanouil Fragkos, Galato Alexandraki, Geadis Geadi

Motion for a resolution

Annex I – Part Β – paragraph 10 – point 32 a (new)

Motion for a resolutionAmendment
(32a) Considering that the organisation and provision of health services fall under the responsibility of the Member States in accordance with Article 168(7) TFEU, the Union's action in the field of rare diseases should focus on supporting, coordinating and complementing national policies, without leading to an excessive regulatory burden or to harmonisation of health systems.

Or. el

Amendment 489

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 32 a (new)

Motion for a resolutionAmendment
(32 a) Recalling the 2009 Council Recommendations and recognising that harmonised policy actions are needed at Union and national level, each Member State should have an up-to-date rare disease action plan, with rare diseases integrated into healthcare systems.

Or. en

Amendment 490

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 32 b (new)

Motion for a resolutionAmendment
(32 b) While the Union has adopted a number of legislative acts to strengthen the Health Union, there are challenges with implementation, which undermine their intended impact. Examples include the implementation of Directives (EU) 2023/970 and 2011/24/EU, Council Directive 89/105/EEC, Regulations (EC) No 883/2004 and (EC) No 987/2009 , and the Union accounting rules. In addition to proposing new legislation, existing legislation must be implemented and made to be fit-for-purpose. Where possible, the Union should also pursue action where legislation may not be necessary.

Or. en

Amendment 491

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 32 c (new)

Motion for a resolutionAmendment
(32 c) While access remains a national competence, the Union can actively contribute to access by providing the appropriate resources to provide EU Joint Clinical Assessments slots to all developers, support early engagement, and horizon scanning initiatives to de-risk the development of novel therapies.

Or. en

Amendment 492

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Annex I – Part B – paragraph 10 – point 32 d (new)

Motion for a resolutionAmendment
(32 d) The Commission should allocate funding in the post-2027 Multiannual Financial Framework (2028-2034) to expand on the Mechanism of Coordinated Access to orphan medicinal products (MoCA) programme and support early engagement between payers, the Agency, patients, and industry. The Coordination Board should, in consultation with the relevant stakeholders, further define the scope of the expanded MoCA programme.

Or. en

Amendment 493

Kateřina Konečná

Motion for a resolution

Article 1 – paragraph 1 – introductory part

Motion for a resolutionAmendment
This Regulation establishes a European rare disease action framework to:This Regulation establishes a European rare disease action framework a coordinated European framework for action in rare diseases, in areas where there is clear European added value, and complements Member States’ efforts by promoting coordination. The framework aims to:

Or. en

Amendment 494

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 1 – paragraph 1 – introductory part

Motion for a resolutionAmendment
This Regulation establishes a European rare disease action framework to:This legislative act establishes a European rare disease action framework to:

Or. en

Amendment 495

Kateřina Konečná

Motion for a resolution

Article 1 – paragraph 1 – point a a (new)

Motion for a resolutionAmendment
(a a) promote well-being for all people living with rare diseases;

Or. en

Amendment 496

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 1 – paragraph 1 – point b

Motion for a resolutionAmendment
(b) enhance equitable access to treatment;(b) enhance equitable access to medicinal products, treatment and advanced therapies;

Or. en

Amendment 497

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 1 – paragraph 1 – point b

Motion for a resolutionAmendment
(b) enhance equitable access to treatment;(b) enhance equitable access to treatment, not least in cross-border healthcare;

Or. en

Amendment 498

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Article 1 – paragraph 1 – point b

Motion for a resolutionAmendment
(b) enhance equitable access to treatment;(b) enhance equitable access to treatments and services;

Or. en

Amendment 499

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Article 1 – paragraph 1 – point f

Motion for a resolutionAmendment
(f) reinforce governance and coordination mechanisms.(f) reinforce governance, cooperation and coordination mechanisms.

Or. en

Amendment 500

Kateřina Konečná

Motion for a resolution

Article 1 – paragraph 1 – point f a (new)

Motion for a resolutionAmendment
(f a) reduce inequalities, and build resilient systems by promoting inclusive and sustainable innovation.

Or. en

Amendment 501

Kateřina Konečná

Motion for a resolution

Article 1 – paragraph 1 a (new)

Motion for a resolutionAmendment
Following this Regulation, the Commission shall adopt a Commission communication establishing a public health action plan dedicated to rare diseases.

Or. en

Amendment 502

Kateřina Konečná

Motion for a resolution

Article 2 – paragraph 1 – point 1

Motion for a resolutionAmendment
(1) ‘rare disease’ means a life-threatening or chronically debilitating condition affecting not more than 5 in 10 000 persons in the Union;(1) ‘rare disease’ means a life-threatening or chronically debilitating condition affecting not more than 5 in 10 000 persons in the Union, including rare cancers, as referred to in Article 3(1), point (a) of Regulation (EC) No 141/2000;

Or. en

Amendment 503

Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Article 2 – paragraph 1 – point 3

Motion for a resolutionAmendment
(3) ʽultra-rare condition’ means a condition affecting fewer than 1 in 50 000 persons;deleted

Or. en

Amendment 504

Kateřina Konečná

Motion for a resolution

Article 2 – paragraph 1 – point 3

Motion for a resolutionAmendment
(3) ʽultra-rare condition’ means a condition affecting fewer than 1 in 50 000 persons;deleted

Or. en

Amendment 505

Emmanouil Fragkos, Galato Alexandraki, Geadis Geadi

Motion for a resolution

Article 2 – paragraph 1 – point 3

Motion for a resolutionAmendment
(3) ʽultra-rare condition’ means a condition affecting fewer than 1 in 50 000 persons;deleted

Or. el

Amendment 506

Kateřina Konečná

Motion for a resolution

Article 2 – paragraph 1 – point 4

Motion for a resolutionAmendment
(4) ʽpatient journey’ means a tool development that captures the natural history of a rare condition and the needs of patients through diary experiences and life-lens of people living with this condition.deleted

Or. en

Amendment 507

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Article 2 – paragraph 1 – point 4

Motion for a resolutionAmendment
(4) ʽpatient journey’ means a tool development that captures the natural history of a rare condition and the needs of patients through diary experiences and life-lens of people living with this condition.(4) ʽpatient journey’ means the set of stages, experiences, and processes that a person goes through from the appearance of the first symptoms, through the search for a diagnosis, access to tests and specialists, obtaining or not obtaining a definitive diagnosis, the start and follow-up of treatments, when they exist, to the adaptation and long-term management of the disease, including the impact on family, social, and work life that captures the natural history of a rare condition and the needs of patients through diary experiences and life-lens of people living with this condition.

Or. en

Amendment 508

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 2 – paragraph 1 – point 4

Motion for a resolutionAmendment
(4) ʽpatient journey’ means a tool development that captures the natural history of a rare condition and the needs of patients through diary experiences and life-lens of people living with this condition.(4) ʽpatient journey’ means a tool development that captures the natural history of a rare condition, including diagnosis, treatment, and follow-up, including the transition from paediatric to adult care and the needs of patients through diary experiences and life-lens of people living with this condition, and their families and caregivers.

Or. en

Amendment 509

Emmanouil Fragkos, Galato Alexandraki, Geadis Geadi

Motion for a resolution

Article 2 – paragraph 1 – point 4 a (new)

Motion for a resolutionAmendment
(4a) By 1 January 2030, Member States shall aim, with the support of the Commission, to significantly reduce the average time to diagnosis for rare diseases and, where medically feasible, make it possible for an accurate diagnosis to be made within one year from the initial medical assessment.

Or. el

Amendment 510

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti, Oliver Schenk

Motion for a resolution

Article 2 – paragraph 1 – point 4 a (new)

Motion for a resolutionAmendment
(4 a) 'orphan medicinal product' means a medicinal product as defined in Regulation (EC) No 141/2000 of the European Parliament and of The Council;

Or. en

Amendment 511

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti, Oliver Schenk

Motion for a resolution

Article 2 – paragraph 1 a (new)

Motion for a resolutionAmendment
‘advanced therapy medicinal product’ means an advanced therapy medicinal product as defined in Article 2(1), point (a) of Regulation (EC) No 1394/2007;

Or. en

Amendment 512

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Article 3 – point 1

Motion for a resolutionAmendment
1. The Commission shall establish a coordination mechanism to support Member States in reducing average time to diagnosis.1. The Commission shall support Member States in their actions aimed at reducing average time to diagnosis.

Or. en

Amendment 513

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 3 – point 1 a (new)

Motion for a resolutionAmendment
1 a. The Commission shall establish a Union expert advisory committee ('Expert advisory committee on newborn screening') to support Member States in the harmonisation of newborn screening programmes across the Union. (a) The Expert advisory committee on newborn screening shall facilitate the exchange of best practices, horizon scanning and cooperation between Member States on the implementation of newborn screening programmes. (b) The Expert advisory committee on newborn screening shall develop Union guidance setting out a harmonised best-in-class Union newborn screening panel, identifying the conditions for which all newborns shall be screened, on the basis of the latest scientific evidence, relevant marketing authorisations issued by the EMA, and appropriate quality standards and ethical safeguards. The Expert advisory committee on newborn screening shall provide guidance on how Member States can collaborate and share experiences and data to extend national screening programmes beyond Union recommendations. (c) The guidance shall be reviewed and updated at least once a year. (d) The Expert advisory committee on newborn screening shall include representatives of the Commission, the Member States and the European Medicines Agency. To inform its work, the Expert advisory committee on newborn screening may receive structured input from relevant stakeholders, including patient organisations, healthcare professionals, researchers, public health authorities, and representatives of industry and industry associations, including on conditions that may merit consideration for inclusion in the Union newborn screening panel. (e) Union financial support may support the establishment and functioning of the national coordinating bodies, as well as the implementation of the guidance.

Or. en

Amendment 514

Kateřina Konečná

Motion for a resolution

Article 3 – point 2

Motion for a resolutionAmendment
2. The mechanism shall facilitate exchange of best practices, voluntary alignment of the screening principles of newborns, genetic prenatal screening, and development of diagnostic and early diagnostic guidelines.2. The mechanism shall facilitate the exchange of best practices, and the voluntary alignment of screening principles, including newborn screening and genetic prenatal testing, such as carrier screening, the collection and sharing of evidence and results of pilots on screening programmes, including with registries, and the reduction of duplicative pilot projects in different Member States. It shall also support the development of diagnostic and early diagnosis guidelines.

Or. en

Amendment 515

François-Xavier Bellamy

Motion for a resolution

Article 3 – point 2

Motion for a resolutionAmendment
2. The mechanism shall facilitate exchange of best practices, voluntary alignment of the screening principles of newborns, genetic prenatal screening, and development of diagnostic and early diagnostic guidelines.2. The mechanism shall facilitate exchange of best practices, voluntary alignment of the screening principles of newborns;

Or. en

Amendment 516

Kateřina Konečná

Motion for a resolution

Article 3 – point 2 a (new)

Motion for a resolutionAmendment
2 a. In particular, the mechanism shall:
- develop guidelines, in consultation with patients and healthcare professionals, to improve access to, and referral pathways towards expert centres, ensuring effective navigation from primary care to specialised services and enabling timely and accurate diagnosis of rare and complex diseases;
- establish a strengthened, structured framework for collaboration and cocuration between Orphanet and the ERNs to improve governance, quality standards, update cycles, and resourcing, and to better coordinate the production and sharing of information, including clinical pathways and healthcare practices, bridging primary and expert care.

Or. en

Amendment 517

Michalis Hadjipantela

Motion for a resolution

Article 3 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The Commission shall ensure that the coordination mechanism provides targeted technical and financial support to Member States with limited domestic capacity for rare disease diagnosis, including island and peripheral Member States, to develop or strengthen access to accredited genomic laboratories and specialist referral centres;

Or. en

Amendment 518

Elena Nevado del Campo, Dolors Montserrat, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 3 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The mechanism shall also support voluntary multicountry cooperation, including where appropriate pilot studies and clinical research relevant to diagnostic and early detection approaches, with a view to strengthening evidence generation and improving timely diagnosis across the Union.

Or. en

Amendment 519

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 3 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The mechanism shall also facilitate exchange of best practices for decreasing inequalities in diagnosis, especially as regards sex- or age-based differences in time to diagnosis.

Or. en

Amendment 520

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 3 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The mechanism shall support the exchange of best practices on providing information, psychosocial support and counselling to parents at the time of diagnosis.

Or. en

Amendment 521

Kateřina Konečná

Motion for a resolution

Article 3 – point 2 b (new)

Motion for a resolutionAmendment
2 b. The guidelines shall also address post-diagnosis support, including access to genetic counselling, psychological support, treatment, and follow-up care, and shall clarify how healthcare systems will support individuals following diagnosis. The guidelines shall further include provisions to support individuals with undiagnosed conditions.

Or. en

Amendment 522

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 3 – point 2 b (new)

Motion for a resolutionAmendment
2 b. The mechanism shall support the use of telemedicine and virtual case discussion tools within the European Reference Networks in order to facilitate cross-border diagnostic consultations and timely access to specialised expertise.

Or. en

Amendment 523

Kateřina Konečná

Motion for a resolution

Article 3 – point 2 c (new)

Motion for a resolutionAmendment
2 c. The guidelines shall propose specific measures for national plans and strategies and indicators for monitoring their implementation.

Or. en

Amendment 524

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 3 a (new)

Motion for a resolutionAmendment
Article3a
The mechanism shall engage ERN Expert Platform in developing Union guidelines for newborn screening, diagnosis and genetic testing. The ERN Expert Platform for Newborn Screening would be well placed to support this, and to position the Union as the central point for information sharing on good practices from existing national programmes.

Or. en

Amendment 525

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 4 – point 1

Motion for a resolutionAmendment
1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible. The Commission shall establish recommendations and guidelines.1. By 1 January 2030, Member States shall put in place measure aiming that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible and that newborns are screened, where appropriate, in accordance with the guidance referred to in the second subparagraph, in order to enable timely and pre-symptomatic diagnosis. The Commission shall establish recommendations and guidelines, including on newborn screening, with the support of the Expert advisory committee.
In developing such recommendations, the Commission shall take into account the role of comprehensive genomic testing, including whole exome and whole genome sequencing where appropriate, as well as the integration of genomic data interpretation into clinical decision-making frameworks.

Or. en

Amendment 526

Kateřina Konečná

Motion for a resolution

Article 4 – point 1

Motion for a resolutionAmendment
1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible. The Commission shall establish recommendations and guidelines.1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically and scientifically feasible. The Commission shall establish a roll-out plan for achieving the diagnostic target through implementing acts, including the mandatory correct use of Orphacodes for diagnosed as well as undiagnosed cases. The Commission shall develop monitoring indicators and milestones in cooperation with Member States, which shall be used as benchmark for the evaluation referred to in Article 21.

Or. en

Amendment 527

András Tivadar Kulja

Motion for a resolution

Article 4 – point 1

Motion for a resolutionAmendment
1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible. The Commission shall establish recommendations and guidelines.1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible, and that newborns are screened, where appropriate, in accordance with the guidance referred to in paragraph 2, in order to enable timely and presymptomatic diagnosis. The Commission shall establish recommendations and guidelines, including on newborn screening with the support of the Expert advisory committee.

Or. en

Amendment 528

Michalis Hadjipantela

Motion for a resolution

Article 4 – point 1

Motion for a resolutionAmendment
1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible. The Commission shall establish recommendations and guidelines.1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible, and that newborns are screened, where appropriate, in accordance with the guidance referred to in paragraph 2, in order to enable timely and pre-symptomatic diagnosis. The Commission shall establish recommendations and guidelines.

Or. en

Amendment 529

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 4 – point 1

Motion for a resolutionAmendment
1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible. The Commission shall establish recommendations and guidelines.1. By 1 January 2030, Member States shall take all the necessary measures to reduce the average time to diagnosis for patients with suspected rare diseases and to ensure that patients with suspected rare diseases receive an accurate diagnosis within a reasonable amount of time from the first medical consultation, where medically feasible. The Commission shall establish recommendations and guidelines.

Or. en

Amendment 530

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek

Motion for a resolution

Article 4 – point 1

Motion for a resolutionAmendment
1. By 1 January 2030, Member States shall ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible. The Commission shall establish recommendations and guidelines.1. By 1 January 2034, Member States are encouraged to aim to ensure that patients with suspected rare diseases receive an accurate diagnosis within one year from the first medical consultation where medically feasible.

Or. en

Amendment 531

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 4 – point 1 a (new)

Motion for a resolutionAmendment
1 a. In the case of rare diseases for which a delay in diagnosis is likely to result in irreversible harm or loss of treatment opportunity, the Commission and Member States shall promote shorter target timelines.

Or. en

Amendment 532

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 4 – point 2

Motion for a resolutionAmendment
2. The Commission shall develop monitoring indicators and milestones in cooperation with Member States.2. The Commission shall develop monitoring indicators and milestones in cooperation with Member States in line with the scope of the legislative act referred to in Article 1. Such indicators shall include equity-focused monitoring indicators.

Or. en

Amendment 533

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 4 – point 2

Motion for a resolutionAmendment
2. The Commission shall develop monitoring indicators and milestones in cooperation with Member States.2. The Commission shall develop monitoring indicators and milestones in cooperation with Member States and relevant stakeholders of the medical and scientific communities.

Or. en

Amendment 534

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 4 – point 2

Motion for a resolutionAmendment
2. The Commission shall develop monitoring indicators and milestones in cooperation with Member States.2. The Commission shall develop monitoring indicators and milestones in cooperation with Member States and patient organisations.

Or. en

Amendment 535

François-Xavier Bellamy

Motion for a resolution

Article 4 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The Commission shall establish guidelines ensuring that genetic and genomic technologies are used in a manner consistent with fundamental rights. Those guidelines shall explicitly prohibit uses aimed at selection based on genetic characteristics without therapeutic purpose. The guidelines shall be developed in consultation with representatives of persons with disabilities.

Or. en

Amendment 536

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 5 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’).1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’) in support and complementary to the efforts of Member States.

Or. en

Amendment 537

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 5 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’).1. The Commission shall supplement this legislative act by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’).

Or. en

Amendment 538

András Tivadar Kulja

Motion for a resolution

Article 5 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’).1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis and blood spot testing in rare diseases (the ʽRoadmap’).

Or. en

Amendment 539

Michalis Hadjipantela

Motion for a resolution

Article 5 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’).1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis and blood spot testing in rare diseases (the ʽRoadmap’).

Or. en

Amendment 540

Kateřina Konečná

Motion for a resolution

Article 5 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of genetic diagnosis in rare diseases (the ʽRoadmap’).1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing and coordinating a Union Roadmap for the improvement of diagnosis in rare diseases (the ʽRoadmap’).

Or. en

Amendment 541

Kateřina Konečná

Motion for a resolution

Article 5 – point 2

Motion for a resolutionAmendment
2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and ensuring equitable access across the Union.2. The Roadmap shall contribute to the achievement of the EU diagnostics target set out in Article 4, and shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and ensuring equitable access across the Union.

Or. en

Amendment 542

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 5 – point 2

Motion for a resolutionAmendment
2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and ensuring equitable access across the Union.2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies, and newborn screening, including blood spot testing, into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield, and enabling timely and pre-symptomatic diagnosis, and ensuring equitable access across the Union.

Or. en

Amendment 543

András Tivadar Kulja

Motion for a resolution

Article 5 – point 2

Motion for a resolutionAmendment
2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and ensuring equitable access across the Union.2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies and newborn screening, including blood spot testing, into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and enabling timely and pre-symptomatic diagnosis, and ensuring equitable access across the Union.

Or. en

Amendment 544

Michalis Hadjipantela

Motion for a resolution

Article 5 – point 2

Motion for a resolutionAmendment
2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and ensuring equitable access across the Union.2. The Roadmap shall define common objectives, minimum recommended standards and measurable targets for the progressive integration of genomic technologies and newborn screening, including blood spot testing, into national healthcare systems, with a view to reducing diagnostic delay, increasing diagnostic yield and enabling timely and pre-symptomatic diagnosis, ensuring equitable access across the Union.

Or. en

Amendment 545

Victor Negrescu

Motion for a resolution

Article 5 – point 3 – point a

Motion for a resolutionAmendment
(a) access to validated next-generation sequencing technologies and accredited genomic laboratories;(a) access to validated next-generation sequencing technologies, prenatal and perinatal diagnostic tools where medically appropriate, and accredited genomic laboratories;

Or. en

Amendment 546

Kateřina Konečná

Motion for a resolution

Article 5 – point 3 – point b

Motion for a resolutionAmendment
(b) interoperable rare diseases registries and secure data infrastructures;(b) interoperable rare diseases registries and secure data infrastructures, within the rare disease data framework, as referred to in Article 10, and in accordance with the European Health Data Space Regulation and its phased implementation;

Or. en

Amendment 547

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 5 – point 3 – point b

Motion for a resolutionAmendment
(b) interoperable rare diseases registries and secure data infrastructures;(b) interoperable rare diseases registries and secure data infrastructures, pursuant to Articles 10 and 11;

Or. en

Amendment 548

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 5 – point 3 – point c

Motion for a resolutionAmendment
(c) multidisciplinary clinical-genomic evaluation frameworks and defined timelines for diagnosis; and(c) facilitating cooperation on multidisciplinary clinical-genomic evaluation approaches and monitoring diagnostic timelines based on commonly agreed indicators; and

Or. en

Amendment 549

Letizia Moratti

Motion for a resolution

Article 5 – point 3 – point d a (new)

Motion for a resolutionAmendment
(d a) the use of synthetic data, where the intrinsic scarcity of clinical data limits the ability to perform statistically robust analyses, in order to contribute to expanding available datasets for research, the development of diagnostic algorithms, and the validation of predictive models.

Or. en

Amendment 550

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 5 – point 3 a (new)

Motion for a resolutionAmendment
3 a. development of methodological guidance and decision-support for the health technology assessment of newborn and early detection screening approaches that make use of genomic technologies, including harmonised health-economic evaluation methods and the use of interoperable real-world evidence to inform Member State decision-making.

Or. en

Amendment 551

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 5 – point 4

Motion for a resolutionAmendment
4. By 1 January 2030, and biannually thereafter unless the delegated act referred to in paragraph 1 provides for more frequent reporting, Member States shall report on the progress made with regard to the indicators set out in the Roadmap. .4. By 1 January 2030, and every year thereafter, Member States shall report on the progress made with regard to the targets set out in the Roadmap and the relevant indicators pursuant to Article 4.

Or. en

Amendment 552

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge

Motion for a resolution

Article 5 – point 4

Motion for a resolutionAmendment
4. By 1 January 2030, and biannually thereafter unless the delegated act referred to in paragraph 1 provides for more frequent reporting, Member States shall report on the progress made with regard to the indicators set out in the Roadmap. .4. By 1 January 2034, and every year thereafter unless the delegated act referred to in paragraph 1 provides for more frequent reporting, Member States shall report on the progress made with regard to the indicators set out in the Roadmap.

Or. en

Amendment 553

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 5 – point 4 a (new)

Motion for a resolutionAmendment
4 a. The Roadmap shall include Union recommended specifications for newborn and early detection screening approaches that incorporate genomic technologies, establishing evidence-based, non-binding guidance – including inclusion criteria, core data elements and quality indicators – to support voluntary convergence and interoperability with rare disease registries and the EHDS.

Or. en

Amendment 554

Elena Nevado del Campo, Dolors Montserrat, Tomislav Sokol, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 5 a (new)

Motion for a resolutionAmendment
Article5a
Directive 2011/24/EU
The Commission shall ensure coherence with Directive 2011/24/EU, with a view to improving access to cross-border healthcare for persons living with rare diseases, accelerating relevant procedures, facilitating reimbursement, and reducing the administrative burden on patients, their families and caregivers.

Or. en

Amendment 555

Billy Kelleher

Motion for a resolution

Article 6

Motion for a resolutionAmendment
Article 6deleted
Accessibility through fostered development of medicines
Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:
(a) the creation of plans for equitable access that outline strategies and timelines for achieving affordability, availability and accessibility for the medicinal product(s);
(b) filing of an application for an open platform technology master file;
(c) accessibility issues related to specific rare diseases.

Or. en

Amendment 556

Kateřina Konečná

Motion for a resolution

Article 6 – title

Motion for a resolutionAmendment
Accessibility through fostered development of medicinesAccessibility of medicines

Or. en

Amendment 557

Kateřina Konečná

Motion for a resolution

Article 6 – paragraph 1 – introductory part

Motion for a resolutionAmendment
Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:The Action Plan shall propose where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, measures for beneficiaries to ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement to stimulate beneficiaries of any fund established by the Union and contributing in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, to ensure that the exploitation of such results contribute to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. For that purpose, the work programme could specify additional exploitation obligations such as:

Or. en

Amendment 558

Letizia Moratti

Motion for a resolution

Article 6 – paragraph 1 – introductory part

Motion for a resolutionAmendment
Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries shall, where appropriate and taking into account the specific characteristics, costs and risks associated with such products, make reasonable efforts to facilitate affordability, availability and accessibility in a manner consistent with public health needs in the Member States. Such efforts shall be implemented in a proportionate and flexible manner, without undermining the economic viability of the products concerned or the need to preserve effective incentives for research, development and innovation. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme may specify appropriate non binding targets that can include:

Or. en

Amendment 559

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 6 – paragraph 1 – introductory part

Motion for a resolutionAmendment
Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. Union funding supporting rare disease research and innovation shall respect national priorities and contribute to improving patient access, without imposing binding obligations on pricing, availability or healthcare organisation. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:

Or. en

Amendment 560

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 6 – paragraph 1 – introductory part

Motion for a resolutionAmendment
Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:Where Union funding under any fund established by the Union contributes in whole or in part to the development of advanced therapy medicinal products intended for the prevention, diagnosis or treatment of rare diseases, beneficiaries must ensure that the exploitation of such results contributes to their affordability, availability, and accessibility in a manner consistent with public health needs in the Member States. Grant agreements shall, where appropriate, include obligations on transparency regarding relevant categories of research and development expenditure and public contributions, as well as equitable access planning. The Commission and the Member States may provide for appropriate measures in the grant agreements to facilitate compliance with that requirement. For that purpose, the work programme shall specify additional exploitation obligations that shall include, as a minimum:

Or. en

Amendment 561

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 6 – paragraph 1 – point b

Motion for a resolutionAmendment
(b) filing of an application for an open platform technology master file;(b) where appropriate, filing of an application for an open platform technology master file;

Or. en

Amendment 562

Kateřina Konečná

Motion for a resolution

Article 6 – paragraph 1 – point b

Motion for a resolutionAmendment
(b) filing of an application for an open platform technology master file;(b) using an open platform technology master file approach;

Or. en

Amendment 563

Kateřina Konečná

Motion for a resolution

Article 6 – paragraph 1 – point c

Motion for a resolutionAmendment
(c) accessibility issues related to specific rare diseases.(c) addressing accessibility issues related to specific rare diseases.

Or. en

Amendment 564

Nicolás González Casares, Vytenis Povilas Andriukaitis, Nikos Papandreou

Motion for a resolution

Article 6 – paragraph 1 – point c a (new)

Motion for a resolutionAmendment
(c a) ensuring a repurposing pathway that simplifies access to new indications for rare disease when medicines are already approved.

Or. en

Amendment 565

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas

Motion for a resolution

Article 6 a (new)

Motion for a resolutionAmendment
Article6a
Commission procurement on behalf of or in the name of Member States
1. Upon a joint request by at least five Member States, the Commission may, where justified by the objectives of this Regulation, conduct procurement procedures on behalf of or in the name of those Member States for medicinal products intended for the diagnosis, prevention or treatment of rare and ultra-rare diseases, where such procedures are likely to improve security of supply, availability, accessibility or affordability across the Union.
2. Such procedures may in particular be considered for orphan medicinal products, advanced therapy medicinal products and other highly specialised rare disease medicinal products where small patient populations, market fragmentation or supply vulnerabilities create barriers to equitable access.
3. Participation in any procurement procedure under this Article shall remain voluntary and open to all Member States.
4. Any procurement procedure under this Article shall be conducted in accordance with the applicable Union legal framework and in a manner that safeguards transparency, fair competition, supply chain resilience, diversification and continuity of supply, and shall be without prejudice to Member States’ competences in pricing, reimbursement and the organisation of healthcare systems.
5. The Commission shall inform the European Parliament and the Council of the launch of any procurement procedure facilitated under this Article.

Or. en

Amendment 566

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas

Motion for a resolution

Article 6 b (new)

Motion for a resolutionAmendment
Article6b
Agreement concerning procedures under Articles 6a and 6b
1. Member States participating in procedures under Articles 6a and 6b shall share with the Commission any information relevant to the conduct of the procurement procedure and shall provide the necessary expertise and resources for its effective implementation.
2. An agreement between the Commission and the participating Member States shall determine the practical arrangements governing procedures under Articles 6a and 6b, including the division of responsibilities, the decision-making process, the information to be shared and, where appropriate, liability provisions, distribution arrangements and storage conditions.
3. In order to ensure transparency, predictability and effective participation, the Commission may issue guidance on common standards and practical arrangements for procedures under Articles 6a and 6b, taking into account the needs of smaller Member States, patients, healthcare professionals, public payers and relevant economic operators.
4. The practical arrangements referred to in paragraph 2 of this Article may, where appropriate and in accordance with Union law, include regulatory flexibilities relating to packaging, labelling and electronic product information, while ensuring that patients retain access to clear and appropriate information.

Or. en

Amendment 567

Elena Nevado del Campo, Dolors Montserrat, Michalis Hadjipantela, Adam Jarubas

Motion for a resolution

Article 6 b (new)

Motion for a resolutionAmendment
Article6b
Voluntary joint procurement of rare disease medicinal products
1. Member States may engage in voluntary joint procurement procedures for medicinal products, advance therapy medicinal products and other rare disease medicinal products intended for the diagnosis, prevention or treatment of rare and ultra rare diseases, where such procedures are likely to improve security of supply, availability, accessibility or affordability across the Union.
2. Upon request of at least five Member States, or on its own initiative where justified by the objectives of this Regulation, the Commission may facilitate or coordinate such joint procurement procedures, in accordance with the applicable Union legal framework.
3. Participation in any joint procurement procedure under this Article shall remain voluntary and open to all Member States. Any procurement procedure under this Article shall be conducted in a manner that safeguards transparency, fair competition, supply chain resilience, diversification and continuity of supply, and shall be without prejudice to Member States’ competences in pricing, reimbursement and the organisation of healthcare systems. The Commission shall inform the European Parliament and the Council of the launch of any joint procurement procedure facilitated under this Article.

Or. en

Amendment 568

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 6 d (new)

Motion for a resolutionAmendment
Article6d
European Biotech Act
The Commission shall take into account the measures set out in the proposed European Biotech Act aimed at facilitating the development of advanced therapy medicinal products for rare diseases, in particular by supporting more efficient cross-border clinical trials for small and geographically dispersed patient populations, promoting combined studies involving medicinal products, devices or diagnostics, and centres of excellence for advanced therapies, with a view to accelerating innovation while maintaining high standards of patient safety, data quality and regulatory oversight.

Or. en

Amendment 569

Kateřina Konečná

Motion for a resolution

Article 7 – point 1

Motion for a resolutionAmendment
1. For the purposes of this Regulation, a platform technology master file shall be considered an ʽopen platform’ where the owner of the underlying technology makes the relevant regulatory data, documentation and validated scientific components available, under transparent and non-discriminatory conditions, to any third party developing a product sharing the same common regulatory data and scientific components.1. A platform technology master file as defined and regulated by the revised Directive 2001/83/EC shall be considered an ʽopen platform’ where the owner of the underlying technology makes the relevant regulatory data, documentation and validated scientific components available, under transparent and non-discriminatory conditions, to any third party developing a product sharing the same common regulatory data and scientific components. Incentives to stimulate the use of such ‘open platform’ approach, shall be explored by the Commission in coordination and synergies with the incentives established by the proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union’s biotechnology and biomanufacturing sectors particularly in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act).

Or. en

Amendment 570

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 7 – point 1

Motion for a resolutionAmendment
1. For the purposes of this Regulation, a platform technology master file shall be considered an ʽopen platform’ where the owner of the underlying technology makes the relevant regulatory data, documentation and validated scientific components available, under transparent and non-discriminatory conditions, to any third party developing a product sharing the same common regulatory data and scientific components.1. For the purposes of this Regulation, a platform technology master file shall be considered an ʽopen platform’ where the owner of the underlying technology makes the relevant regulatory data, documentation and validated scientific components available, under transparent and non-discriminatory conditions, to any third party developing a product sharing the same common regulatory data and scientific components, while ensuring that intellectual property rights and incentives for innovation are preserved.

Or. en

Amendment 571

Kateřina Konečná

Motion for a resolution

Article 7 – point 2

Motion for a resolutionAmendment
2. The Commission shall adopt an implementing act establishing the procedural and technical requirements for the submission, assessment and maintenance of open platform technology master files, with particular regard to their use in the development of advanced therapy medicinal products, including those targeting rare diseases. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.deleted

Or. en

Amendment 572

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 7 – point 2

Motion for a resolutionAmendment
2. The Commission shall adopt an implementing act establishing the procedural and technical requirements for the submission, assessment and maintenance of open platform technology master files, with particular regard to their use in the development of advanced therapy medicinal products, including those targeting rare diseases. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.2. The Commission shall adopt an implementing act establishing the procedural and technical requirements for the submission, assessment and maintenance of open platform technology master files, with particular regard to their use in the development of advanced therapy medicinal products, including those targeting rare diseases. Such conditions shall include clear and proportionate terms of access, including any fees, which shall be publicly available and justified. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19. In developing the implementing act, the Commission shall consult relevant stakeholders, including patient organisations, academic developers, healthcare providers and industry representatives.

Or. en

Amendment 573

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 7 – point 2

Motion for a resolutionAmendment
2. The Commission shall adopt an implementing act establishing the procedural and technical requirements for the submission, assessment and maintenance of open platform technology master files, with particular regard to their use in the development of advanced therapy medicinal products, including those targeting rare diseases. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.2. The Commission shall adopt a delegated act establishing the procedural and technical requirements for the submission, assessment and maintenance of open platform technology master files, with particular regard to their use in the development of advanced therapy medicinal products, including those targeting rare diseases.

Or. en

Amendment 574

Kateřina Konečná

Motion for a resolution

Article 7 – point 3

Motion for a resolutionAmendment
3. A holder of a hospital exemption authorisation may apply for the establishment or extension of an open platform technology master file on the basis of the investigational medicinal product dossier supporting the authorised product, provided that the platform demonstrates scientific validity, reproducibility and potential applicability to other products based on the same technological principle.deleted

Or. en

Amendment 575

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 7 – point 3

Motion for a resolutionAmendment
3. A holder of a hospital exemption authorisation may apply for the establishment or extension of an open platform technology master file on the basis of the investigational medicinal product dossier supporting the authorised product, provided that the platform demonstrates scientific validity, reproducibility and potential applicability to other products based on the same technological principle.deleted

Or. en

Amendment 576

Kateřina Konečná

Motion for a resolution

Article 7 – point 4

Motion for a resolutionAmendment
4. The application referred to in paragraph 3 shall be accompanied by a report from the competent national regulatory authority confirming that the platform has been favourably assessed and that its use could facilitate the development, scaling-up or regulatory evaluation of products sharing the same scientific or technological basis, irrespective of whether a marketing authorisation is immediately sought.deleted

Or. en

Amendment 577

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 7 – point 4

Motion for a resolutionAmendment
4. The application referred to in paragraph 3 shall be accompanied by a report from the competent national regulatory authority confirming that the platform has been favourably assessed and that its use could facilitate the development, scaling-up or regulatory evaluation of products sharing the same scientific or technological basis, irrespective of whether a marketing authorisation is immediately sought.deleted

Or. en

Amendment 578

Michalis Hadjipantela

Motion for a resolution

Article 8 – point 1

Motion for a resolutionAmendment
1. By...[two years from the date of entry into force of this Regulation], the Commission shall adopt an implementing act establishing a European rare disease research agenda (the ʽAgenda’). That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. By...[two years from the date of entry into force of this Regulation], the Commission shall adopt an implementing act establishing a European rare disease research agenda (the ʽAgenda’). In preparing the Agenda, the Commission shall consult relevant stakeholders, including patient representatives, industry, healthcare professionals, and existing Union research initiatives. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.

Or. en

Amendment 579

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 8 – point 1

Motion for a resolutionAmendment
1. By...[two years from the date of entry into force of this Regulation], the Commission shall adopt an implementing act establishing a European rare disease research agenda (the ʽAgenda’). That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. By...[two years from the date of entry into force of this Regulation], the Commission shall adopt an implementing act establishing a European rare disease research agenda (the ʽAgenda’). In preparing the Agenda, the Commission shall consult relevant stakeholders, including patient representatives, industry, healthcare professionals, and existing Union research initiatives. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.

Or. en

Amendment 580

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 8 – point 2

Motion for a resolutionAmendment
2. The Agenda shall establish Union-level research priorities in the field of rare diseases, including translational research, advanced therapies, genomic medicine, real-world evidence generation and clinical trial innovation.2. The Agenda shall establish Union-level research priorities in the field of rare diseases, including translational research, research on environmental determinants of rare diseases. advanced therapies, genomic medicine, real-world evidence generation and clinical trial innovation.

Or. en

Amendment 581

Kateřina Konečná

Motion for a resolution

Article 8 – point 2

Motion for a resolutionAmendment
2. The Agenda shall establish Union-level research priorities in the field of rare diseases, including translational research, advanced therapies, genomic medicine, real-world evidence generation and clinical trial innovation.2. The Agenda shall establish Union-level research priorities in the field of rare diseases, including basic, to translational research, advanced therapies, genomic medicine, real-world evidence generation and clinical trial innovation, and research in social studies.

Or. en

Amendment 582

Kateřina Konečná

Motion for a resolution

Article 8 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The Agenda shall contribute to and accelerate directly the goals set by the International Rare Diseases Research Consortium (IRDiRC), and build upon the Strategic Research and Innovation Agenda developed by the European Rare Diseases Research Alliance (ERDERA).

Or. en

Amendment 583

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 8 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The Agenda shall also include research into rare conditions for which treatments exist but unmet needs remain for at least one subset of the patient population.

Or. en

Amendment 584

Kateřina Konečná

Motion for a resolution

Article 8 – point 4 a (new)

Motion for a resolutionAmendment
4 a. The Agenda shall be co-created through structured and deepened collaboration with relevant stakeholders, including patients.

Or. en

Amendment 585

Elena Nevado del Campo, Dolors Montserrat, Tomislav Sokol, Michalis Hadjipantela, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 8 a (new)

Motion for a resolutionAmendment
Article8a
European Reference Networks
1. The European Reference Networks shall contribute to the implementation of the Agenda by supporting the identification of research priorities, facilitating access to expertise, and promoting collaboration in the field of rare diseases across the Union.
2. The Commission and the Member States shall support the involvement of the European Reference Networks in cross-border and multinational research activities, including clinical trials, real-world evidence generation, data sharing and the development of clinical pathways and guidelines.
3. Particular attention shall be given to the sustainable financing of the European Reference Networks and to their better integration into national healthcare systems, in order to strengthen their contribution to research, diagnosis, treatment and continuity of care for persons living with rare diseases.
4. The Commission shall promote closer cooperation between the European Reference Networks, research infrastructures, patient organisations and relevant Union initiatives in order to translate the research results into more accurate diagnosis, treatment and care for persons living with rare diseases.

Or. en

Amendment 586

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 8 a (new)

Motion for a resolutionAmendment
Article8a
Supporting innovation in the Union
1. Within two years of the entry into force of any major revision of the Union’s pharmaceutical legislation with the potential or aim to impact incentives or capacity to develop innovative therapies for rare diseases, the Commission shall evaluate the effect of those legislative changes on research capacity, innovation output and patient access to novel diagnostics and treatments.
2. If the evaluation shows that the Union’s research capacity or innovation output, or patient access to novel diagnostics and treatments, has been negatively affected, or insufficiently positively affected, by such legislative changes, the Commission shall propose further measures to stimulate research, development and patient access to novel diagnostics and treatments.
3. Such supportive measures can include, but are not limited to:
(a) increasing incentives to research by strengthening intellectual property protections for orphan drugs and novel therapies;
(b) increasing flexibility for clinical trials through more efficient and timely approval processes;
(c) providing for more harmonisation of Union frameworks for cross-border clinical trials for therapies targeting rare diseases.

Or. en

Amendment 587

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 9 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt an implementing act establishing a Union framework for the responsible deployment of artificial intelligence systems in the field of rare diseases, with the objective of improving early diagnosis, clinical decision-making, research efficiency and patient outcomes. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. The Commission shall adopt an implementing act establishing a Union framework for the responsible deployment of artificial intelligence systems in the field of rare diseases, with the objective of supporting and complementing Member States´ efforts for improving early diagnosis, clinical decision-making, research efficiency and patient outcomes. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.

Or. en

Amendment 588

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 9 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt an implementing act establishing a Union framework for the responsible deployment of artificial intelligence systems in the field of rare diseases, with the objective of improving early diagnosis, clinical decision-making, research efficiency and patient outcomes. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. The Commission shall work on a Union framework for the responsible deployment of artificial intelligence systems in the field of rare diseases, with the objective of improving early diagnosis, clinical decision-making, research efficiency and patient outcomes. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.

Or. en

Amendment 589

Letizia Moratti

Motion for a resolution

Article 9 – point 1 a (new)

Motion for a resolutionAmendment
1 a. Artificial intelligence systems deployed pursuant to this Article shall be coordinated with Regulation (EU) 2024/1689, explicitly distinguishing between systems intended for clinical use, which are subject to risk classification, and systems employed exclusively for scientific research, which are not subject to the Regulation (EU) 2024/1689 risk pyramid and whose use shall remain unrestricted.

Or. en

Amendment 590

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 9 – point 2

Motion for a resolutionAmendment
2. The framework shall define common requirements for the development, validation and use of artificial intelligence tools applied to rare disease screening, diagnosis, prognosis, treatment optimisation and research, ensuring scientific robustness, transparency, traceability and human oversight.2. The framework shall define clear common requirements for the development, validation and use of artificial intelligence tools applied to rare disease screening, diagnosis, prognosis, treatment optimisation and research, ensuring scientific robustness, transparency, traceability and human oversight, while supporting the use of artificial intelligence to enhance research, support clinical trials and promote better patient outcomes.

Or. en

Amendment 591

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 9 – point 2

Motion for a resolutionAmendment
2. The framework shall define common requirements for the development, validation and use of artificial intelligence tools applied to rare disease screening, diagnosis, prognosis, treatment optimisation and research, ensuring scientific robustness, transparency, traceability and human oversight.2. Without prejudice to Regulation (EU) 2024/1689, the framework shall define common requirements for the development, validation and use of artificial intelligence tools applied to rare disease screening, diagnosis, prognosis, treatment optimisation and research, ensuring scientific robustness, transparency, traceability and human oversight.

Or. en

Amendment 592

Letizia Moratti

Motion for a resolution

Article 9 – point 3

Motion for a resolutionAmendment
3. Artificial intelligence systems deployed pursuant to this Article shall comply with applicable Union legislation on data protection, medical devices, digital health and high-risk artificial intelligence systems, and shall ensure interoperability with national and Union health data infrastructures.3. Artificial intelligence systems deployed pursuant to this Article shall comply with applicable Union legislation on data protection, medical devices, digital health and high-risk artificial intelligence systems, and shall ensure interoperability with national and Union health data infrastructures. The framework shall ensure interoperability with national and Union health data infrastructures in accordance with clearly defined technical standards (e.g. HL7 FHIR), integration requirements with the EHDS, or criteria established in subsequent implementing acts.

Or. en

Amendment 593

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 9 – point 3

Motion for a resolutionAmendment
3. Artificial intelligence systems deployed pursuant to this Article shall comply with applicable Union legislation on data protection, medical devices, digital health and high-risk artificial intelligence systems, and shall ensure interoperability with national and Union health data infrastructures.3. Artificial intelligence systems deployed pursuant to this Article shall comply with applicable Union legislation on data protection (Regulation (EU) 2016/679), medical devices legislation, digital health (EHDS) and artificial intelligence systems (Regulation (EU) 2016/679) including high-risk artificial intelligence systems, and shall ensure interoperability with national and Union health data infrastructures.

Or. en

Amendment 594

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 9 – point 4

Motion for a resolutionAmendment
4. Member States shall promote the integration of validated artificial intelligence tools into rare disease reference centres, genomic diagnostic pathways and research networks, including through cross-border data collaboration where appropriate.4. Member States shall promote the integration of validated artificial intelligence tools into rare disease reference centres, genomic diagnostic pathways and research networks, including in cooperation with the European Reference Networks and through cross-border data collaboration where appropriate.

Or. en

Amendment 595

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 9 – point 4

Motion for a resolutionAmendment
4. Member States shall promote the integration of validated artificial intelligence tools into rare disease reference centres, genomic diagnostic pathways and research networks, including through cross-border data collaboration where appropriate.4. Member States may promote the integration of validated artificial intelligence tools into rare disease reference centres, genomic diagnostic pathways and research networks, including through cross-border data collaboration where appropriate.

Or. en

Amendment 596

Letizia Moratti

Motion for a resolution

Article 9 – point 4 a (new)

Motion for a resolutionAmendment
4 a. Artificial intelligence systems deployed pursuant to this Article shall promote the creation and use of synthetic data to address the intrinsic scarcity of clinical data in rare diseases, thereby supporting research, algorithm development, and validation of predictive models.

Or. en

Amendment 597

Kateřina Konečná

Motion for a resolution

Article 10 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt an implementing act establishing a federated European rare disease data network , unifying existing rare diseases data networks and according to the criteria established under the European Health Data Space. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. The Commission shall adopt an implementing act establishing a federated European rare disease data network, unifying existing rare diseases data networks and according to the criteria established under the European Health Data Space. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19, and it shall mandate the consistent implementation and recognition of OrphaCodes by relevant Member States’ institutions.

Or. en

Amendment 598

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 10 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt an implementing act establishing a federated European rare disease data network , unifying existing rare diseases data networks and according to the criteria established under the European Health Data Space. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. The Commission shall adopt an implementing act establishing a European rare disease data network, building on existing Union infrastructures, including the European Platform on Rare Disease Registration, and according to the criteria established under the European Health Data Space. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.

Or. en

Amendment 599

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 10 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt an implementing act establishing a federated European rare disease data network , unifying existing rare diseases data networks and according to the criteria established under the European Health Data Space. That implementing act shall be adopted in accordance with the advisory procedure referred to in Article 19.1. The Commission shall undertake concrete measures to further develop the collection and use of data on rare diseases, building upon the expertise of the European Reference Networks, unifying existing rare diseases data networks and according to the criteria established under the European Health Data Space.

Or. en

Amendment 600

Romana Jerković

Motion for a resolution

Article 10 – point 1 a (new)

Motion for a resolutionAmendment
1 a. In developing interoperable rare disease registries, the Union and the Member States shall draw on best practices from existing population-based data structures, including those developed under Europe’s Beating Cancer Plan such as the European Cancer Information System (ECIS). The interoperable rare disease data network shall support the monitoring of inequalities in access to diagnosis, treatment and participation in clinical trials across Member States.

Or. en

Amendment 601

Kateřina Konečná

Motion for a resolution

Article 10 – point 1 a (new)

Motion for a resolutionAmendment
1 a. The rare diseases data network shall build upon existing infrastructures and initiatives, notably the Data Hub set up under the European Rare Diseases Research Alliance (ERDERA), a network of Findable, Accessible, Interoperable, and Reusable (FAIR) data sources, analysis methods, knowledge bases, and computing tools.

Or. en

Amendment 602

Kateřina Konečná

Motion for a resolution

Article 10 – point 2

Motion for a resolutionAmendment
2. Registries shall apply common data standards and coding systems. The Commission shall update and maintain common data standards according to technological improvement, through the establishment of common mandatory criteria, enabling communication and transmission of rare diseases related health data across Europe under criteria of interoperability.2. Registries shall apply common data standards and coding systems and shall be built upon FAIR principles for data management (Findable, Accessible, Interoperable, Reusable) . The Commission, with the support of the European Rare Disease Registry Infrastructure (ERDRI) and the European Platform on Rare Disease Registration (EU RD platform), shall update and maintain common data standards tools and guidance to support the interoperability of existing and new registries according to technological improvement, through the establishment of common mandatory criteria, enabling communication and transmission of rare diseases related health data across Europe under criteria of interoperability.

Or. en

Amendment 603

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 10 – point 2

Motion for a resolutionAmendment
2. Registries shall apply common data standards and coding systems. The Commission shall update and maintain common data standards according to technological improvement, through the establishment of common mandatory criteria, enabling communication and transmission of rare diseases related health data across Europe under criteria of interoperability.2. Registries shall apply common data standards and coding systems, including recognised rare diseases nomenclatures such as ORPHAcodes maintained by Orphanet, in order to ensure consistent identification and classification of rare diseases and the interoperability of data. The Commission shall update and maintain common data standards according to technological improvement, through the establishment of common mandatory criteria, enabling communication and transmission of rare diseases related health data across the Union in accordance to interoperability requirements and the highest legal and data protection standards.

Or. en

Amendment 604

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 10 – point 2

Motion for a resolutionAmendment
2. Registries shall apply common data standards and coding systems. The Commission shall update and maintain common data standards according to technological improvement, through the establishment of common mandatory criteria, enabling communication and transmission of rare diseases related health data across Europe under criteria of interoperability.2. Registries shall apply common data standards and coding systems, based on the currently established "Orphacodes". The Coordination Board, in accordance with Article 15, shall regularly update and maintain common data standards according to technological improvement, based on scientific data and its exchanges with the relevant experts, enabling communication and transmission of rare diseases related health data across Europe under criteria of interoperability.

Or. en

Amendment 605

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 10 a (new)

Motion for a resolutionAmendment
Article10a
To support quality assurance and long‑term impact assessment of screening programmes, outcome registries linked to screening activities shall be encouraged. Those registries shall collect standardised data on screening results, diagnostic confirmation, timeliness of follow‑up and clinical outcomes, and be interoperable with rare disease registries and the European Health Data Space.

Or. en

Amendment 606

Kateřina Konečná

Motion for a resolution

Article 11 – point 1

Motion for a resolutionAmendment
1. Rare disease data infrastructures shall be technically aligned with the European Health Data Space to enable secure secondary use. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing interoperability criteria and common data standards.1. European and national rare disease data infrastructures shall be technically aligned with the European Health Data Space to enable secure secondary use as well as primary use to support the delivery of cross-border healthcare and referral pathways between national health systems and ERNs. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing interoperability criteria and common data standards for primary and secondary uses.

Or. en

Amendment 607

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 11 – point 1

Motion for a resolutionAmendment
1. Rare disease data infrastructures shall be technically aligned with the European Health Data Space to enable secure secondary use. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing interoperability criteria and common data standards.1. Rare disease data infrastructures shall be technically aligned with the European Health Data Space to enable secure secondary use and secure cross-border data sharing. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing interoperability criteria and common data standards.

Or. en

Amendment 608

Kateřina Konečná

Motion for a resolution

Article 11 – point 2

Motion for a resolutionAmendment
2. The Commission shall ensure the integration of the ERNs as agents of the development of cross-country standards in exchange of cross-border data exchange within Europe as promoters of standardised practices in common data elements, and rare disease registry interoperability.deleted

Or. en

Amendment 609

Kateřina Konečná

Motion for a resolution

Article 11 – point 3

Motion for a resolutionAmendment
3. The Commission shall support improved referral pathways between national systems and ERNs.deleted

Or. en

Amendment 610

Emmanouil Fragkos, Galato Alexandraki, Geadis Geadi

Motion for a resolution

Article 11 – point 3

Motion for a resolutionAmendment
3. The Commission shall support improved referral pathways between national systems and ERNs.3. The Commission, in cooperation with the Member States, shall support improved referral pathways between national systems and ERNs.

Or. el

Amendment 611

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 12 – point 1

Motion for a resolutionAmendment
1. The Commission shall promote the use of rare disease registry-based real-world evidence to support regulatory and health technology assessment in decision-making of the Member States on rare diseases.1. The Commission shall promote the use of rare disease registry-based real-world evidence, including on long-term adherence, treatment burden, quality of life and socio-economic impact, to support regulatory and health technology assessment in decision-making of the Member States on rare diseases.

Or. en

Amendment 612

Kateřina Konečná

Motion for a resolution

Article 12 – point 1

Motion for a resolutionAmendment
1. The Commission shall promote the use of rare disease registry-based real-world evidence to support regulatory and health technology assessment in decision-making of the Member States on rare diseases.1. The Commission shall promote the use of rare disease registry-based real-world evidence to support clinical practice, regulatory and health technology assessment in decision-making of the Member States on rare diseases.

Or. en

Amendment 613

Kateřina Konečná

Motion for a resolution

Article 12 – point 2

Motion for a resolutionAmendment
2. Facilitate cross-border data sharing and collaboration among Member States, ensuring compliance with data protection laws and promoting the use of real-world evidence in joint clinical assessments under Regulation (EU) 2021/2282 of the European Parliament and of the Council7 .2. The Commission shall also facilitate cross-border data sharing and collaboration among Member States, ensuring compliance with data protection laws and promoting the use of real-world evidence in joint clinical assessments under Regulation (EU) 2021/2282 of the European Parliament and of the Council 7.
7 Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (OJ L 458, 22.12.2021, p. 1, ELI: http://data.europa.eu/eli/reg/2021/2282/oj).7 Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (OJ L 458, 22.12.2021, p. 1, ELI: http://data.europa.eu/eli/reg/2021/2282/oj).

Or. en

Amendment 614

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 12 – point 2

Motion for a resolutionAmendment
2. Facilitate cross-border data sharing and collaboration among Member States, ensuring compliance with data protection laws and promoting the use of real-world evidence in joint clinical assessments under Regulation (EU) 2021/2282 of the European Parliament and of the Council7 .2. Facilitate cross-border data sharing and collaboration among Member States, ensuring the highest compliance with data protection laws and promoting the use of real-world evidence in joint clinical assessments under Regulation (EU) 2021/2282 of the European Parliament and of the Council7 .
7 Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (OJ L 458, 22.12.2021, p. 1, ELI: http://data.europa.eu/eli/reg/2021/2282/oj).7 Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (OJ L 458, 22.12.2021, p. 1, ELI: http://data.europa.eu/eli/reg/2021/2282/oj).

Or. en

Amendment 615

Kateřina Konečná

Motion for a resolution

Article 12 – point 2 a (new)

Motion for a resolutionAmendment
2 a. Appropriate measures to track progress in this area, including alignment between regulatory and HTA processes, shall be included in the EU rare Disease Action Plan as referred to by Article 16.

Or. en

Amendment 616

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Article 12 – point 2 a (new)

Motion for a resolutionAmendment
2 a. The EU rare diseases benchmark shall enable real-time data exchange in transnational ecosystems that facilitate in-depth integrated clinical cooperation and the development of expertise.

Or. en

Amendment 617

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 12 a (new)

Motion for a resolutionAmendment
Article12a
Member States and the Commission shall facilitate the early and continuous involvement of healthcare professionals, patients and caregivers in the design, collection and use of real-world data, in order to address uncertainties related to the long-term safety and effectiveness of advanced therapy medicinal products

Or. en

Amendment 618

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Annex I – Chapter VI – title

Motion for a resolutionAmendment
VI PATIENT SUPPORT AND SOCIAL INCLUSIONVI PATIENT AND FAMILY SUPPORT AND SOCIAL INCLUSION

Or. en

Amendment 619

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 13 – point 1

Motion for a resolutionAmendment
1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support.1. Member States shall be supported, both financially and technically, in developing multidisciplinary care pathways, including psychosocial and mental health support for patients, caregivers, and families across the lifespan, with a particular focus on significant life stages, including transition from paediatric to adult care, pregnancy, and hormonal transitions.

Or. en

Amendment 620

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 13 – point 1

Motion for a resolutionAmendment
1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support.1. Member States shall be supported in developing holistic, patient-centred, multidisciplinary care pathways, including psychosocial and mental health support for both patients and their families, taking into account the needs of individuals living with undiagnosed rare conditions and of those without access to effective treatment.

Or. en

Amendment 621

Victor Negrescu

Motion for a resolution

Article 13 – point 1

Motion for a resolutionAmendment
1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support.1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support, as well as appropriate follow-up, patient monitoring and coordination of care across different stages of life, in particular during transitions between paediatric and adult services.

Or. en

Amendment 622

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 13 – point 1

Motion for a resolutionAmendment
1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support.1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support, educational and employment support and measures for caregivers, including respite care and socio-economic support.

Or. en

Amendment 623

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 13 – point 1

Motion for a resolutionAmendment
1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support.1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support, and ensuring the integration of primary care practitioners as key entry points into the healthcare system.

Or. en

Amendment 624

Kateřina Konečná

Motion for a resolution

Article 13 – point 1

Motion for a resolutionAmendment
1. Member States shall be supported in developing multidisciplinary care pathways, including psychosocial and mental health support.1. Member States shall be supported in developing multidisciplinary, person-centred and integrated care pathways, including psychosocial and mental health support.

Or. en

Amendment 625

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 13 – point 1 a (new)

Motion for a resolutionAmendment
1 a. Member States shall be supported in training healthcare professionals and raising awareness about the specific needs of different rare diseases, including the lifelong nature of many rare diseases, varying treatment and care requirements across different life stages and the continuity of pharmacological and non-pharmacological treatments across adulthood.

Or. en

Amendment 626

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Article 13 – point 1 a (new)

Motion for a resolutionAmendment
1 a. Member States shall be supported in developing adapted care pathways for children with rare diseases, including coordinated paediatric care, support for families and continuity of care into adulthood.

Or. en

Amendment 627

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek, Viktória Ferenc

Motion for a resolution

Article 13 – point 1 b (new)

Motion for a resolutionAmendment
1 b. Member States shall be supported in strengthening their support mechanisms for carers, including financial assistance, access to social protection and adapted care services.

Or. en

Amendment 628

Kateřina Konečná

Motion for a resolution

Article 13 – point 2

Motion for a resolutionAmendment
2. The Commission shall establish a plan to coordinate the elaboration, publication and update of guidelines on patient journeys according to the state of the art, with ERN’s and patient organisations advice and participation. Paediatric patient journeys shall take priority in the design of the implementation of those guidelines.2. The Commission shall build on the recommendations and guidelines developed by the Joint Action on ERN Integration into Healthcare Systems (JARDIN), notably those on generic healthcare pathway and case management to establish a plan to support and coordinate the elaboration by Member States of generic care pathways for rare and complex conditions, in partnership with patient organisations, clinicians and other relevant stakeholders. Patient organisations shall receive adequate funding to fulfill this task and be formally engaged in this effort

Or. en

Amendment 629

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 13 – point 2

Motion for a resolutionAmendment
2. The Commission shall establish a plan to coordinate the elaboration, publication and update of guidelines on patient journeys according to the state of the art, with ERN’s and patient organisations advice and participation. Paediatric patient journeys shall take priority in the design of the implementation of those guidelines.2. The Commission shall provide support to ERNs to coordinate the elaboration, publication and update of guidelines on patient journeys according to the state of the art, with ERN’s and patient organisations advice and participation. Paediatric patient journeys shall take priority in the design of the implementation of those guidelines.

Or. en

Amendment 630

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14

Motion for a resolutionAmendment
Article 14deleted
Cross-border continuity of care
1. Cross-border exchange of hospital exemption medicinal products may be authorised when patient mobility is not feasible due to medical condition, treatment duration, or when the expected patient population in the receiving Member State so requires.
2. The receiving hospital may apply for a hospital exemption authorisation in its own Member State, provided that it demonstrates its capacity to replicate the treatment in accordance with the same quality, safety, and process standards as those applied in the originating Member State.
3. In the circumstances referred to in paragraph 1, the competent national regulatory authority of the receiving Member State may allow the cross-border transfer of the hospital exemption-advanced therapy medicinal product under the hospital exemption framework and designate a treating reference centre in the receiving Member State, subject to the authorisation of the competent national regulatory authorities of the originating Member State for the production and export of the product.
4. This shall not require that all manufacturing activities be carried out within a single Member State, provided that the originating site serves as the reference model for quality, manufacturing, and process standards.
5. Manufacturing activities may be outsourced to the originating Member State. Regulatory responsibility shall remain with the medical practitioner and the hospital requesting the hospital exemption in the receiving Member State, in cooperation with its competent national regulatory authority.
6. The implementation of this Article shall be subject to:
(a) the conduct of appropriate comparability studies;
(b) the use of harmonised clinical and quality protocols; and
(c) the capacity to aggregate and analyse clinical data jointly with the data generated by the hospital holding the original hospital exemption authorisation, in compliance with Union data protection rules.
7. Member States shall ensure that the implementation of this Article does not undermine patient safety, data integrity, or the non-commercial nature of the hospital exemption framework.

Or. en

Amendment 631

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 1

Motion for a resolutionAmendment
1. Cross-border exchange of hospital exemption medicinal products may be authorised when patient mobility is not feasible due to medical condition, treatment duration, or when the expected patient population in the receiving Member State so requires.deleted

Or. en

Amendment 632

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek

Motion for a resolution

Article 14 – point 1

Motion for a resolutionAmendment
1. Cross-border exchange of hospital exemption medicinal products may be authorised when patient mobility is not feasible due to medical condition, treatment duration, or when the expected patient population in the receiving Member State so requires.1. Member States may, on a voluntary basis, cooperate to facilitate access to advanced therapy medicinal products authorised under the hospital exemption framework, where this is justified by unmet medical needs or where patient mobility is not feasible.

Or. en

Amendment 633

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 2

Motion for a resolutionAmendment
2. The receiving hospital may apply for a hospital exemption authorisation in its own Member State, provided that it demonstrates its capacity to replicate the treatment in accordance with the same quality, safety, and process standards as those applied in the originating Member State.deleted

Or. en

Amendment 634

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Ondřej Knotek

Motion for a resolution

Article 14 – point 2

Motion for a resolutionAmendment
2. The receiving hospital may apply for a hospital exemption authorisation in its own Member State, provided that it demonstrates its capacity to replicate the treatment in accordance with the same quality, safety, and process standards as those applied in the originating Member State.2. Member States may, on a voluntary basis, cooperate to facilitate access to advanced therapy medicinal products authorised under the hospital exemption framework, where this is justified by unmet medical needs and where patient mobility is not feasible.

Or. en

Amendment 635

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 3

Motion for a resolutionAmendment
3. In the circumstances referred to in paragraph 1, the competent national regulatory authority of the receiving Member State may allow the cross-border transfer of the hospital exemption-advanced therapy medicinal product under the hospital exemption framework and designate a treating reference centre in the receiving Member State, subject to the authorisation of the competent national regulatory authorities of the originating Member State for the production and export of the product.deleted

Or. en

Amendment 636

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge

Motion for a resolution

Article 14 – point 3

Motion for a resolutionAmendment
3. In the circumstances referred to in paragraph 1, the competent national regulatory authority of the receiving Member State may allow the cross-border transfer of the hospital exemption-advanced therapy medicinal product under the hospital exemption framework and designate a treating reference centre in the receiving Member State, subject to the authorisation of the competent national regulatory authorities of the originating Member State for the production and export of the product.3. Cross-border use or replication of hospital exemption therapies shall only be permitted where:
(a) the competent authorities of both the originating and receiving Member States have granted prior authorisation;
(b) the receiving healthcare provider demonstrates its capacity to ensure equivalent standards of quality, safety and traceability;
(c) the non-routine and non-commercial nature of the hospital exemption framework is preserved.

Or. en

Amendment 637

Billy Kelleher, Stine Bosse

Motion for a resolution

Article 14 – point 4

Motion for a resolutionAmendment
4. This shall not require that all manufacturing activities be carried out within a single Member State, provided that the originating site serves as the reference model for quality, manufacturing, and process standards.deleted

Or. en

Amendment 638

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 4

Motion for a resolutionAmendment
4. This shall not require that all manufacturing activities be carried out within a single Member State, provided that the originating site serves as the reference model for quality, manufacturing, and process standards.deleted

Or. en

Amendment 639

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge

Motion for a resolution

Article 14 – point 4

Motion for a resolutionAmendment
4. This shall not require that all manufacturing activities be carried out within a single Member State, provided that the originating site serves as the reference model for quality, manufacturing, and process standards.4. The Commission shall support the exchange of best practices and may develop non-binding guidance to facilitate cooperation between Member States, including on regulatory procedures, data exchange and patient safety requirements.

Or. en

Amendment 640

Billy Kelleher, Stine Bosse

Motion for a resolution

Article 14 – point 5

Motion for a resolutionAmendment
5. Manufacturing activities may be outsourced to the originating Member State. Regulatory responsibility shall remain with the medical practitioner and the hospital requesting the hospital exemption in the receiving Member State, in cooperation with its competent national regulatory authority.deleted

Or. en

Amendment 641

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 5

Motion for a resolutionAmendment
5. Manufacturing activities may be outsourced to the originating Member State. Regulatory responsibility shall remain with the medical practitioner and the hospital requesting the hospital exemption in the receiving Member State, in cooperation with its competent national regulatory authority.deleted

Or. en

Amendment 642

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 14 – point 5

Motion for a resolutionAmendment
5. Manufacturing activities may be outsourced to the originating Member State. Regulatory responsibility shall remain with the medical practitioner and the hospital requesting the hospital exemption in the receiving Member State, in cooperation with its competent national regulatory authority.5. Any exchange of clinical data, manufacturing protocols or patient information under this Article shall be subject to strict compliance with Union data protection law, including explicit, informed and freely given patient consent, and shall ensure that Member States retain control over sensitive health data.

Or. en

Amendment 643

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 6

Motion for a resolutionAmendment
6. The implementation of this Article shall be subject to:deleted
(a) the conduct of appropriate comparability studies;
(b) the use of harmonised clinical and quality protocols; and
(c) the capacity to aggregate and analyse clinical data jointly with the data generated by the hospital holding the original hospital exemption authorisation, in compliance with Union data protection rules.

Or. en

Amendment 644

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 14 – point 6 – introductory part

Motion for a resolutionAmendment
6. The implementation of this Article shall be subject to:6. This Article shall not create a new Union authorisation pathway and shall not replace or circumvent existing Union procedures for the marketing authorisation of medicinal products.

Or. en

Amendment 645

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 6 – point a

Motion for a resolutionAmendment
(a) the conduct of appropriate comparability studies;deleted

Or. en

Amendment 646

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 14 – point 6 – point a

Motion for a resolutionAmendment
(a) the conduct of appropriate comparability studies;deleted

Or. en

Amendment 647

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 14 – point 6 – point b

Motion for a resolutionAmendment
(b) the use of harmonised clinical and quality protocols; anddeleted

Or. en

Amendment 648

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 6 – point b

Motion for a resolutionAmendment
(b) the use of harmonised clinical and quality protocols; anddeleted

Or. en

Amendment 649

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 14 – point 6 – point c

Motion for a resolutionAmendment
(c) the capacity to aggregate and analyse clinical data jointly with the data generated by the hospital holding the original hospital exemption authorisation, in compliance with Union data protection rules.deleted

Or. en

Amendment 650

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 6 – point c

Motion for a resolutionAmendment
(c) the capacity to aggregate and analyse clinical data jointly with the data generated by the hospital holding the original hospital exemption authorisation, in compliance with Union data protection rules.deleted

Or. en

Amendment 651

Elena Nevado del Campo, Dolors Montserrat, Letizia Moratti

Motion for a resolution

Article 14 – point 7

Motion for a resolutionAmendment
7. Member States shall ensure that the implementation of this Article does not undermine patient safety, data integrity, or the non-commercial nature of the hospital exemption framework.deleted

Or. en

Amendment 652

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 15 – point 1

Motion for a resolutionAmendment
1. A permanent European rare disease coordination board (the ‘Coordination Board’) is hereby established. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing the structure and functionality of that board.1. A permanent European rare disease coordination board (the ‘Coordination Board’) is hereby established.

Or. en

Amendment 653

Kateřina Konečná

Motion for a resolution

Article 15 – point 1

Motion for a resolutionAmendment
1. A permanent European rare disease coordination board (the ‘Coordination Board’) is hereby established. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing the structure and functionality of that board.1. A permanent European rare disease coordination board (the ‘Coordination Board’) is hereby established. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing the structure and functionality and role of that board.

Or. en

Amendment 654

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 15 – point 1 a (new)

Motion for a resolutionAmendment
1 a. The responsibilities of the Coordination Board shall include: (a) periodically review the implementation and impact of existing legislation to OMP research and delivery to make recommendations, including necessary legislative and non-legislative reforms; (b) undertake a study to identify regulatory simplification and guidelines to support the approval of OMPs in consultation with the EMA and other relevant stakeholders; (c) commission a study of the core barriers faced by Union citizens, beyond pricing and reimbursement negotiations and affordability, preventing pharmaceuticals from reaching people living with a rare disease at the right time; (d) collaborate with the Coordination Board established in the proposal for a Regulation of the European Parliament and of the Council on establishing a framework of measures for strengthening Union's biotechnology and biomanufacturing sectors particular in the area of health and amending Regulations (EC) No 178/2002, (EC) No 1394/2007, (EU) No 536/2014, (EU) 2019/6, (EU) 2024/795 and (EU) 2024/1938 (European Biotech Act) to identify strategic projects and public-private partnerships that can support the develop infrastructure or research collaboration. Prepare a report on the rare disease ecosystem at national level every two years, including the various incentives and support provided to be presented to the NCAPR and Pharmaceutical Committee in order to share best practices.

Or. en

Amendment 655

Billy Kelleher, Stine Bosse

Motion for a resolution

Article 15 – point 2

Motion for a resolutionAmendment
2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board..2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, representatives of the industry and industry associations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board.

Or. en

Amendment 656

Michalis Hadjipantela

Motion for a resolution

Article 15 – point 2

Motion for a resolutionAmendment
2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board..2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, representatives of the industry, industry associations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board.

Or. en

Amendment 657

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 15 – point 2

Motion for a resolutionAmendment
2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board..2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, caregiver organisations, researchers, the European Medicines Agency, HERA, and the Commission. The representative of the Commission shall be the Chair of the Coordination Board..

Or. en

Amendment 658

Letizia Moratti

Motion for a resolution

Article 15 – point 2

Motion for a resolutionAmendment
2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board..2. The Coordination Board shall include representatives of Member States, ERNs, industry representatives, patient organisations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board.

Or. en

Amendment 659

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 15 – point 2

Motion for a resolutionAmendment
2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, researchers and the Commission. The representative of the Commission shall be the Chair of the Coordination Board..2. The Coordination Board shall include representatives of Member States, ERNs, patient organisations, researchers and the Commission. A representative of the Commission shall be the Chair of the Coordination Board.

Or. en

Amendment 660

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 15 – point 3

Motion for a resolutionAmendment
3. The Coordination Board shall establish the priorities in the development of the ERNs, and its joint work programme.deleted

Or. en

Amendment 661

Kateřina Konečná

Motion for a resolution

Article 15 – point 3

Motion for a resolutionAmendment
3. The Coordination Board shall establish the priorities in the development of the ERNs, and its joint work programme.3. The Coordination Board shall establish-amongst others- the priorities in the development of the ERNs, and its joint work programme. It shall support the Commission in the implementation of this Regulation in all relevant decisions stemming from this Regulation, as laid out in the delegated and implementing acts. The Board shall, in particular, support and advise the Commission in the drafting of the EU Action Plan for Rare Diseases.

Or. en

Amendment 662

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 15 – point 3

Motion for a resolutionAmendment
3. The Coordination Board shall establish the priorities in the development of the ERNs, and its joint work programme.3. The Coordination Board shall establish the priorities in the development of the ERNs, and its joint work programme, as well as update and maintain the common data standards and coding systems necessary to the functioning of the registries referred to in Article 10.

Or. en

Amendment 663

Emmanouil Fragkos, Galato Alexandraki, Geadis Geadi

Motion for a resolution

Article 15 – point 3

Motion for a resolutionAmendment
3. The Coordination Board shall establish the priorities in the development of the ERNs, and its joint work programme.3. The Coordination Board shall advise the Commission on strategic priorities, monitoring indicators and the coordination of relevant Union action.

Or. el

Amendment 664

Letizia Moratti

Motion for a resolution

Article 15 – point 3 a (new)

Motion for a resolutionAmendment
3 a. The responsibilities of the Coordination Board shall include:
(a) periodically reviewing the implementation and impact of existing legislation on orphan drug development and the wider rare disease ecosystem;
(b) developing recommendations to streamline regulatory guidelines and processes to accelerate orphan drug approvals;
(c) assessing current gaps in cross-border access to healthcare.

Or. en

Amendment 665

Stine Bosse, Billy Kelleher, Vlad Vasile-Voiculescu

Motion for a resolution

Article 15 – point 3 a (new)

Motion for a resolutionAmendment
3 a. The Coordination Board shall consider and initiate novel trust-based cooperation models, within or complementary to existing structures such as the European Reference Networks (ERNs), aimed at overcoming the patient and data volume issue, developing clinical expertise, and facilitating real-time data exchange to improve patient outcomes.

Or. en

Amendment 666

Kateřina Konečná

Motion for a resolution

Article 15 – point 3 a (new)

Motion for a resolutionAmendment
3 a. The patient participation in the Board have equal voting rights as other stakeholders and their participation shall be financially supported by the Coordination Board.

Or. en

Amendment 667

Michalis Hadjipantela

Motion for a resolution

Article 15 a (new)

Motion for a resolutionAmendment
Article15a
European Rare Disease Coordination Agency
1. The Commission shall assess, within [18 months of the date of entry into force of this Regulation], the feasibility of establishing a dedicated European Rare Disease Coordination Agency (the 'Agency') to serve as the central institutional anchor for the implementation of this Regulation.
2. The feasibility assessment shall consider:
(a) the consolidation of existing rare disease-related mandates, including those of the EMA Committee for Orphan Medicinal Products, the ERN governance structure and the European Rare Disease Research Alliance (ERDERA), within or alongside the Agency, with a view to reducing institutional fragmentation and duplicated expenditure;
(b) the Agency's functions in coordinating the European diagnostic coordination mechanism established under Article 3, the Union Newborn Screening Panel guidance, the European rare disease research agenda established under Article 8, the Coordination Board established under Article 15, and the EU rare diseases benchmark established under Article 16;
(c) appropriate governance arrangements ensuring representation of Member States, patient organisations, healthcare professionals, researchers and industry, and regular accountability reporting to the European Parliament and the Council;
(d) funding options, including through rationalisation of existing Union expenditure on rare disease-related coordination activities.
3. On the basis of the feasibility assessment, the Commission shall, where appropriate, submit a legislative proposal for the establishment of the Authority.

Or. en

Amendment 668

Kateřina Konečná

Motion for a resolution

Article 16 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing an EU rare diseases benchmark.1. The Commission shall adopt a communication supplementing this Regulation, that shall establish an EU rare diseases action plan including a European benchmark.

Or. en

Amendment 669

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 16 – point 1

Motion for a resolutionAmendment
1. The Commission shall adopt a delegated act in accordance with Article 18 in order to supplement this Regulation by establishing an EU rare diseases benchmark.1. The Commission shall adopt guidelines, targets and indicators in order to supplement this legislative act by establishing an EU rare diseases benchmark.

Or. en

Amendment 670

Kateřina Konečná

Motion for a resolution

Article 16 – point 2 – introductory part

Motion for a resolutionAmendment
2. The EU rare diseases benchmark shall measure and monitor performance across five strategic pillars:2. The EU rare diseases benchmark shall propose measures and monitor performance across seven strategic pillars:

Or. en

Amendment 671

András Tivadar Kulja

Motion for a resolution

Article 16 – point 2 – point a

Motion for a resolutionAmendment
(a) diagnosis and early detection;(a) diagnosis, newborn screening, and early detection;

Or. en

Amendment 672

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 16 – point 2 – point a

Motion for a resolutionAmendment
(a) diagnosis and early detection;(a) diagnosis, newborn screening, and early detection;

Or. en

Amendment 673

Michalis Hadjipantela

Motion for a resolution

Article 16 – point 2 – point a

Motion for a resolutionAmendment
(a) diagnosis and early detection;(a) diagnosis, newborn screening and early detection;

Or. en

Amendment 674

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 16 – point 2 – point a

Motion for a resolutionAmendment
(a) diagnosis and early detection;(a) screening, diagnosis and early detection;

Or. en

Amendment 675

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 16 – point 2 – point b

Motion for a resolutionAmendment
(b) access to treatment and care;(b) access to orphan medicinal products, advanced therapy medicinal products, treatment and care;

Or. en

Amendment 676

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 16 – point 2 – point e

Motion for a resolutionAmendment
(e) patient-centered outcomes and social support.(e) patient-centered outcomes and social support, including quality of life, participation in education and the workforce, and financial impact on households.

Or. en

Amendment 677

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 16 – point 2 – point e

Motion for a resolutionAmendment
(e) patient-centered outcomes and social support.(e) patient-centered outcomes and social support, including indicators to monitor the socio-economic wellbeing of patients and caregivers.

Or. en

Amendment 678

Kateřina Konečná

Motion for a resolution

Article 16 – point 2 – point e a (new)

Motion for a resolutionAmendment
(e a) social inclusion and social support for people living with rare diseases and families;

Or. en

Amendment 679

Kateřina Konečná

Motion for a resolution

Article 16 – point 2 – point e b (new)

Motion for a resolutionAmendment
(e b) health data sharing, registries and digital infrastructure.

Or. en

Amendment 680

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 16 – point 2 a (new)

Motion for a resolutionAmendment
2 a. Indicators under points (a) and (b) shall include equity measures capturing within- and between-Member-State disparities in access to genomic diagnostics, newborn screening follow-up, and timeliness of care.

Or. en

Amendment 681

Billy Kelleher, Stine Bosse

Motion for a resolution

Article 17

Motion for a resolutionAmendment
Article 17deleted
Eligibility conditions for rare disease funding under the NRPP
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.
2. For the purposes of paragraph 1, Union benchmark standards shall include, as a minimum:
(a) defined screening panels based on scientific evidence, clinical utility, and public health relevance;
(b) nationwide or regionally coordinated coverage ensuring equitable access;
(c) quality assurance and accreditation mechanisms;
(d) effective referral pathways for confirmatory diagnosis and specialised care; and
(e) compliance with Union law on data protection, informed consent, and non-discrimination.
3. Member States applying for support under NRPP shall demonstrate effective implementation or present a binding implementation plan with a defined timeline to achieve compliance with the standards referred to in paragraph 2.
4. The Commission shall assess compliance with this Article prior to the allocation of funds and may suspend, reduce, or condition financial support where the requirements set out herein are not fulfilled.
5. That conditionality shall aim to ensure that Union funding for rare diseases contributes to early detection, reduction of diagnostic delay, and convergence of standards of care across the Union.

Or. en

Amendment 682

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 17 – title

Motion for a resolutionAmendment
Eligibility conditions for rare disease funding under the NRPPSupport for rare disease screening, coordination and long-term care under the NRPP

Or. en

Amendment 683

François-Xavier Bellamy

Motion for a resolution

Article 17 – point 1

Motion for a resolutionAmendment
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional newborn screening programme. Funding for genetic or prenatal screening shall be limited to cases where there is a clear therapeutic or clinical benefit and shall not support practices aimed at selection based on genetic characteristics, in line with the Union’s obligations under the United Nations Convention on the Rights of Persons with Disabilities and the recommendations of the Committee on the Rights of Persons with Disabilities.

Or. en

Amendment 684

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 17 – point 1

Motion for a resolutionAmendment
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence and implementation of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established and implemented in accordance with Union benchmark standards and the Union guidance setting out the harmonised best-in-class Union newborn screening panel, and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.

Or. en

Amendment 685

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 17 – point 1

Motion for a resolutionAmendment
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.1. Member States shall be encouraged to include in their NRPP strategic projects, reforms, investments and other interventions in the field of rare diseases aimed at supporting early detection, reducing diagnostic delay and strengthening long-term care services. Such measures may include, where appropriate, newborn screening programmes, prenatal genetic screening frameworks, coordination programmes, referral pathways, specialised care structures and long-term care services, in accordance with national priorities and competences.

Or. en

Amendment 686

András Tivadar Kulja

Motion for a resolution

Article 17 – point 1

Motion for a resolutionAmendment
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence and implementation of a national or regional screening programme of newborns, established and implemented in accordance with Union benchmark standards and the Union guidance setting out an exemplary Union newborn screening panel, and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.

Or. en

Amendment 687

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 17 – point 1

Motion for a resolutionAmendment
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall support the existence and implementation of evidence-based national or regional screening programmes of newborns and, where applicable, prenatal genetic screening frameworks, established in accordance with Union benchmark standards.

Or. en

Amendment 688

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 17 – point 1

Motion for a resolutionAmendment
1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be conditional upon the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.1. Access to financial support under the NRPP for strategic projects in the field of rare diseases shall be facilitated by the existence of a national or regional screening programme of newborns and, where applicable, a prenatal genetic screening framework, established in accordance with Union benchmark standards.

Or. en

Amendment 689

Kateřina Konečná

Motion for a resolution

Article 17 – point 1 a (new)

Motion for a resolutionAmendment
1 a. The Union recognises that targeted investment in early diagnosis pathways is essential to:
(a) safeguard the fiscal sustainability of healthcare systems by reducing long-term palliative and chronic care expenditure;
(b) enhance workforce stability and labour pool participation by preventing early-onset disability and mitigating the economic inactivity of informal caregivers and families;
(c) foster high-value innovation in the European biotechnology and diagnostic sectors, in line with the Union's industrial strategy.

Or. en

Amendment 690

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 17 – point 2 – introductory part

Motion for a resolutionAmendment
2. For the purposes of paragraph 1, Union benchmark standards shall include, as a minimum:2. In developing such measures, Member States shall be encouraged to take into account, where relevant, Union benchmark standards, including:

Or. en

Amendment 691

Kateřina Konečná

Motion for a resolution

Article 17 – point 2 – point d

Motion for a resolutionAmendment
(d) effective referral pathways for confirmatory diagnosis and specialised care; and(d) effective referral pathways for confirmatory diagnosis and specialised care at national level and within European Reference Networks;

Or. en

Amendment 692

Kateřina Konečná

Motion for a resolution

Article 17 – point 2 – point d a (new)

Motion for a resolutionAmendment
(d a) existence of pathways of clinical and psychosocial care for undiagnosed population; and

Or. en

Amendment 693

Kateřina Konečná

Motion for a resolution

Article 17 – point 2 – point d b (new)

Motion for a resolutionAmendment
(d b) full technical interoperability of screening data within the European Health Data Space (EHDS), ensuring compliance with Regulation (EU) 2016/679.

Or. en

Amendment 694

François-Xavier Bellamy

Motion for a resolution

Article 17 – point 2 – point e

Motion for a resolutionAmendment
(e) compliance with Union law on data protection, informed consent, and non-discrimination.(e) compliance with Union law on data protection, informed consent, non-discrimination, and respect for the dignity and rights of persons with disabilities, including avoiding practices aimed at selection based on genetic characteristics.

Or. en

Amendment 695

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 17 – point 3

Motion for a resolutionAmendment
3. Member States applying for support under NRPP shall demonstrate effective implementation or present a binding implementation plan with a defined timeline to achieve compliance with the standards referred to in paragraph 2.3. Union financial support under the NRPP shall contribute to early detection, the reduction of diagnostic delay and the strengthening of long-term care and coordination for persons living with rare diseases, while leaving the design and implementation of the relevant measures to the discretion of Member States.

Or. en

Amendment 696

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 17 – point 3

Motion for a resolutionAmendment
3. Member States applying for support under NRPP shall demonstrate effective implementation or present a binding implementation plan with a defined timeline to achieve compliance with the standards referred to in paragraph 2.3. Member States applying for support under NRPP for projects in the field of rare diseases shall demonstrate effective implementation or present an implementation plan with a defined timeline to achieve compliance with the standards referred to in paragraph 2.

Or. en

Amendment 697

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 17 – point 3

Motion for a resolutionAmendment
3. Member States applying for support under NRPP shall demonstrate effective implementation or present a binding implementation plan with a defined timeline to achieve compliance with the standards referred to in paragraph 2.3. Member States applying for support under NRPP shall demonstrate effective implementation or present a coherent implementation plan with a defined timeline to achieve compliance with the standards referred to in paragraph 2.

Or. en

Amendment 698

François-Xavier Bellamy

Motion for a resolution

Article 17 – point 3 a (new)

Motion for a resolutionAmendment
3 a. Member States shall ensure that no genetic or prenatal screening programme leads, directly or indirectly, to discriminatory practices against persons with disabilities or to pressure on parents to make decisions based on the presence of a disability. Particular attention shall be given to ensuring that conditions such as Down syndrome are addressed within a framework that respects the equal dignity and rights of persons living with such conditions, and that avoids any form of systematic targeting or selection. All genetic and prenatal testing shall be accompanied by comprehensive, balanced and non-directive counselling, including information on living with the condition concerned, available support, and the perspectives of persons with disabilities and their families.

Or. en

Amendment 699

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 17 – point 4

Motion for a resolutionAmendment
4. The Commission shall assess compliance with this Article prior to the allocation of funds and may suspend, reduce, or condition financial support where the requirements set out herein are not fulfilled.deleted

Or. en

Amendment 700

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 17 – point 4

Motion for a resolutionAmendment
4. The Commission shall assess compliance with this Article prior to the allocation of funds and may suspend, reduce, or condition financial support where the requirements set out herein are not fulfilled.4. The Commission shall assess compliance with this Article prior to the allocation of funds and may require Member States which require capacity building to reallocate financial support under the NRPP towards targeted technical assistance for newborn screening.

Or. en

Amendment 701

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 17 – point 4

Motion for a resolutionAmendment
4. The Commission shall assess compliance with this Article prior to the allocation of funds and may suspend, reduce, or condition financial support where the requirements set out herein are not fulfilled.4. The Commission shall assess compliance with this Article and provide support to Member States where the requirements set out herein are not fulfilled.

Or. en

Amendment 702

Marie-Luce Brasier-Clain, Margarita de la Pisa Carrión, Laurent Castillo, Aleksandar Nikolic, Valérie Deloge, Viktória Ferenc

Motion for a resolution

Article 17 – point 5

Motion for a resolutionAmendment
5. That conditionality shall aim to ensure that Union funding for rare diseases contributes to early detection, reduction of diagnostic delay, and convergence of standards of care across the Union.deleted

Or. en

Amendment 703

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 17 – point 5

Motion for a resolutionAmendment
5. That conditionality shall aim to ensure that Union funding for rare diseases contributes to early detection, reduction of diagnostic delay, and convergence of standards of care across the Union.deleted

Or. en

Amendment 704

Jessica Polfjärd, Niels Flemming Hansen

Motion for a resolution

Article 17 – point 5

Motion for a resolutionAmendment
5. That conditionality shall aim to ensure that Union funding for rare diseases contributes to early detection, reduction of diagnostic delay, and convergence of standards of care across the Union.5. The support shall aim to ensure that Union funding for rare diseases contributes to early detection, reduction of diagnostic delay, and convergence of standards of care across the Union.

Or. en

Amendment 705

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 18

Motion for a resolutionAmendment
Article 18deleted
Exercise of the delegation
1. The power to adopt delegated acts is conferred on the Commission subject to the conditions laid down in this Article.
2. The power to adopt delegated acts referred to in Articles 5, 11, 15 and 16 shall be conferred on the Commission for an indeterminate period of time from... [date of entry into force of this Regulation].
3. The delegation of power referred to in Articles 5, 11, 15 and 16 may be revoked at any time by the European Parliament or by the Council. A decision to revoke shall put an end to the delegation of the power specified in that decision. It shall take effect the day following the publication of the decision in the Official Journal of the European Union or at a later date specified therein. It shall not affect the validity of any delegated acts already in force.
4. Before adopting a delegated act, the Commission shall consult experts designated by each Member State in accordance with the principles laid down in the Interinstitutional Agreement of 13 April 2016 on Better Law-Making.
5. As soon as it adopts a delegated act, the Commission shall notify it simultaneously to the European Parliament and to the Council.
6. A delegated act adopted pursuant to Articles 5, 11, 15 and 16 shall enter into force only if no objection has been expressed either by the European Parliament or by the Council within a period of two months of notification of that act to the European Parliament and the Council or if, before the expiry of that period, the European Parliament and the Council have both informed the Commission that they will not object. That period shall be extended by two months at the initiative of the European Parliament or of the Council.

Or. en

Amendment 706

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 18 – point 4

Motion for a resolutionAmendment
4. Before adopting a delegated act, the Commission shall consult experts designated by each Member State in accordance with the principles laid down in the Interinstitutional Agreement of 13 April 2016 on Better Law-Making.4. Before adopting a delegated act, the Commission shall consult experts, including patient associations and healthcare professionals, designated by each Member State in accordance with the principles laid down in the Interinstitutional Agreement of 13 April 2016 on Better Law-Making.

Or. en

Amendment 707

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 19

Motion for a resolutionAmendment
Article 19deleted
Committee procedure
1. The Commission shall be assisted by a committee. That committee shall be a committee within the meaning of Regulation (EU) No 182/2011.
2. Where reference is made to this paragraph, Article 4 of Regulation (EU) No 182/2011 shall apply.

Or. en

Amendment 708

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 19 a (new)

Motion for a resolutionAmendment
Article19a
Where appropriate, the committee shall consult with patients, medicine and medical technology developers, healthcare professionals, industries or other stakeholders as relevant.

Or. en

Amendment 709

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 20 – point 1

Motion for a resolutionAmendment
1. Support for rare diseases under the NRPP in the post-2027 Multiannual Financial Framework (2028-2034).1. The Union shall support innovation and research in rare diseases as well as equal access to diagnosis and treatment, including in cross- border situations, digital health infrastructure and cross-border cooperation, through several funding instruments in the post-2027 Multiannual Financial Framework (2028-2034), including Horizon and the European Competitiveness Fund with a particular attention to reducing disparities between and within Member States.

Or. en

Amendment 710

Elena Nevado del Campo, Dolors Montserrat, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 20 – point 1

Motion for a resolutionAmendment
1. Support for rare diseases under the NRPP in the post-2027 Multiannual Financial Framework (2028-2034).1. Support for rare diseases under the European Competitiveness Fund, Horizon Europe and the NRPP in the post-2027 Multiannual Financial Framework (2028-2034).

Or. en

Amendment 711

Kateřina Konečná

Motion for a resolution

Article 20 – point 1

Motion for a resolutionAmendment
1. Support for rare diseases under the NRPP in the post-2027 Multiannual Financial Framework (2028-2034).1. Support for rare diseases under the NRPP and under other Union funding programmes in the post-2027 Multiannual Financial Framework (2028-2034).

Or. en

Amendment 712

Billy Kelleher, Stine Bosse

Motion for a resolution

Article 20 – point 2

Motion for a resolutionAmendment
2. For the duration of the post-2027 Multiannual Financial Framework, strategic projects in the field of rare diseases may be supported under the NRPP. Such support shall aim to strengthen early diagnosis, equitable access to treatment, research capacity, digital health infrastructure, and cross-border cooperation, with particular attention to reducing disparities between and within Member States.deleted

Or. en

Amendment 713

Elena Nevado del Campo, Dolors Montserrat, Tomislav Sokol, Adam Jarubas, Letizia Moratti

Motion for a resolution

Article 20 – point 2

Motion for a resolutionAmendment
2. For the duration of the post-2027 Multiannual Financial Framework, strategic projects in the field of rare diseases may be supported under the NRPP. Such support shall aim to strengthen early diagnosis, equitable access to treatment, research capacity, digital health infrastructure, and cross-border cooperation, with particular attention to reducing disparities between and within Member States.2. For the duration of the post-2027 Multiannual Financial Framework, support for rare disease may be provided through different Union instruments, in accordance with their respective objectives and legal bases. Horizon Europe may support research and innovation activities, the European Competitiveness Fund may support innovation, strategic technologies and industrial scale-up, the NRPPs may support strategic projects, reforms, investments and other interventions aimed at strengthening healthcare-related measures, including early diagnosis, equitable access to treatment, research capacity, digital health infrastructure, coordination and long-term care services.

Or. en

Amendment 714

Nicolae Ștefănuță

on behalf of the Verts/ALE Group

Motion for a resolution

Article 20 – point 2

Motion for a resolutionAmendment
2. For the duration of the post-2027 Multiannual Financial Framework, strategic projects in the field of rare diseases may be supported under the NRPP. Such support shall aim to strengthen early diagnosis, equitable access to treatment, research capacity, digital health infrastructure, and cross-border cooperation, with particular attention to reducing disparities between and within Member States.2. For the duration of the post-2027 Multiannual Financial Framework, Member States shall ensure that strategic projects in the field of rare diseases are supported under the NRPP, via the European Competitiveness Fund and its Health/Biotech/Agriculture/Bioeconomy window, and where relevant bolstered by Horizon Europe, the European Social Fund Plus and cohesion policy funds. Such support shall aim to strengthen early diagnosis, equitable access to treatment, research capacity, digital health infrastructure, and cross-border cooperation, with particular attention to reducing disparities between and within Member States.

Or. en

Amendment 715

Billy Kelleher, Stine Bosse

Motion for a resolution

Article 20 – point 3

Motion for a resolutionAmendment
3. Funding under the NRPP may complement and create synergies with Union programmes, provided that such support remains consistent with the objectives and legal bases of those programmes.deleted

Or. en

Amendment 716

Kateřina Konečná

Motion for a resolution

Article 20 – point 4

Motion for a resolutionAmendment
4. Priority shall be given to projects delivering measurable Union added value, including the development of interoperable rare disease registries, reinforcement of specialised centres and networks, support for clinical research in small populations, and deployment of secure cross-border data systems. Implementation shall be subject to monitoring and evaluation to ensure transparency, effectiveness, and alignment with Union health objectives.4. Under the current and future Multiannual Financial Frameworks, infrastructures and initiatives with clear EU added value shall continue to be financed through Union centralised funding programmes. Priority shall be given to projects delivering measurable Union added value, including the support to Orphanet, the support to the functioning of the European Reference Networks, the development of interoperable rare disease registries, reinforcement of specialised centres and networks, support for clinical research in small populations, and deployment of secure crossborder data systems, and the support to the functioning of patients organisations.
Implementation shall be subject to monitoring and evaluation to ensure transparency, effectiveness, and alignment with Union health objectives

Or. en

Amendment 717

Letizia Moratti

Motion for a resolution

Article 20 – point 4 a (new)

Motion for a resolutionAmendment
4 a. Support for genetic and genomic databases and biobanks shall be included, recognising their critical relevance for rare disease research, as well as the creation and use of synthetic data, in line with the provisions referred to in Articles 5 and 9, in order to ensure a systematic approach to the use of these resources across the entire regulatory framework.

Or. en

Amendment 718

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 21 – point 1

Motion for a resolutionAmendment
1. By ....[2 years after the date of entry into force of this Regulation] and every two years thereafter, the Commission shall submit a report to the European Parliament and to the Council on implementation progress of this Regulation.1. By ....[2 years after the date of entry into force of this Regulation] and every two years thereafter, the Commission shall submit a report to the European Parliament and to the Council on implementation progress of this legislative act.

Or. en

Amendment 719

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 21 – point 2

Motion for a resolutionAmendment
2. The Commission shall evaluate this Regulation by...[five years after the date of entry into force of this Regulation].2. The Commission shall evaluate this legislative act by...[five years after the date of entry into force of this legal act].

Or. en

Amendment 720

Billy Kelleher, Vlad Vasile-Voiculescu, Stine Bosse

Motion for a resolution

Article 22 – paragraph 1

Motion for a resolutionAmendment
This Regulation shall enter into force on the twentieth day following that of its publication in the Official Journal of the European Union.This legislative act shall enter into force on the twentieth day following that of its publication in the Official Journal of the European Union.

Or. en