What changed between the draft committee report and the plenary report

DRAFT EUROPEAN PARLIAMENT LEGISLATIVE RESOLUTION

7 unchanged paragraphs

on the proposal for a regulation of the European Parliament and of the Council laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006

(COM(2023)0193 – C90144/2023 – 2023/0131(COD))

(Ordinary legislative procedure: first reading)

The European Parliament,

– having regard to the Commission proposal to Parliament and the Council (COM(2023)0193),

– having regard to Article 294(2) and Article 114 and Article 168(4), point (c), of the Treaty on the Functioning of the European Union, pursuant to which the Commission submitted the proposal to Parliament (C90144/2023),

– having regard to Article 294(3) of the Treaty on the Functioning of the European Union,

Changed:– having regard to the opinion of the European Economic and Social Committee of ...,25 October 2023,

Change 1

Changed:– having regard to the opinionafter ofconsulting the Committee of the Regions of ...,Regions,

– having regard to Rule 59 of its Rules of Procedure,

– having regard to the opinion of the Committee on Industry, Research and Energy,

– having regard to the letters from the Committee on Budgets and the Committee on Agriculture and Rural Development,

Changed:– having regard to the report of the Committee on the Environment, Public Health and Food Safety (A90000/2023),(A9-0141/2024),

1. Adopts its position at first reading hereinafter set out;

2. Calls on the Commission to refer the matter to Parliament again if it replaces, substantially amends or intends to substantially amend its proposal;

3. Instructs its President to forward its position to the Council, the Commission and the national parliaments.

Change 2

Added:Recital -1 (new): (-1) Ensuring that patients receive the medicines they need, when they need them, regardless of where they live in the Union, is a central objective of the European Health Union. Ensuring the competitiveness of the European pharmaceutical industry, whilst providing better availability of medicines and more equal and timely access for patients, is a key objective of the proposed Union pharmaceutical reform.

Added:Recital 1 a (new): (1a) This Regulation should contribute to the implementation of the One Health Approach, stressing the well-established interconnectedness between human, animal and ecosystem health, and the need to include those three dimensions when addressing public health threats. Environmental stress and degradation, including biodiversity loss, contribute to the transmission of diseases between, and the disease burden of, humans and animals. In addition, pollution from active pharmaceutical ingredients negatively affects the quality of waters and ecosystems, causes antimicrobial resistance to increase rapidly, posing risks to public health globally.

Added:Recital 2: (2) The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by aiming to create an attractive environment for research, development and production of medicinal products in the Union, along with a modern framework that makes innovative and established medicinal products available to patients and healthcare systems at affordable prices, while strengthening the fight against shortages of medicinal products and ensuring security of supply and addressing environmental concerns.

Added:Recital 2 a (new): (2a) To supplement the measures to address shortages of medicinal products, the communication of the Commission of 24 October 2023 entitled ‘Addressing medicine shortages in the EU’ aims to address critical shortages of medicines and strengthen security of supply in the Union by, among other things, introducing the launch of a European voluntary solidarity mechanism for medicines allowing Member States to redistribute their available stock in the event of shortages.

Added:Recital 3: (3) Addressing unequal patient access of medicinal products has become a key priority of the Pharmaceutical Strategy for Europe as has been highlighted by the Council and the European Parliament. Member States and the European Parliament have called for revised mechanisms and incentives for development of medicinal products tailored to the level of unmet medical need, while ensuring that the process is transparent, patient access and availability as well as affordability of medicinal products in all Member States.

Added:Recital 4: (4) Previous amendments to the Union pharmaceutical legislation have addressed access to medicinal products by providing for accelerated assessment for marketing authorisation applications or by allowing conditional marketing authorisation for medicinal products for unmet medical need. While these measures accelerated the authorisation of innovative and promising therapies in some areas, and many unaddressed public health priorities remain, these medicinal products do not always reach the patient and patients in the Union still have different levels of access to medicines.

Added:Recital 5: (5) The COVID-19 pandemic further underlined critical issues, which require a reform of the Union pharmaceuticals framework to strengthen its resilience, while improving the availability of medicinal products and to ensure that it corresponds to public health needs and serves the people under all circumstances.

Added:Recital 5 a (new): (5a) The COVID-19 pandemic also highlighted disparities in terms of, the capacity of health systems, national immunisation infrastructure, shortages and preparation. In addition to the measures in this Regulation, Member States should strengthen their national immunisation programmes, ensuring their population is better sufficiently protected against infectious diseases and strengthening pandemic preparedness and response.

Added:Recital 6: (6) It is therefore necessary to replace Regulation (EC) No 726/2004 of the European Parliament and of the Council38 with a new Regulation.

Recital 9: (9) As to the scope of this Regulation, the authorisation of antimicrobials is in the interest of patients' health at Union level and therefore it should be made possible to authorise them at Union level.

Change 3

Removed:Recital 30: (30) The Agency should be empowered to give scientific recommendations on whether a product under development, which could potentially fall under the mandatory scope of the centralised procedure, meets the scientific criteria to be a medicinal product. Such an advisory mechanism would address, as early as possible, questions related to borderline cases with other areas such as in particular substances of human origin, cosmetics or medical devices, which may arise as science develops. To ensure that recommendations given by the Agency take into account the views of equivalent advisory mechanisms in other legal frameworks, the Agency should consult the relevant advisory or regulatory bodies.

Added:Recital 12: (12) The structure and operation of the various bodies making up the Agency should be designed in such a way as to take into account the need to constantly renew scientific expertise, the need for cooperation between Union and national bodies, the need for adequate involvement of civil society, and the future enlargement of the Union. The various bodies of the Agency should establish and develop appropriate contacts with the parties concerned, in particular with representatives of patients, consumers and healthcare professionals.

Removed:Recital 36: (36) The expertise of the Committee for Advanced Therapies (CAT), the Committee for Orphan Medicinal Products (COMP), the Paediatric Committee (PDCO) and Committee for Herbal Medicinal Products (HMPC) is retained through working groups, ad-hoc working groups, working parties and a pool of experts who are organised based on different domains and who are giving input to the CHMP and PRAC. The CHMP and PRAC consists of experts from all Member States while working parties consist in majority of experts appointed by the Member States, based on their expertise, and of external experts. The model of rapporteurs remains unchanged. Representation of patients and health care professionals, with expertise in all areas, including rare and paediatric diseases, is increased at the CHMP and PRAC, in addition to the dedicated working groups representing patients and health care professionals.

Added:Recital 13: (13) The chief task of the Agency should be to provide Union institutions and Member States with the best possible scientific opinions to enable them to exercise the powers of authorisation and supervision of medicinal products conferred on them by Union legal acts in the field of medicinal products. Marketing authorisation should be granted by the Commission only after a single scientific evaluation procedure addressing the quality, safety, efficacy of high-technology medicinal products has been conducted by the Agency, applying the highest possible standards and the completion of an environmental risk assessment.

Removed:Recital 39: (39) To allow for a more informative decision making and for exchange of information and pooling of knowledge on general issues of scientific or technical nature related to the tasks of the Agency regarding medicinal products for human use, in particular to scientific guidelines on unmet medical needs and the design of clinical trials, or other studies and the generation of evidence along the life cycle of medicinal product, the Agency should be able to have recourse to a consultation process of authorities or bodies active along the life cycle of medicinal products. These authorities could be, as appropriate, representatives from Heads of Medicines Agencies, the Clinical Trial Coordination and Advisory Group, the SoHO Coordination Board, the Coordination Group on Health Technology Assessment, Medical Devices Coordination Group, medical devices national competent authorities, national competent authorities for pricing and reimbursement of medicines, national insurance funds or healthcare payers. The Agency should also be able to extend the consultation mechanism to consumers, patients, healthcare professionals, industry, associations representing payers, academia or other stakeholders, as relevant.

Added:Recital 15: (15) The Agency's budget should be transparent and composed of fees and charges paid by the private sector and contributions from the Union budget to implement Union policies and contributions paid from third countries. Although the majority of its funding comes from fees, the Agency is a public authority. It is of utmost importance to safeguard its integrity and independence in order to maintain public trust in the Union regulatory framework.

Removed:Recital 43: (43) In the interest of public health, marketing authorisation decisions under the centralised procedure should be taken on the basis of the objective scientific criteria of quality, safety and efficacy of the medicinal product concerned, to the exclusion of economic and other considerations. However, Member States should be able, exceptionally, to prohibit the use in their territory of medicinal products for human use. Member States should provide due justification for such prohibition to the Agency.

Added:Recital 18 a (new): (18a) The Agency should set transparent criteria for the appointment of patients’ and healthcare professionals’ representatives to the Committee for Medicinal Products for Human Use and the Pharmacovigilance Risk Assessment Committee in order to ensure there is a well-balanced representation of medical specialties and diseases amongst appointed members and alternates, and there are robust rules on the prevention of conflicts of interests. Declaration of direct or indirect financial or other interests in the pharmaceutical or other medical industry which could affect the impartiality of appointed stakeholders should be an integral part of the selection process and subsequently should be made publicly available.

Removed:Recital 60: (60) Regulatory decision-making on the development, authorisation and supervision of medicinal products may be supported by access and analysis of health data, including real world data, where appropriate, i.e. health data generated outside of clinical studies. The Agency should be able to use such data, including via the Data Analysis and Real World Interrogation Network (DARWIN) and the European Health Data Space interoperable infrastructure. Through these capabilities the Agency may take advantage of all the potential of supercomputing, artificial intelligence and big data science to fulfil its mandate, without compromising privacy rights. In this regard, the Agency should also engage in a systematic early and continuous dialogue on the collection of real-world-data with the Member State Coordination Group on Health Technology Assessment in accordance with Regulation (EU) 2021/2282, payers, healthcare professionals, patient and consumer organisations and developers. Where necessary the Agency may cooperate with the competent authorities of the Member States towards this objective.

Added:Recital 19: (19) Scientific advice for future applicants seeking a marketing authorisation should be provided more generally and in greater depth and should be adapted to the specificities of the medicinal product concerned. Similarly, structures allowing the development of advice for companies, in particular small and medium-sized enterprises (‘SMEs’) and not-for-profit entities, should be put in place. The Agency should also promote open and public exchanges about latest scientific developments and updates of scientific guidelines.

Removed:Recital 67: (67) The Agency, in consultation with the Member States and the Commission, should set the scientific selection criteria for medicinal products that receive pre-authorisation support with priority to be given to the most promising developments in therapies. In the case of medicinal products for unmet medical needs, based on the scientific selection criteria set by the Agency, any interested developer can submit preliminary evidence to demonstrate that the medicinal product has the potential to provide a major therapeutic advancement with respect to the identified unmet medical need. Information on the advice given in relation to the enhanced scientific and regulatory support for priority medicinal products should be included in a dedicated specific section within the European public assessment report.

Added:Recital 20: (20) Promising medicinal products and certain combinations products of medicinal products and medical devices, as well as medicinal products in exclusive use with medical devices that have the potential to significantly address patients’ unmet medical needs should benefit from early and enhanced scientific support, including through supporting patient-relevant in vitro and in silico technologies which are key to the development of those products. Such support will ultimately help patients benefit from new therapies as early as possible.

Removed:Recital 68: (68) Before a medicinal product for human use is authorised for placing on the market of one or more Member States, it generally has to undergo extensive studies to ensure that it is safe, of high quality and effective for use in the target population. However, in the case of certain categories of medicinal products for human use, in order to meet unmet medical needs of patients and in the interest of public health, it may be necessary to grant a conditional marketing authorisation on the basis of less complete data than is normally the case. Such marketing authorisation should be granted subject to specific obligations. The categories of medicinal products for human use concerned should be the medicinal products, including orphan medicinal products, that aim at the treatment, prevention or medical diagnosis of seriously debilitating or life-threatening diseases, or that are intended to be used in emergency situations in response to public health threats.

Added:Recital 20 a (new): (20a) Next to unmet medical needs already recognised in the pediatric, antimicrobial, oncological, rare, and neurodegenerative diseases, attention should also be given to unmet medical needs in the mental health sphere and treatments therein.

Removed:Recital 76: (76) It is considered appropriate to also have the possibility for the Commission to grant temporary emergency marketing authorisations to address public health emergencies. Temporary emergency marketing authorisations may be granted provided that, having regard to the circumstances of the public health emergency, the benefit of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent to the fact that additional comprehensive quality, non-clinical, clinical data may still be required. A temporary emergency marketing authorisation should be valid only during the public health emergency. The Commission should be given the possibility to vary, suspend or revoke such marketing authorisations in order to protect public health or when the marketing authorisation holder has not complied with the conditions and obligations set out in the temporary emergency marketing authorisation or when the marketing authorisation holder has been granted a standard or conditional marketing authorisation for the relevant indication.

Added:Recital 21 a (new): (21a) Based on the European Ombudsman’s decision in its strategic inquiry OI/7/2017/KR of 17 July 2019 on how the European Medicines Agency engages with medicine developers in the period leading up to applications for authorisations to market new medicines in the Union, the Agency should enhance the transparency of scientific advice. In addition, staff and experts from national competent authorities providing scientific advice should, to the extent possible, not be involved in a subsequent evaluation of a marketing authorisation application for the same products. However, in duly justified cases, such as where the indication of a medicinal product concerns a rare disease, that expert should be able to carry out a subsequent evaluation of the same product, provided that that is duly documented.

Removed:Recital 76 a (new): (76a) It is imperative to have in place robust transparency measures and standards regarding the Agency’s regulatory activities in relation to medicinal products that receive a temporary emergency marketing authorisation. Those measures should include the timely publication of all relevant information on approved medicinal products and medical devices and of clinical data, including clinical trial protocols. The public information regarding clinical trials and marketing authorisation decisions should be in accordance with Regulation (EU) 2022/123 of the European Parliament and of the Council1a. / 1a Regulation (EU) 2022/123 of the European Parliament and of the Council of 25 January 2022 on a reinforced role for the European Medicines Agency in crisis preparedness and management for medicinal products and medical devices (OJ L 20, 31.1.2022, p. 1).

Added:Recital 25: (25) In certain cases, shortcomings in Member States’ system of supervision and related enforcement activities could risk to substantially hinder the achievement of the objectives of this Regulation and those of revised Directive 2001/83/EC which could even lead to the emergence of risks to public health or to the environment. To address these challenges, harmonised inspection standards should be ensured through the establishment of a joint audit programme within the Agency. This joint audit programme will also further harmonise the interpretation of good manufacturing and distribution practices on the basis of Union legislative requirements. Moreover, it will support further mutual recognition of inspection outcomes between Member States and with strategic partners. Within the joint audit programme, the competent authorities are subject to regular audits conducted by other Member States to maintain an equivalent and harmonised quality system and to ensure an appropriate implementation of relevant good manufacturing and distribution practices into national laws and equivalence with other EEA inspectorates.

Removed:Recital 77: (77) The development of antimicrobial resistance is a growing concern and the pipeline of effective antimicrobials is obstructed due to a market failure whereby pharmaceutical companies neglect investment in research and development (R&D) concerning novel antibiotics due to profitability considerations; it is therefore necessary to consider new measures to promote the development of priority antimicrobials that are effective against antimicrobial resistance and to support undertakings, often SMEs, which choose to invest in this area.

Added:Recital 26 a (new): (26a) Pharmaceutical research plays a decisive role in the continuing improvement in public health and in ensuring the Union’s competitiveness. Medicinal products, in particular those that are the result of long, costly research will not continue to be developed in the Union unless they are covered by favourable rules that provide for sufficient protection to encourage such research. However, it is difficult to establish a direct link between these favourable rules and Union competitiveness. Such rules, while making Union markets more attractive, are agnostic to the medicines’ geographical origin and authorised medicines from third countries are equally eligible to receive Union incentives, just as Union-based innovative companies can equally benefit from incentives in third countries.

Removed:Recital 78 a (new): (78a) In addition to the growing threat of antimicrobial resistance, there are other market failures present in the pharmaceutical sector for which further action at Union level is required to meet the public health needs of Union citizens. In particular, there is a stark misalignment between the R&D priorities of the pharmaceutical industry and the public health needs of Union citizens. The market failures in the Union have, in certain instances, also resulted in excessive prices for medicinal products and have led to shortages of medicinal products. This Regulation should therefore address those market failures to better deliver on the objectives of affordability, accessibility and availability of medicinal products in the Union.

Added:Recital 29: (29) Legal entities that are not engaged in an economic activity such as universities, public bodies, research centres or not-for-profit organisations, represent an important source of research in unmet medical needs, of research in different subpopulations, repurposing and optimisation and of innovation and should also benefit from this support scheme. Whereas it should be possible to take account of the particular situation of these entities on an individual basis, such support can best be achieved by means of a dedicated support scheme, including administrative support and through the reduction, deferral and waiver of fees.

Removed:Recital 78 b (new): (78b) Member States, the Union, third countries, international organisations and agencies have identified the key areas of priority pharmaceutical research, including novel antibiotics and antivirals, improved diagnostics and treatments for emerging infectious diseases, affordable and efficacious medicinal products in the areas oncology, neurodegenerative diseases and other areas of unmet medical need for which the development of orphan medicinal products is needed. However, while Member States and the Union offer generous corporate R&D subsidies as well as R&D tax incentives to stimulate pharmaceutical research, there is little evidence to suggest, and no obligation to ensure, that the composition of R&D portfolios of pharmaceutical companies is determined by such direct or indirect subsidies. It is therefore increasingly evident that effective public health policy is undermined by shortcomings in transparency and accountability in public expenditure directed towards health.

Added:Recital 30: (30) The Agency should be empowered to give scientific recommendations on whether a product under development, which could potentially fall under the mandatory scope of the centralised procedure, meets the scientific criteria to be a medicinal product. Such an advisory mechanism would address, as early as possible, questions related to borderline cases with other areas such as in particular substances of human origin, cosmetics or medical devices, which may arise as science develops. To ensure that recommendations given by the Agency take into account the views of equivalent advisory mechanisms in other legal frameworks, the Agency should consult the relevant advisory or regulatory bodies. Where there is a doubt about whether the regulatory status of a particular product under development, which could potentially fall under the mandatory scope of the centralised procedure, meets the scientific criteria to be a medicinal product, the Agency and the relevant advisory bodies responsible for other regulatory frameworks, namely medical devices and substances of human origin should engage in consultations. In such cases, the compendium referred to in Regulation (EU) 2024 /... of the European Parliament and of the Council1a[SoHO Regulation] should be consulted, where relevant. If after consulting the compendium, there remains doubt about the regulatory status the relevant bodies should further consult to determine the regulatory status. The Commission should facilitate the cooperati…

Removed:Recital 78 c (new): (78c) Those market failures highlight the need for a paradigm shift in both the Member States’ and the Union’s approach to generating pharmaceutical R&D in key areas of priority and in public expenditure directed towards health. The establishment of a mission oriented R&D facility at Union level which acts in the public interest could alleviate those market failures. The European Medicines Facility (‘EMF’) should therefore be established as an independent agency under this Regulation. The EMF should be tasked with conducting research and developing novel antimicrobials as well as with regard to other areas of unmet medical need, to support the Union to overcome market failures.

Added:Recital 31: (31) To increase transparency of scientific assessments and all other activities, a user-friendly European medicines web-portal should be created and maintained by the Agency. The portal should provide information for all centrally authorised medicinal products, inter alia on safety, efficacy, environmental risk, patient populations, and where relevant information on antimicrobial resistance, shortages, and pending obligations for marketing authorisation holders. Sufficient budgetary resources should be allocated to the Agency to ensure its transparency obligations and commitments are appropriately implemented.

Removed:Recital 79: deleted

Added:Recital 31 a (new): (31a) The Union Register of medicinal products lists all medicinal products for human and veterinary use as well as orphan medicinal products that have received a marketing authorisation by the Commission through the centralised procedure. The information provided in the Union Register can be used to search for pertinent information on the medicinal product in question, including the active substance, the international non-proprietary name, the anatomical therapeutic chemical (ATC), the indications of the medicinal product, information on the authorisation and any post-authorisation requirements as well as applicable regulatory protection periods.

Removed:Recital 80: deleted

Added:Recital 33 a (new): (33a) To ensure the adequate expertise and evaluation of the environmental risk assessments of pharmaceutical substances, the Agency should establish a new ad hoc Environmental Risk Assessment working party. That working party should be involved where necessary depending on the application for a marketing authorisation. The working party should have the scientific knowledge necessary to characterise and assess the risks, and the mitigation measures for such risks, related to the manufacture, use and disposal of medicinal products. The working party should contribute towards the implementation of the One Health Approach and closing the gap between pharmaceutical and environmental assessment.

Removed:Recital 81: deleted

Added:Recital 35: (35) The Agency’s scientific committees should be supported, in relation to their evaluation duties, by working parties which should be open to experts from the scientific world and appointed for this purpose whilst retaining complete responsibility for the scientific opinions issued by them.

Removed:Recital 82: deleted