Sittings · Document

Draft report (COM(2023)0193 – C9-0144/2023 – 2023/0131(COD)) 2023-11-21

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

Committee on the Environment, Public Health and Food Safety

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AM_Com_LegReport

Amendment 202

Tiemo Wölken

Proposal for a regulation

Recital -1 (new)

Text proposed by the Commission

Amendment

(-1) Ensuring that Europeans receive the medicines they need, when they need them, regardless of where they live in the EU, is a central objective of the European Health Union. Boosting the competitiveness of the European pharmaceutical industry, whilst also ensuring better availability of medicines and more equal and timely access for patients, is a key deliverable of the proposed EU pharmaceutical reform.

Or. en

Amendment 203

Tilly Metz

Proposal for a regulation

Recital 1 a (new)

Text proposed by the Commission

Amendment

(1 a) This Regulation contributes 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. The loss of biodiversity, changes in land use, deforestation, urbanisation, agricultural expansion and intensification, poor air quality, wildlife trafficking and consumption patterns and transmissions of diseases between humans and animals are all factors that have contributed to the increasing disease burden. In addition, pollution from active pharmaceutical ingredients negatively affects the quality of waters and ecosystems, posing risks to public health globally.

Or. en

Amendment 204

Billy Kelleher, Catherine Amalric, Ondřej Knotek

Proposal for a regulation

Recital 2

Text proposed by the Commission

Amendment

(2) The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by creating a modern framework that makes innovative and established medicinal products available to patients and healthcare systems at affordable prices, while ensuring security of supply and addressing environmental concerns.

(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 medicines in the Union, along with a modern framework that makes innovative and established medicinal products available to patients and healthcare systems while ensuring security of supply and addressing environmental concerns.

Or. en

Amendment 205

István Ujhelyi

Proposal for a regulation

Recital 2

Text proposed by the Commission

Amendment

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

Or. en

Amendment 206

Susana Solís Pérez, Catherine Amalric

Proposal for a regulation

Recital 2

Text proposed by the Commission

Amendment

(2) The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by supporting a conducive environment for the research, development, and manufacturing of pharmaceuticals within the Union along with a modern framework that makes innovative and established medicinal products available to patients and healthcare systems at affordable prices, while ensuring security of supply and addressing environmental concerns.

Or. en

Amendment 207

Nathalie Colin-Oesterlé

Proposal for a regulation

Recital 2

Text proposed by the Commission

Amendment

(2) The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by creating 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, in particular by ensuring security of supply and addressing environmental concerns.

Or. fr

Amendment 208

Nathalie Colin-Oesterlé

Proposal for a regulation

Recital 2 a (new)

Text proposed by the Commission

Amendment

(2 a) Addressing the issue of shortages of medicinal products has been a long-standing priority for the Member States and European Parliament as illustrated by several reports from the European Parliament such as the European Parliament resolution of 17 September 2020 on the shortage of medicines – how to address an emerging problem1 a, as well as by discussions within the Council of the European Union. However, that issue has remained, to date, unaddressed.

_________________

1 a OJ C 385, 22.9.2021, p. 83.

Or. fr

Amendment 209

Nathalie Colin-Oesterlé

Proposal for a regulation

Recital 2 b (new)

Text proposed by the Commission

Amendment

(2 b) The Commission's communication published on 24 October 2023 to address critical shortages of medicines and strengthen security of supply in the EU envisages, among other things, the launch of a European voluntary solidarity mechanism for medicines allowing Member States to redistribute their available stock in the event of shortages1 aa.

_________________

1 aa Commission communication 'Addressing medicine shortages in the EU' (COM (2023) 672)

Or. fr

Amendment 210

Nathalie Colin-Oesterlé

Proposal for a regulation

Recital 2 c (new)

Text proposed by the Commission

Amendment

(2 c) The implementation of the 'Strategic Technologies for Europe Platform' (STEP) should contribute to reducing the Union's dependencies on third countries in the field of biotechnologies and turn into a fully-fledged sovereignty fund for the pharmaceutical industry in the future1 aa. The European pharmaceutical industry must be protected from international competition, and fiscal and financial incentives should be permitted in order to encourage manufacturers to relocate to Europe the production of active ingredients and medicines of strategic importance for healthcare.

_________________

1 aa Regulation of the European Parliament and of the Council establishing the Strategic Technologies for Europe Platform (‘STEP’) and amending Directive 2003/87/EC, Regulations (EU) 2021/1058, (EU) 2021/1056, (EU) 2021/1057, (EU) No 1303/2013, (EU) No 223/2014, (EU) 2021/1060, (EU) 2021/523, (EU) 2021/695, (EU) 2021/697 and (EU) 2021/241.

Or. fr

Amendment 211

Tilly Metz

Proposal for a regulation

Recital 3

Text proposed by the Commission

Amendment

(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 have called for revised mechanisms and incentives for development of medicinal products tailored to the level of unmet medical need, while ensuring patient access and availability of medicinal products in all Member States.

(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 have called for revised mechanisms and incentives for development of medicinal products tailored to the level of unmet medical need, while ensuring conditionalities related to transparency, affodability and availability are in place to ensure patient access of medicinal products in all Member States.

Or. en

Amendment 212

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Ondřej Knotek, Billy Kelleher, Erik Poulsen

Proposal for a regulation

Recital 3

Text proposed by the Commission

Amendment

(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 Parliament have called for revised mechanisms and incentives for development of medicinal products tailored to the level of unmet medical need, while ensuring patient access and availability of medicinal products in all Member States.

Or. en

Justification

European Parliament resolution of 24 November 2021 on a pharmaceutical strategy for Europe (2021/2013(INI))

https://www.europarl.europa.eu/doceo/document/TA-9-2021-0470_EN.html

Amendment 213

Tilly Metz

Proposal for a regulation

Recital 4

Text proposed by the Commission

Amendment

(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, these medicinal products do not always reach the patient and patients in the Union still have different levels of access to medicines.

(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 there still exist many unaddressed public health priorities, these medicinal products do not always reach the patient and patients in the Union still have different levels of access to medicines, for instance due to their high prices, inadequate infrastructures in Member States or the industry’s business strategy.

Or. en

Amendment 214

Maria Angela Danzì

Proposal for a regulation

Recital 4 a (new)

Text proposed by the Commission

Amendment

(4a) In the interests of public health and with a view to the proper functioning of the EU regulatory framework, an innovative medicinal product should be understood as a medicinal product that satisfies a substantial health need that was previously unsatisfactorily or inadequately met and offers greater efficacy or other incremental benefits than existing therapeutic alternatives.

Or. it

Justification

Un certo numero di termini e concetti richiedono definizioni comuni. Una di queste è la "medicina innovativa". "Innovazione" e "innovativo" sono termini ampiamente utilizzati ma raramente definiti esplicitamente. Ci sono prove che non tutti i medicinali "innovativi" apportano un reale valore aggiunto ai pazienti. Vi è una chiara necessità di definire meglio ciò che costituisce "innovazione" a beneficio dei pazienti e dei sistemi sanitari. Questa definizione è necessaria anche per ottimizzare il sistema di incentivazione e per migliorare l'accesso ai medicinali a prezzi accessibili.

Amendment 215

Tilly Metz

Proposal for a regulation

Recital 5

Text proposed by the Commission

Amendment

(5) The COVID-19 pandemic further underlined critical issues which require a reform of the Union pharmaceuticals framework to improve medicines availablity, to strengthen its resilience and to ensure that it corresponds to public health needs and serves the people under all circumstances.

Or. en

Amendment 216

Kateřina Konečná

Proposal for a regulation

Recital 5

Text proposed by the Commission

Amendment

(5) The COVID-19 pandemic has spotlighted critical issues which require a reform of the Union pharmaceuticals framework to strengthen its resilience and to ensure that it serves the people under all circumstances. In light of the disparities in terms of national immunization infrastructures and preparation highlighted by the COVID-19 pandemic, Member States should strengthen their national immunization programmes, ensuring a proper protection of their population against infectious diseases and building sustainable pandemic responses.

Or. en

Amendment 217

Andrey Slabakov

Proposal for a regulation

Recital 5

Text proposed by the Commission

Amendment

(5) The COVID-19 pandemic has spotlighted critical issues which require a reform of the Union pharmaceuticals framework to strengthen its resilience and to ensure that it serves the people under all circumstances. In light of the disparities in terms of national immunization infrastructures and preparation, Member States should evaluate their national immunization programmes, and determine whether they are fit to protect their population.

Or. en

Amendment 218

Susana Solís Pérez, Catherine Amalric

Proposal for a regulation

Recital 5 a (new)

Text proposed by the Commission

Amendment

(5 a) The pharmaceutical framework should be aligned with the EU's ambitions in industry, digitalization and trade, acknowledging the critical role of the European life sciences sector, especially the pharmaceutical industry, in upholding the EU's competitive edge. Bolstering robust European research and development is crucial for European sovereignty within the ambit of a globally competitive geopolitical landscape. The pharmaceutical legislative framework should be attuned to the broader EU industrial strategy, echoing the Council's emphasis from 23 March 2023 on amplifying incentives for investment in innovation and the 2016 Council's guidance that any amendments, including those affecting the incentive system, should not hinder the creation of drugs for rare disease treatment. Advancements in innovation are pivotal for enhancing patient health outcomes and the wider public health sector.

Or. en

Amendment 219

Tomislav Sokol

Proposal for a regulation

Recital 5 a (new)

Text proposed by the Commission

Amendment

(5 a) The pharmaceutical framework should be consistent with overarching EU industrial policy, including the Council Conclusions from 23 March 2023 which stress the importance of strengthening incentives for investment in innovation and the 2016 Council Conclusions which stress any revision, including to the incentive framework, should not discourage the development of medicinal products needed for the treatment of rare diseases; increased innovation will further support patient outcomes and public health.

Or. en

Amendment 220

Tilly Metz

Proposal for a regulation

Recital 6

Text proposed by the Commission

Amendment

(6) For the sake of clarity, it is necessary to replace Regulation (EC) No 726/2004 of the European Parliament and of the Council38 with a new Regulation.

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

_________________

38 Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency (OJ L 136, 30.4.2004, p. 1).

Or. en

Amendment 221

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 9

Text proposed by the Commission

Amendment

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

(9) As to the scope of this Regulation, the authorisation of antimicrobials is in the sole interest of safeguarding patients' health at Union level and managing public health concerns related to antimicrobial resistance, and therefore it should be made possible to authorise them at Union level.

Or. en

Amendment 222

Tilly Metz

Proposal for a regulation

Recital 12

Text proposed by the Commission

Amendment

(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 and healthcare professionals.

Or. en

Amendment 223

Maria Angela Danzì

Proposal for a regulation

Recital 12 a (new)

Text proposed by the Commission

Amendment

(12a) To prevent influence on the development or implementation of pharmaceutical policies or legislation or the decision-making processes of the Agency and the other EU institutions, the Executive Director, the Deputy Executive Director, the members of the Management Board and the members of the Committees may not, for two years after leaving office, hold any office or accept posts or engage in any activity whether remunerated or free of charge, on behalf of any person over whom they have exercised regulatory, negotiating or authorising powers.

Or. it

Amendment 224

Tilly Metz

Proposal for a regulation

Recital 13

Text proposed by the Commission

Amendment

(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 and efficacy of high-technology medicinal products has been conducted by the Agency, applying the highest possible standards.

(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 and environmetal risk of medicinal products has been conducted by the Agency, applying the highest possible standards.

Or. en

Amendment 225

Tilly Metz

Proposal for a regulation

Recital 15

Text proposed by the Commission

Amendment

(15) The Agency's budget should be 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.

(15) The Agency's budget should be transparent 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 and it is of utmost importance to safeguard its integrity and independence in order to ensure public trust in the Union regulatory framework.

Or. en

Justification

agreed text on EMA Fees Regulation

Amendment 226

Tilly Metz

Proposal for a regulation

Recital 18 a (new)

Text proposed by the Commission

Amendment

(18 a) 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 a well-balanced representation of medical specialties and diseases amongst appointed members and alternates, as well as to ensure 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.

Or. en

Amendment 227

Andrey Slabakov

Proposal for a regulation

Recital 19

Text proposed by the Commission

Amendment

(19) Scientific advice for future applicants seeking a marketing authorisation should be provided more generally and in greater depth. Similarly, structures allowing the development of advice for companies, in particular small and medium-sized enterprises (‘SMEs’), should be put in place. It is important to ensure that this robust, efficient and transparent advice mechanism also applies to health technologies, in order to support timely and effective evaluation of all health products and technologies, including particularly promising medical devices that have the potential to significantly address patients’ unmet medical needs.

Or. en

Amendment 228

Tilly Metz

Proposal for a regulation

Recital 19

Text proposed by the Commission

Amendment

Or. en

Amendment 229

Tiemo Wölken

Proposal for a regulation

Recital 20

Text proposed by the Commission

Amendment

(20) Promising medicinal products that have the potential to significantly address patients’ unmet medical needs should benefit from early and enhanced scientific support. Such support will ultimately help patients benefit from new therapies as early as possible.

(20) Promising medicinal products that have the potential to significantly address patients’ unmet medical needs should benefit from early and enhanced scientific support, including through supporting innovative patient-relevant in vitro and in silico technologies which are key to the development of these products. Such support will ultimately help patients benefit from new therapies as early as possible.

Or. en

Amendment 230

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Billy Kelleher, Erik Poulsen

Proposal for a regulation

Recital 20

Text proposed by the Commission

Amendment

Or. en

Amendment 231

Andrey Slabakov

Proposal for a regulation

Recital 20

Text proposed by the Commission

Amendment

(20) Promising medicinal products and certain medical devices that have the potential to significantly address patients’ unmet medical needs should benefit from early and enhanced scientific support. Such support will ultimately help patients benefit from new therapies as early as possible.

Or. en

Amendment 232

Stelios Kympouropoulos, Peter Liese

Proposal for a regulation

Recital 20

Text proposed by the Commission

Amendment

(20) Promising medicinal products and medical devices that have the potential to significantly address patients’ unmet medical needs should benefit from early and enhanced scientific support. Such support will ultimately help patients benefit from new therapies as early as possible.

Or. en

Amendment 233

Tilly Metz

Proposal for a regulation

Recital 20 a (new)

Text proposed by the Commission

Amendment

(20 a) Despite the evolution of science and technology at a rapid pace and the regulatory incentives currently in place, there are many diseases without or only with suboptimal treatments. This Regulation should contribute to the development of treatments to tackle these unmet medical needs. These are in particular life threatening or severely debilitating diseases where no treatment alternative exists or where the disease still causes high morbidity or mortality, despite available therapeutic options. Products receiving regulatory incentives should result in a meaningful reduction in disease morbidity or mortality for the relevant patient population and a significant increase in patient’s quality of life.

Or. en

Amendment 234

Cyrus Engerer

Proposal for a regulation

Recital 20 a (new)

Text proposed by the Commission

Amendment

(20 a) Given that poor physical and mental health has a profound impact on societies and communities, high unmet medical needs shall be evaluated also within the broader context of public health and societal challenges.

Or. en

Amendment 235

Cyrus Engerer

Proposal for a regulation

Recital 20 b (new)

Text proposed by the Commission

Amendment

(20 b) Next to unmet medical needs already recognized in the antimicrobial, oncological, rare, and neurodegenerative diseases, particular attention should be given to unmet medical needs related to mental health sector and thus supporting novel mental health treatments which are showing promising potential.

Or. en

Amendment 236

Cyrus Engerer

Proposal for a regulation

Recital 20 c (new)

Text proposed by the Commission

Amendment

(20 c) The revision shall aim to support innovative mental health care treatments that combine pharmacological, and psychological aspects. Moreover, generation of empirical evidence for the quality and safety of novel mental health treatments should be further explored and supported. This includes understanding the interactions between the pharmacological substances and the psychotherapy as well as assistive technologies that can assist to patient preparation, monitoring or treatment integration.

Or. en

Amendment 237

Tilly Metz

Proposal for a regulation

Recital 21 a (new)

Text proposed by the Commission

Amendment

(21 a) Based on the Ombudsman’s decision in the strategic inquiry 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 EU1a, the Agency should enhance transparency of scientific advice. In addition, staff and experts from national competent authorities providing scientific advice should not be involved in a subsequent evaluation of marketing authorisation application for the same products.

_________________

1a European Ombudsman’s Decision in strategic inquiry OI/7/2017/KR on how the European Medicines Agency engages with medicine developers in the period leading up to applications for authorizations to market new medicines in the EU, July 2019.

Or. en

Amendment 238

Sirpa Pietikäinen

Proposal for a regulation

Recital 25

Text proposed by the Commission

Amendment

(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. 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.

(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 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.

Or. en

Amendment 239

Tiemo Wölken

Proposal for a regulation

Recital 26 a (new)

Text proposed by the Commission

Amendment

(26 a) Pharmaceutical research plays a decisive role in the continuing improvement in public health. 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 hard to establish a direct link between between EU incentives and EU competitiveness because while the incentives make the EU markets more attractive, they are agnostic to the medicines’ geographical origin and authorised medicines from third countries are equally eligible to all EU incentives, as EU based innovative companies can equally benefit from incentives elsewhere.

Or. en

Amendment 240

Tilly Metz

Proposal for a regulation

Recital 29

Text proposed by the Commission

Amendment

(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 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.

(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, research in different subpopulations, repurposing, optimisation and 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.

Or. en

Amendment 241

Stanislav Polčák

Proposal for a regulation

Recital 29

Text proposed by the Commission

Amendment

(29) Legal entities that are not engaged in an economic activity as their main activity such as universities, public bodies, research centres or not-for-profit organisations, represent an important source 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.

Or. cs

Amendment 242

Tiemo Wölken

Proposal for a regulation

Recital 30

Text proposed by the Commission

Amendment

(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 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 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) No [reference to be added after adoption cf. COM(2022)338 final] should be consulted. 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 cooperation between the Agency and advisory bodies established by other Union legislation. Where an agreement by consensus cannot be reached by the Agency and the relevant advisory bodies, the Commission should be empowered to take a decision on the regulatory status of the product in question. The opinions and inclusions of the Agency and the relevant advisory bodies on the regulatory status of the product should be made publicly available after the consultations have taken place.

Or. en

Amendment 243

Susana Solís Pérez, Ondřej Knotek

Proposal for a regulation

Recital 30

Text proposed by the Commission

Amendment

Or. en

Amendment 244

Ondřej Knotek, Frédérique Ries, Billy Kelleher

Proposal for a regulation

Recital 30

Text proposed by the Commission

Amendment

Or. en

Amendment 245

Stelios Kympouropoulos

Proposal for a regulation

Recital 30

Text proposed by the Commission

Amendment

Or. en

Amendment 246

Tilly Metz

Proposal for a regulation

Recital 30

Text proposed by the Commission

Amendment

(30) The Agency should 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 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 opinions differ between the Agency and other advisory bodies, the diverging opinions and their reasoning shall be included in the recommendations of the Agency.

Or. en

Amendment 247

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 30

Text proposed by the Commission

Amendment

Or. en

Amendment 248

Susana Solís Pérez, Catherine Amalric

Proposal for a regulation

Recital 30 a (new)

Text proposed by the Commission

Amendment

(30 a) For informed policy development, the Agency should maintain its authority to carry out pilot programs, fostering a regulatory environment that is adaptive to future challenges. Efforts like the 2022 pilot program that provided augmented assistance to academic and non-profit developers of advanced therapy medicinal products should inform policy decisions and refine regulatory guidance.

Or. en

Amendment 249

Tomislav Sokol

Proposal for a regulation

Recital 30 a (new)

Text proposed by the Commission

Amendment

(30 a) To support evidence-based policy making, the Agency should continue to be empowered to undertake pilot programmes to ensure a future-proof regulatory ecosystem. Initiatives such as the pilot programme launched in 2022 offering enhanced support to academic and non-profit developers of advanced therapy medicinal products should guide policy-making and update regulatory support.

Or. en

Amendment 250

Tilly Metz

Proposal for a regulation

Recital 31

Text proposed by the Commission

Amendment

(31) To increase transparency of scientific assessments and all other activities, a European medicines web-portal should be created and maintained by the Agency.

(31) The Agency has an important role in sharing of information with the general public in order to ensure trust in its work and to support health literacy of patient and consumers. In addition, it is key to share up to date information with healthcare professionals, including pharmacists, and the scientific community. 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 serve an information hub for all centrally authorised medicinal products and include information about safety, efficacy, environmental risk, patient populations, antimicrobial resistance, shortages, pending obligations for marketing authorisation holders and status on intellectual property, regulatory and market protection periods. Sufficient budget should be allocated to the Agency to ensure appropriate implementation of its transparency obligations and commitments.

Or. en

Amendment 251

Sunčana Glavak

Proposal for a regulation

Recital 33

Text proposed by the Commission

Amendment

(33) To optimise the functioning and efficiency of the regulatory system, the structure of the Agency’s scientific committees is simplified and reduced to two main Committees for medicinal products for human use, the Committee for Medicinal Products for Human Use (CHMP) and Pharmacovigilance Risk Assessment Committee (PRAC).

Or. en

Amendment 252

Maria Angela Danzì

Proposal for a regulation

Recital 34

Text proposed by the Commission

Amendment

(34) The simplification of procedures should not have an impact on standards or the quality of scientific evaluation of the medicinal products to guarantee the quality, safety and efficacy of medicinal products. It should also allow for the reduction of the scientific evaluation period from 210 days to 180 days.

Or. it

Amendment 253

Tiemo Wölken

Proposal for a regulation

Recital 34

Text proposed by the Commission

Amendment

Or. en

Amendment 254

Tomislav Sokol

Proposal for a regulation

Recital 35

Text proposed by the Commission

Amendment

(35) The Agency’s scientific committees should be able to delegate some of their evaluation duties to 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.

(35) The Agency’s scientific committees should be supported, for their evaluation duties by working parties which should be open to experts from the scientific world and appointed for this purpose and by additional experts drawn from the pool of accredited experts, whilst retaining complete responsibility for the scientific opinions issued by them.

Or. en

Amendment 255

Frédérique Ries

Proposal for a regulation

Recital 35

Text proposed by the Commission

Amendment

(35) The Agency’s scientific committees should be supported for their evaluation duties by working parties which should be open to experts from the scientific world and appointed for this purpose and by additional experts drawn from the pool of accredited experts, whilst retaining complete responsibility for the scientific opinions issued by them.

Or. en

Amendment 256

Tilly Metz

Proposal for a regulation

Recital 35

Text proposed by the Commission

Amendment

(35) The Agency’s scientific committees should be supported for their evaluation duties by working parties which should be open to experts from the scientific world and appointed for this purpose, and by additional experts drawn from the pool of accredited experts, whilst retaining complete responsibility for the scientific opinions issued by them.

Or. en

Justification

Recitals 35 and 36 were amended by the EMA to clarify that restructuring of the agency would not lead to lowering of scientific expertise and standards

Amendment 257

Tomislav Sokol

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

(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, working parties and a pool of experts who are organised based on different domains and who are giving input to the CHMP and PRAC. Their evaluation will continue to encompass all the necessary expertise for each product as part of the rapporteur teams, with the possibility for CHMP and PRAC to call upon additional scientific experts to provide specific input and advice on specific aspects raised during the evaluation. In addition patients and healthcare professionals will be part of the pool of experts and will also be brought into EMA’s work according to their expertise in a certain disease area. The CHMP and PRAC consists of experts from all Member States while working parties and expert groups 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.

Or. en

Amendment 258

Tilly Metz

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

(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, working parties and a pool of experts who are organised based on different domains and who are giving input to the CHMP and PRAC. Their evaluation will continue to encompass all the necessary expertise for each product as part of the rapporteur teams, with the possibility for CHMP and PRAC to call upon additional scientific experts to provide specific input and advice on specific aspects raised during the evaluation. In addition, patients and healthcare professionals will be part of the pool of experts and will also be brought into EMA’s work according to their expertise in a certain disease area. The CHMP and PRAC consists of experts from all Member States while working parties and expert groups 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.

Or. en

Justification

Recitals 35 and 36 were amended by the EMA to clarify that restructuring of the agency would not lead to lowering of scientific expertise and standards

Amendment 259

Frédérique Ries

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

(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, working parties and a pool of experts who are organised based on different domains and who are giving input to the CHMP and PRAC. Their evaluation will continue to encompass all the necessary expertise for each product as part of the rapporteur teams, with the possibility for CHMP and PRAC to call upon additional scientific experts to provide specific input and advice on specific aspects raised during the evaluation. In addition patients and healthcare professionals will be part of the pool of experts and will also be brought into EMA’s work according to their expertise in a certain disease area. 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.

Or. en

Amendment 260

Maria Angela Danzì

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

(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, 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. What is more, with regard to the scientific assessment and the agreement of the paediatric investigation plans and other issues of paediatric interest not falling under the responsibilities of the PRAC and the CHMP, the expertise and experience of the Paediatric Committee will be retained in a paediatric working group that will provide a contribution to the Agency on paediatric issues. 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.

Or. it

Justification

The main objective of this amendment is to prevent the vast experience gained at PDCO level from being lost. This experience cannot be used within the CHMP, as the new regulation will assign responsibility for evaluating, amending, assessing the needs and value of paediatric investigation plans to the Agency, not to the CHMP.

Amendment 261

Susana Solís Pérez, Catherine Amalric, Ondřej Knotek

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

(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, 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. The composition, responsibilities, operating methods, and areas of expertise of the Committees, Working Parties, and Working Groups, along with their work programs and suggestions, will be disclosed to the public and may be opened for stakeholder feedback.

Or. en

Amendment 262

Pernille Weiss

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

(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, 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. Information regarding the composition and work of the committees and working groups should be publically available.

Or. en

Amendment 263

Ondřej Knotek, Frédérique Ries, Catherine Amalric, Billy Kelleher

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

Or. en

Amendment 264

Stelios Kympouropoulos, Pernille Weiss

Proposal for a regulation

Recital 36

Text proposed by the Commission

Amendment

Or. en

Amendment 265

Ondřej Knotek, Billy Kelleher

Proposal for a regulation

Recital 37

Text proposed by the Commission

Amendment

(37) Scientific committees like the CAT have been instrumental to ensure expertise and capacity building in an emerging technological field. However, after more than 15 years, advanced therapy medicinal products are now more common. The full integration of their assessment in the work of the CHMP will facilitate the assessment of medicinal products within the same therapeutic class, independent of the technology on which they are based. It will also ensure that all biological medicinal products are assessed by the same committee.

Or. en

Amendment 266

Tilly Metz

Proposal for a regulation

Recital 37 a (new)

Text proposed by the Commission

Amendment

(37 a) To ensure the adequate expertise and evaluation of the environmental risk assessments of pharmaceutical substances, the Agency establishes a new Environmental Risk Assessment working party which will have the scientific knowledge to characterise and assess the risks and hazards related to the development, production, use and disposal of medicines and their impact on the environment, animals, humans and public health. The working party will ensure the One Health Approach in medicines evaluation and closing the gap between pharmaceutical and environmental assessment. The working party ensures, based on cross-sectorial scientific knowledge, that substance is adequately assessed and the risk minimisation and containment strategies proposed by the applicants are sufficient in order to place the product on the market.

Or. en

Amendment 267

Tomislav Sokol

Proposal for a regulation

Recital 38 a (new)

Text proposed by the Commission

Amendment

(38 a) For medicinal products that are likely to offer an exceptional therapeutic advancement in the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the Union, with a view to enabling earlier availability for patients, the Agency should be able to perform a ‘phased review’ of data packages concerning finalised tests and trials before a formal application for marketing authorisation is submitted, to allow a more efficient assessment of medicinal products, while guaranteeing a high level of human health protection.

Or. en

Amendment 268

Tiemo Wölken

Proposal for a regulation

Recital 39

Text proposed by the Commission

Amendment

(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, or other stakeholders, as relevant.

(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. Additionally, to improve regulatory certainty and cross-sectoral cooperation the Commission should on an annual basis, or more frequently where deemed necessary, organise joint meetings with the advisory bodies established in other Union legislation to assess emerging trends and questions on regulatory status of products and find agreement on common regulatory status principles. 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, or other stakeholders, as relevant.

Or. en

Amendment 269

Susana Solís Pérez, Catherine Amalric, Ondřej Knotek

Proposal for a regulation

Recital 39

Text proposed by the Commission

Amendment

(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 and their caregivers, healthcare professionals, industry, associations representing payers, academia, or other stakeholders, as relevant.

Or. en

Amendment 270

Ondřej Knotek, Frédérique Ries, Catherine Amalric, Billy Kelleher

Proposal for a regulation

Recital 39

Text proposed by the Commission

Amendment

(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 and their caregivers, healthcare professionals, academia, industry, associations representing payers, or other stakeholders, as relevant.

Or. en

Amendment 271

Stelios Kympouropoulos, Pernille Weiss

Proposal for a regulation

Recital 39

Text proposed by the Commission

Amendment

(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, their caregivers, healthcare professionals, acdemia, industry, associations representing payers, or other stakeholders, as relevant.

Or. en

Amendment 272

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Ondřej Knotek, Billy Kelleher, Erik Poulsen

Proposal for a regulation

Recital 39

Text proposed by the Commission

Amendment

(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 and their caregivers, healthcare professionals, industry, associations representing payers, or other stakeholders, as relevant.

Or. en

Amendment 273

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 39

Text proposed by the Commission

Amendment

(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 extend the consultation mechanism to consumers, patients, healthcare professionals, industry, associations representing payers, academia or other stakeholders from all Member States, as relevant.

Or. en

Amendment 274

Andrey Slabakov

Proposal for a regulation

Recital 40

Text proposed by the Commission

Amendment

(40) Member States should ensure adequate funding of competent authorities to carry out their tasks under this Regulation and under [revised Directive 2001/83/EC]. In addition, in line with the Joint Statement of the European Parliament, the Council of the EU and the European Commission on decentralised agencies48 , Member States should ensure adequate resources are assigned by the competent authorities of the Member States for the purpose of their contributions to the work of the Agency, taking into account the cost-based remuneration they receive from the Agency.

_________________

48 https://europa.eu/european-union/sites/europaeu/files/docs/body/joint_statement_and_common_approach_2012_en.pdf

Or. en

Amendment 275

Tilly Metz

Proposal for a regulation

Recital 41 a (new)

Text proposed by the Commission

Amendment

(41 a) Protecting global health is one of the priorities of the EU and under Article 178 of the Treaty, the Union should take into account the development policy aspects in any measure and promote the creation of conditions fit for human beings worldwide. To this end, this Regulation should particularly allow for the development of efficacious, safe, accessible, and affordable innovations to address global public health needs, including antimicrobial resistance, poverty-related and neglected diseases, widespread tropical disease and ensure high quality standards for medicinal products that are exported.

Or. en

Amendment 276

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 43

Text proposed by the Commission

Amendment

(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.

(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, demand, trade, geopolitical, expediency or commercial interest or any other considerations. However, Member States should be able, exceptionally, to prohibit the use in their territory of medicinal products for human use, biological products, food supplements and nutraceuticals, dietary products, biotechnology derived products.

Or. en

Amendment 277

Tiemo Wölken

Proposal for a regulation

Recital 43 a (new)

Text proposed by the Commission

Amendment

(43 a) The Union is required, pursuant to Article 208 of the Treaty on the Functioning of the European Union, to take account of development objectives in policies that are likely to have an impact on low- and middle-income countries. Pharmaceutical law has a role to play in the realisation of global public health objectives by promoting the development of efficacious, safe, accessible, and affordable innovations for antimicrobial resistance, poverty-related and neglected diseases, and other conditions of global public health interest and it should continue to ensure the highest standards in exported health products and contribute to stronger and more harmonised international technical requirements applicable to medicinal products. The Commission should consider to explore novel mechanisms to encourage research, development and innovation in areas of major global health interest, such as poverty-related, emerging and re-emerging, and neglected infectious diseases; chronic diseases; or comorbidities, in line with its international commitments and within the limits of its competences.

Or. en

Amendment 278

Tiemo Wölken

Proposal for a regulation

Recital 43 b (new)

Text proposed by the Commission

Amendment

(43 b) The Commission is empowered to explore novel mechanisms to encourage research, development and innovation in areas of major global health interest, such as poverty-related, emerging and re-emerging, and neglected infectious diseases; chronic diseases; or comorbidities, in line with its international commitments and within the limits of its competences.

Or. en

Amendment 279

Tilly Metz

Proposal for a regulation

Recital 44

Text proposed by the Commission

Amendment

(44) The quality, safety and efficacy criteria of [revised Directive 2001/83/EC] should apply to medicinal products authorised by the Union under the centralised procedure. The benefit-risk balance of all medicinal products will be assessed when they are placed on the market, and at any other time the competent authority deems appropriate.

(44) The quality, safety, efficacy and environmental risk criteria of [revised Directive 2001/83/EC] should apply to medicinal products authorised by the Union under the centralised procedure. The benefit-risk balance of all medicinal products will be assessed when they are placed on the market, five years after approval and at any other time the competent authority deems appropriate.

Or. en

Amendment 280

Stanislav Polčák

Proposal for a regulation

Recital 45

Text proposed by the Commission

Amendment

(45) Marketing authorisation applications, like any other application submitted to the Agency, should follow the digital by default principle and hence be sent to the Agency in electronic form. Applications should be assessed based on the file submitted by the applicant in accordance with the different legal basis provided by [revised Directive 2001/83/EC]. At the same time, the Agency and the relevant committees may take into account any information that is in its possession. Applicants shall be requested to generally submit raw data, in particular with regard to the clinical trials performed by the applicant in order to ensure a full assessment of the quality, safety and efficacy of the medicinal product.

(45) To avoid unnecessary administrative and financial burdens for applicants, marketing authorisation holders and competent authorities, certain streamlining measures should be introduced. Marketing authorisation applications, like any other application submitted to the Agency, including applications for a variation to the terms of marketing authorisations, should follow the digital by default principle and hence be sent to the Agency in electronic form. Applications should be assessed based on the file submitted by the applicant in accordance with the different legal basis provided by [revised Directive 2001/83/EC]. At the same time, the Agency and the relevant committees may take into account any information that is in its possession. Applicants shall be requested to generally submit raw data, in particular with regard to the clinical trials performed by the applicant in order to ensure a full assessment of the quality, safety and efficacy of the medicinal product.

Or. cs

Amendment 281

Tilly Metz

Proposal for a regulation

Recital 45

Text proposed by the Commission

Amendment

Or. en

Amendment 282

Tilly Metz

Proposal for a regulation

Recital 45 a (new)

Text proposed by the Commission

Amendment

(45 a) Particular attention should be given to the gender balance of clinical trials so that women can fully and safely benefit from medicines throughout their life-course.

Or. en

Amendment 283

Tilly Metz

Proposal for a regulation

Recital 46

Text proposed by the Commission

Amendment

(46) Directive 2010/63/EU of the European Parliament and of the Council on the protection of animals used for scientific purposes49 lays down provisions on the protection of animals used for scientific purposes based on the principles of replacement, reduction and refinement. Any study involving the use of live animals, which provides essential information on the quality, safety and efficacy of a medicinal product, should take into account those principles of replacement, reduction and refinement, where they concern the care and use of live animals for scientific purposes, and should be optimised in order to provide the most satisfactory results whilst using the minimum number of animals. The procedures of such testing should be designed to avoid causing pain, suffering, distress or lasting harm to animals and should follow the available Agency and the International Committee for Harmonisation (ICH) guidelines. In particular, the marketing authorisation applicant and the marketing authorisation holder should take into account the principles laid down in Directive 2010/63/EU, including, where possible, use of new approach methodologies in place of animal testing. These can include but are not limited to: in vitro models, such as microphysiological systems including organ-on-chips, (2D and 3D) cell culture models, organoids and human stem cells-based models; in silico tools or read-across models.

(46) Directive 2010/63/EU of the European Parliament and of the Council on the protection of animals used for scientific purposes lays down provisions on the protection of animals used for scientific purposes based on the principles of replacement, reduction and refinement. Ultimately, efforts should be made to fully replace procedures on live animals for scientific purposes. Any study involving the use of live animals, which provides essential information on the quality, safety and efficacy of a medicinal product, should take into account those principles of replacement, reduction and refinement, where they concern the care and use of live animals for scientific purposes, and should be optimised in order to provide the most satisfactory results whilst using the minimum number of animals. The procedures of such testing should be designed to avoid causing pain, suffering, distress or lasting harm to animals and should follow the available Agency and the International Committee for Harmonisation (ICH) guidelines. In particular, the marketing authorisation applicant and the marketing authorisation holder should take into account the principles laid down in Directive 2010/63/EU, including, where possible, use of new approach methodologies (NAMs) in place of animal testing. These can include but are not limited to: in vitro models, such as microphysiological systems including organ-on-chips, (2D and 3D) cell culture models, organoids and human stem cells-based models; in silico tools grouping or read-across, aquatic egg models as well as invertebrate species.

_________________

49 Directive 2010/63/EU of the European Parliament and of the Council of 22 September 2010 on the protection of animals used for scientific purposes (OJ L 276, 20.10.2010, p. 33).

Or. en

Amendment 284

Tiemo Wölken

Proposal for a regulation

Recital 46

Text proposed by the Commission

Amendment

(46) Directive 2010/63/EU of the European Parliament and of the Council on the protection of animals used for scientific purposes49 lays down provisions on the protection of animals used for scientific purposes based on the principles of replacement, reduction and refinement. Any study involving the use of live animals, which provides essential information on the quality, safety and efficacy of a medicinal product, should take into account those principles of replacement, reduction and refinement, where they concern the care and use of live animals for scientific purposes, and should be optimised in order to provide the most satisfactory results whilst using the minimum number of animals. The procedures of such testing should be undertaken as a last resort and be designed to avoid causing pain, suffering, distress or lasting harm to animals and should follow the available Agency and the International Committee for Harmonisation (ICH) guidelines. In particular, the marketing authorisation applicant and the marketing authorisation holder should take into account the principles laid down in Directive 2010/63/EU, including, where possible, use of new approach methodologies in place of animal testing. These can include but are not limited to: in vitro models, such as microphysiological systems including organ-on-chips, (2D and 3D) cell culture models, organoids and human stem cells-based models; in silico tools or read-across models.

_________________

49 Directive 2010/63/EU of the European Parliament and of the Council of 22 September 2010 on the protection of animals used for scientific purposes (OJ L 276, 20.10.2010, p. 33).

Or. en

Amendment 285

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Billy Kelleher, Erik Poulsen

Proposal for a regulation

Recital 46

Text proposed by the Commission

Amendment

(46) Directive 2010/63/EU of the European Parliament and of the Council on the protection of animals used for scientific purposes49 lays down provisions on the protection of animals used for scientific purposes based on the principles of replacement, reduction and refinement. Any study involving the use of live animals, which provides essential information on the quality, safety and efficacy of a medicinal product, should take into account those principles of replacement, reduction and refinement, where they concern the care and use of live animals for scientific purposes, and should be optimised in order to provide the most satisfactory results whilst using the minimum number of animals. The procedures of such testing should be designed to avoid causing pain, suffering, distress or lasting harm to animals and should follow the available Agency and the International Committee for Harmonisation (ICH) guidelines. In particular, the marketing authorisation applicant and the marketing authorisation holder should take into account the principles laid down in Directive 2010/63/EU, giving priority to new approach methodologies in place of animal testing. These can include but are not limited to: in vitro models, such as microphysiological systems including organ-on-chips, (2D and 3D) cell culture models, organoids and human stem cells-based models; in silico tools or read-across models.

_________________

49 Directive 2010/63/EU of the European Parliament and of the Council of 22 September 2010 on the protection of animals used for scientific purposes (OJ L 276, 20.10.2010, p. 33).

Or. en

Amendment 286

Tiemo Wölken

Proposal for a regulation

Recital 47

Text proposed by the Commission

Amendment

(47) Procedures should be in place to facilitate joint animal testing, wherever possible, in order to avoid unnecessary duplication of testing using live animals covered by Directive 2010/63/EU. Marketing authorisation applicants and marketing authorisation holders should make all efforts to reuse animal study results and make the results obtained from animal studies publicly available. For abridged applications marketing authorisation applicants should refer to the relevant studies conducted for the reference medicinal product.

(47) Procedures should be in place to facilitate joint animal testing, wherever possible, in order to avoid unnecessary testing using live animals covered by Directive 2010/63/EU. Marketing authorisation applicants and marketing authorisation holders should make all efforts to reuse animal study results and make the results obtained from animal studies publicly available. For abridged applications marketing authorisation applicants should refer to the relevant studies conducted for the reference medicinal product.

Or. en

Amendment 287

Andrey Slabakov

Proposal for a regulation

Recital 48

Text proposed by the Commission

Amendment

(48) The summary of product characteristics and the package leaflet should reflect the assessment of the Agency and be part of its scientific opinion. The opinion may recommend certain conditions that should be part of the marketing authorisation, for example on the safe and efficacious use of the medicinal product or on post-authorisation obligations that have to be complied with by the marketing authorisation holder. Those conditions may include the requirement to conduct post-authorisation safety or efficacy studies or other studies that are considered necessary to optimise the treatment, for example where the proposed dose scheme by the applicant, whilst acceptable and justifying a positive benefit-risk balance, could be further optimised post-authorisation. Where the applicant disagrees with parts of the opinion, the applicant may request its re-examination.

Or. en

Amendment 288

Tilly Metz

Proposal for a regulation

Recital 49

Text proposed by the Commission

Amendment

(49) Due to the need to reduce overall approval times for medicinal products, the time between the opinion of the Committee for Medicinal Products for Human Use (CHMP) and the final decision on the application for a marketing authorisation should in principle be no longer than 46 days.

deleted

Or. en

Justification

Does not correspond to any article and might be a bit rigid

Amendment 289

Frédérique Ries

Proposal for a regulation

Recital 51

Text proposed by the Commission

Amendment

(51) As a general rule a marketing authorisation should be granted for an unlimited time; however, one renewal may be decided only on justified grounds related to the safety of the medicinal product.

(51) Given that the marketing authorisation holder has to forthwith submit any new data that might impact the benefit-risk balance of its products and given that the Agency has several tools available to continuously monitor the benefits and risks of authorised medicines, such as assessment of Periodic Safety Update Reports, signal detection and referrals, regulatory action will be taken as needed throughout the lifecycle of the product. Therefore, as a general rule a marketing authorisation should be granted for an unlimited time; however, one renewal may be decided only on justified grounds related to the safety of the medicinal product.

Or. en

Amendment 290

Tomislav Sokol

Proposal for a regulation

Recital 51

Text proposed by the Commission

Amendment

(51) As a general rule a marketing authorisation should be granted for an unlimited time; however, one renewal may be decided only on justified grounds related to the safety of the medicinal product.

(51) Given that the marketing authorisation holder has to forthwith submit any new data that might impact the benefit-risk balance of its products and given that the Agency has several tools available to continuously monitor the benefits and risks of authorised medicines, such as assessment of PSURs, signal detection and referrals, regulatory action will be taken as needed throughout the lifecycle of the product. Therefore, as a general rule a marketing authorisation should be granted for an unlimited time; however, one renewal may be decided only on justified grounds related to the safety of the medicinal product.

Or. en

Amendment 291

Tilly Metz

Proposal for a regulation

Recital 51

Text proposed by the Commission

Amendment

(51) As a general rule a marketing authorisation should be granted for an unlimited time; however, one renewal may be decided only on justified grounds related to the safety of the medicinal product.

(51) As a general rule a marketing authorisation for products other than generics should be granted for a period of five years in order to allow for the integration of real world evidence and re-assessment of the risk-benefit balance and in case of orphan medicines, also the related criteria related to population size and generated profit; once renewed, the marketing authorisation shall be valid for an unlimited period, unless the Agency decides on justified grounds related to the safety of the medicinal product to proceed with an additional five-year renewal period or revocation of the marketing authorisation.

Or. en

Amendment 292

Stanislav Polčák

Proposal for a regulation

Recital 51

Text proposed by the Commission

Amendment

(51) As a general rule a marketing authorisation should be granted for an unlimited time; however, one renewal may be decided only on justified grounds related to the safety of the medicinal product.

(51) As a general rule a marketing authorisation should be granted for an unlimited time; however, the granting of marketing authorisation for a limited period may be decided only on justified grounds related to the safety of the medicinal product. The marketing authorisation holder should be given the opportunity to apply for an extension of the marketing authorisation. If granted by the Commission, the extension of the marketing authorisation should be valid for an indefinite period.

Or. cs

Amendment 293

Tilly Metz

Proposal for a regulation

Recital 51 a (new)

Text proposed by the Commission

Amendment

(51 a) As a general rule, marketing authorisation should be granted based on comparative clinical trials on patients who are representative of the population to be treated with the product. To this end, placebo controlled trials should only be accepted where duly justified, necessary and ethical. In addition, patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) should be an integral part of clinical data submitted with the marketing authorisation application in order to assess the quality of care and the impact of the treatments on patients.

Or. en

Amendment 294

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 53

Text proposed by the Commission

Amendment

(53) Environmental risks may arise from medicinal products containing or consisting of genetically modified organisms. It is thus necessary to subject such medicinal products to an environmental risk-assessment procedure similar to the procedure under Directive 2001/18/EC of the European Parliament and of the Council51 , to be conducted in parallel with the evaluation, under a single Union procedure, of the quality, safety and efficacy of the medicinal product concerned. The environmental risk-assessment should be conducted in accordance with the requirements set out in this Regulation and in [revised Directive 2001/83/EC] which are based on the principles set out in Directive 2001/18/EC but taking into account the specificities of medicinal products.

(53) Environmental risks may arise from medicinal products containing or consisting of genetically modified organisms. It is thus necessary to subject such medicinal products to an environmental risk-assessment procedure similar to the procedure under Directive 2001/18/EC of the European Parliament and of the Council, to be conducted in parallel with the evaluation, under a single Union procedure, of the quality, safety and efficacy of the medicinal product and the risks to the environment. Where the environmental risks are absent or negligible, the normal requirements for the environmental risk assessment shall apply. To ensure the Union legislation on genetically modified organisms does not impede the development and approval of novel medicines containing and consisting of genetically modified organisms, it is also appropriate to foresee the adaptation of this Regulation and [revised Directive 2001/83/EC] to lay down specific rules for these categories of medicines on the basis on an evaluation.

_________________

51 Directive 2001/18/EC of the European Parliament and of the Council of 12 March 2001 on the deliberate release into the environment of genetically modified organisms and repealing Council Directive 90/220/EEC (OJ L 106, 17.4.2001, p. 1).

Or. en

Amendment 295

Susana Solís Pérez, Ondřej Knotek

Proposal for a regulation

Recital 53 a (new)

Text proposed by the Commission

Amendment

(53 a) The distinct properties of advanced therapy medicinal products (ATMPs) create substantial infrastructural and knowledge hurdles, along with systemic obstacles, making the 'release and continuous supply' of many ATMPs across all 27 Member States within a brief period challenging. It is essential to investigate alternative care options to ensure the availability of these therapies throughout the Member States, potentially utilizing frameworks for cross-border healthcare access, like Directive 2011/24/EU and Regulation (EC) No 883/2004.

Or. en

Amendment 296

Stelios Kympouropoulos, Pernille Weiss

Proposal for a regulation

Recital 53 a (new)

Text proposed by the Commission

Amendment

(53 a) Several care pathways should be explored to make therapies available in all Member States, including by advancing provisions for access to cross border care such as Directive 2011/24/EU and Regulation (EC) No 883/2004. This is particularly important for the advanced therapy medicinal products (ATMPs), as their unique characteristics result in significant infrastructural complexities and system barriers, which can substantially limit their continuously supply.

Or. en

Amendment 297

Stanislav Polčák

Proposal for a regulation

Recital 54

Text proposed by the Commission

Amendment

(54) [revised Directive 2001/83/EC] permits Member States to temporarily allow the use and supply of unauthorised medicinal products for public health reasons or individual patient needs and that includes medicinal products to be authorised under this Regulation. It is also necessary, that Member States are allowed under this Regulation to make a medicinal product available for compassionate use prior to its marketing authorisation. In those exceptional and urgent situations, where there is a lack of a suitable authorised medicinal product, the need to protect public health or the health of individual patients must prevail over other considerations, in particular the need to obtain a marketing authorisation and consequently, to have available complete information about the risks posed by the medicinal product, including any risks to the environment from medicinal products containing or consisting of genetically modified organisms (GMOs). To avoid delays in making these products available or uncertainties as regards their status in certain Member States, it is appropriate, in those exceptional and urgent situations, that for a medicinal product containing or consisting of GMOs, an environmental risk assessment or consent in accordance with Directive 2001/18/EC or Directive 2009/41/EC of the European Parliament and of the Council52should not be a prerequisite. Nevertheless, in these cases, Member States should implement appropriate measures to minimise foreseeable negative environmental impacts resulting from the intended or unintended release of the medicinal products containing or consisting of GMOs into the environment.

(54) [revised Directive 2001/83/EC] permits Member States to temporarily allow the use and supply of unauthorised medicinal products for public health reasons or individual patient needs and that includes medicinal products to be authorised under this Regulation. It is also necessary, that Member States are allowed under this Regulation to make a medicinal product available for compassionate use prior to its marketing authorisation. In those exceptional and urgent situations, where there is a lack of a suitable authorised medicinal product, the need to protect public health or the health of individual patients must prevail over other considerations, in particular the need to obtain a marketing authorisation and consequently, to have available complete information about the risks posed by the medicinal product, including any risks to the environment from medicinal products containing or consisting of genetically modified organisms (GMOs). To avoid delays in making these products available or uncertainties as regards their status in certain Member States, it is appropriate, in those exceptional and urgent situations, that for a medicinal product containing or consisting of GMOs, an environmental risk assessment or consent in accordance with Directive 2001/18/EC or Directive 2009/41/EC of the European Parliament and of the Council[1] should not be a prerequisite. Nevertheless, in these cases, Member States should implement appropriate measures in line with the precautionary principle to minimise negative environmental impacts resulting from the intended or unintended release of the medicinal products containing or consisting of GMOs into the environment. [1] Directive 2009/41/EC of the European Parliament and of the Council of 6 May 2009 on the contained use of genetically modified micro-organisms (Recast) (OJ L 125, 21.5.2009, p. 75).

_________________

52 Directive 2009/41/EC of the European Parliament and of the Council of 6 May 2009 on the contained use of genetically modified micro-organisms (Recast) (OJ L 125, 21.5.2009, p. 75).

Or. cs

Amendment 298

Tilly Metz

Proposal for a regulation

Recital 54

Text proposed by the Commission

Amendment

(54) [revised Directive 2001/83/EC] permits Member States to temporarily allow the use and supply of unauthorised medicinal products for public health reasons or individual patient needs and that includes medicinal products to be authorised under this Regulation. It is also necessary, that Member States are allowed under this Regulation to make a medicinal product available for compassionate use prior to its marketing authorisation. In those exceptional and urgent situations, where there is a lack of a suitable authorised medicinal product, the need to protect public health or the health of individual patients must prevail over other considerations, in particular the need to obtain a marketing authorisation and consequently, to have available complete information about the risks posed by the medicinal product, including any risks to the environment from medicinal products containing or consisting of genetically modified organisms (GMOs). To avoid delays in making these products available or uncertainties as regards their status in certain Member States, it is appropriate, in those exceptional and urgent situations, that for a medicinal product containing or consisting of GMOs, an environmental risk assessment or consent in accordance with Directive 2001/18/EC or Directive 2009/41/EC of the European Parliament and of the Council52 should not be a prerequisite. Nevertheless, in these cases, Member States should implement appropriate measures to minimise foreseeable negative environmental impacts resulting from the intended or unintended release of the medicinal products containing or consisting of GMOs into the environment.

(54) [revised Directive 2001/83/EC] permits Member States to temporarily allow the use and supply of unauthorised medicinal products for public health reasons or individual patient needs and that includes medicinal products to be authorised under this Regulation. It is also necessary, that Member States are allowed under this Regulation to make a medicinal product available for compassionate use prior to its marketing authorisation. In those exceptional and urgent situations, where there is a lack of a suitable authorised medicinal product, the need to protect public health or the health of individual patients must prevail over other considerations, in particular the need to obtain a marketing authorisation and consequently, to have available complete information about the risks posed by the medicinal product, including any risks to the environment from medicinal products containing or consisting of genetically modified organisms (GMOs). To avoid delays in making these products available or uncertainties as regards their status in certain Member States, it is appropriate, in those exceptional and urgent situations, that for a medicinal product containing or consisting of GMOs, an environmental risk assessment or consent in accordance with Directive 2001/18/EC or Directive 2009/41/EC of the European Parliament and of the Council52 should not be a prerequisite. Nevertheless, in these cases, Member States should implement appropriate measures to prevent foreseeable negative environmental impacts resulting from the intended or unintended release of the medicinal products containing or consisting of GMOs into the environment and agree on an appropriate timeline for the delivery of the environmental risk data.

_________________

52 Directive 2009/41/EC of the European Parliament and of the Council of 6 May 2009 on the contained use of genetically modified micro-organisms (Recast) (OJ L 125, 21.5.2009, p. 75).

Or. en

Amendment 299

Maria Angela Danzì

Proposal for a regulation

Recital 56 a (new)

Text proposed by the Commission

Amendment

(56a) To prevent delays in treatment and to provide timely access for patients to medicines undergoing clinical trials, the EU body responsible for centralised procurement of medicinal products shall use the framework agreement, as regulated by Directive 2014/24/EU, for the purchase of experimental drugs, as soon as the first phase of Regulation (EU) No 536/2014 has been completed. It is also recommended that the Member States use that institution.

Or. it

Amendment 300

Peter Liese

Proposal for a regulation

Recital 56 a (new)

Text proposed by the Commission

Amendment

(56 a) The role of patient organisations is crucial in European Health Policy. Their finance, particularly their finance independent of individual pharmaceutical companies, is of particular importance. That is why the Commission should examine the situation and, if appropriate, make proposals to strengthen independent financing.

Or. en

Amendment 301

Cyrus Engerer

Proposal for a regulation

Recital 57 a (new)

Text proposed by the Commission

Amendment

(57 a) Given the unmet needs in the area of mental health, the revision shall support early access to novel treatments for patients who need them most through means like compassionate use programmes and conditional marketing authorisation. These programs shall play a pivotal role in providing treatment experience for providers and generating valuable real-world data to inform safety policies and future product labelling.

Or. en

Amendment 302

Cyrus Engerer

Proposal for a regulation

Recital 57 b (new)

Text proposed by the Commission

Amendment

(57 b) Use of early access pilot programs to treat a diverse set of patients with complex comorbidities who are often excluded from clinical trials focused on novel mental health treatments should be supported. Allowing this would support gathering critical additional data on the safety and efficacy of these treatments in a broader population. Furthermore, these programs shall explore different treatment protocols, such as group therapy and peer support specialists, to find the optimal balance of safety, efficacy, affordability, and equitable access.

Or. en

Amendment 303

Cyrus Engerer

Proposal for a regulation

Recital 57 c (new)

Text proposed by the Commission

Amendment

(57 c) If an applicant for compassionate use is an SME, research institute, or not-for-profit entity, the administrative burden associated with making available for compassionate use a medicinal product for human use shall be reduced. This might include procedural and administrative assistance by the national competent authorities of the Member States and the Agency.

Or. en

Amendment 304

Tilly Metz

Proposal for a regulation

Recital 58

Text proposed by the Commission

Amendment

(58) There is the possibility under certain circumstances for marketing authorisations to be granted, subject to specific obligations or conditions, on a conditional basis or under exceptional circumstances. The legislation should allow under similar circumstances for medicinal products with a standard marketing authorisation for new indications to be authorised on a conditional basis or under exceptional circumstances. The medicinal products authorised on a conditional basis or under exceptional circumstances should in principle satisfy the requirements for a standard marketing authorisation with the exception of the specific derogations or conditions outlined in the relevant conditional or exceptional marketing authorisation and shall be subject to specific review of the fulfilment of the imposed specific conditions or obligations. It is also understood that the grounds for refusal of a marketing authorisation shall apply mutatis mutandis for such cases.

(58) There is the possibility under certain duly justified circumstances for marketing authorisations to be granted, subject to specific obligations or conditions, on a conditional basis or under exceptional circumstances. The legislation should allow under similar circumstances for medicinal products with a standard marketing authorisation for new indications to be authorised on a conditional basis or under exceptional circumstances. The medicinal products authorised on a conditional basis or under exceptional circumstances should in principle satisfy the requirements for a standard marketing authorisation with the exception of the specific derogations or conditions outlined in the relevant conditional or exceptional marketing authorisation and shall be subject to specific review of the fulfilment of the imposed specific conditions or obligations. It is also understood that the grounds for refusal of a marketing authorisation shall apply mutatis mutandis for such cases.

Or. en

Amendment 305

Stelios Kympouropoulos

Proposal for a regulation

Recital 60

Text proposed by the Commission

Amendment

(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. Where necessary the Agency may cooperate with the competent authorities of the Member States towards this objective.

(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, and/or data generated via in silico methods, such as computational modelling and simulation, digital molecular representation and mechanistic modelling, digital twin and artificial intelligence (AI). 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, including results of studies conducted via insilico methods, to fulfil its mandate, without compromising privacy rights. Where necessary the Agency may cooperate with the competent authorities of the Member States towards this objective.

Or. en

Justification

The use of in silico methods (or predictive medicine) for regulatory approval in healthcare can be time and cost effective, leading to the prevention of critical medicines shortages, and enhancing patients' timely access to treatment.

Amendment 306

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Erik Poulsen

Proposal for a regulation

Recital 60

Text proposed by the Commission

Amendment

(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 and/or through the use of in silico methods, such as computational modelling and simulation (CM&S) which includes PBPK, molecular modelling and mechanistic modelling, digital twin & artificial intelligence (AI). 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. Where necessary the Agency may cooperate with the competent authorities of the Member States towards this objective.

Or. en

Amendment 307

Tiemo Wölken

Proposal for a regulation

Recital 65

Text proposed by the Commission

Amendment

(65) In the preparation of scientific advice and in duly justified cases, the Agency should also be able to consult authorities established in other relevant Union legal acts or other public bodies established in the Union, as applicable. These may include experts in clinical trials, medical devices, substances of human origin or any other as required for the provision of the scientific advice in question.

(65) In the preparation of scientific advice and in duly justified cases, the Agency should consult authorities established in other relevant Union legal acts or other public bodies established in the Union, as applicable. These may include experts in clinical trials, medical devices, substances of human origin or any other as required for the provision of the scientific advice in question.

Or. en

Amendment 308

Tilly Metz

Proposal for a regulation

Recital 65

Text proposed by the Commission

Amendment

(65) In the preparation of scientific advice and in duly justified cases, the Agency should promote an open discussion about latest scientific developments and the update of scientific guidelines and should be able to consult authorities established in other relevant Union legal acts or other public bodies established in the Union, as applicable. These may include experts in clinical trials, medical devices, substances of human origin or any other as required for the provision of the scientific advice in question.

Or. en

Amendment 309

István Ujhelyi

Proposal for a regulation

Recital 66

Text proposed by the Commission

Amendment

(66) Through the Priority Medicines (PRIME) scheme, the Agency has gained experience of the provision of early scientific and regulatory support to developers of certain medicinal products that, based on preliminary evidence, are likely to address an unmet medical need and are considered promising at an early stage of development. It is appropriate to recognise this early support mechanism, including for priority antimicrobials and repurposed medicinal products when they fulfil the criteria for the scheme, and allow the Agency, in consultation with the Member States and the Commission, to establish selection criteria for promising medicinal products.

(66) Expedited Regulatory Pathways are a well-utilised tool to make assessments of pharmaceuticals more efficient and to modernise regulatory systems. Through the Priority Medicines (PRIME) scheme, the Agency has gained experience of the provision of early scientific and regulatory support to developers of certain medicinal products that, based on preliminary evidence, are likely to address an unmet medical need and are considered promising at an early stage of development. Given the objectives of this Regulation – to futureproof and simplify the European regulatory system, it is appropriate to recognise and strengthen this early support mechanism, including for priority antimicrobials and repurposed medicinal products when they fulfil the criteria for the scheme, and allow the Agency, in consultation with the Member States and the Commission, to establish selection criteria for promising medicinal products.

Or. en

Amendment 310

Billy Kelleher, Catherine Amalric

Proposal for a regulation

Recital 66

Text proposed by the Commission

Amendment

Or. en

Amendment 311

Tilly Metz

Proposal for a regulation

Recital 67

Text proposed by the Commission

Amendment

(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.

(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 public health need and 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.

Or. en

Amendment 312

Andrey Slabakov

Proposal for a regulation

Recital 67 a (new)

Text proposed by the Commission

Amendment

(67 a) To ensure patients with unmet medical needs, or suffering from life-threatening, or highly debilitating conditions treatable through the use or implant of a medical device are subject to the same quality, access and availability of treatment as other patients, EMA should have a a greater role in the evaluation of all health products and technologies. This may include the introduction of a dedicated priority review process which enhances patient access and facilitates interaction and early dialogue with manufacturers of certain class III and class IIb medical devices addressing unmet needs.

Or. en

Amendment 313

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 68

Text proposed by the Commission

Amendment

(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 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.

(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. The decision to grant conditional authorization will be subjected to the principles of risk-mitigation, risk assessment, informed consent, close monitoring, transparency, following strict procedures of monitoring and reporting.

Or. en

Amendment 314

Peter Liese

Proposal for a regulation

Recital 68 a (new)

Text proposed by the Commission

Amendment

(68 a) There is still a lack of sufficiently detailed and comparable data at Union level to determine the trends and identify possible risk factors that could lead to the development of further measures to limit the risk from antimicrobial resistance and to monitor the effect of measures already introduced. Therefore it is important to collect data on the sales and use of antimicrobials, and data on antimicrobial resistant organisms found in animals, humans and food. To ensure that the information collected can be used effectively, appropriate rules should be laid down concerning the collection and the exchange of data. The Member States should be responsible for collecting data on the use of antimicrobials under the coordination of the Agency.

Or. en

Amendment 315

Stanislav Polčák

Proposal for a regulation

Recital 70

Text proposed by the Commission

Amendment

(70) In the event of a risk to public health, the marketing authorisation holder or the competent authorities should be able to make urgent safety or efficacy restrictions on their own initiative to ensure a swift adaption of the marketing authorisation to maintain the safe and efficacious use of the medicinal product by healthcare professionals and patients. If a review is launched on the same safety or efficacy concern addressed by urgent restrictions initiated by a competent authority, any written observations by the marketing authorisation holder should be considered in that review to avoid duplication of assessment.

(70) In the event of a risk to public health or the environment, the marketing authorisation holder or the competent authorities should be able to make urgent safety or efficacy restrictions on their own initiative to ensure a swift adaption of the marketing authorisation to maintain the safe and efficacious use of the medicinal product by healthcare professionals and patients. If a review is launched on the same safety or efficacy concern addressed by urgent restrictions initiated by a competent authority, any written observations by the marketing authorisation holder should be considered in that review to avoid duplication of assessment.

Or. cs

Amendment 316

Andreas Glück, Frédérique Ries, Catherine Amalric

Proposal for a regulation

Recital 71 a (new)

Text proposed by the Commission

Amendment

(71 a) Phased reviews have been a success during the COVID-19 pandemic and led to a rapid authorisation of the urgently needed vaccines. An application in times outside of public health emergencies is therefore appropriate and the procedure should be extended to orphan medicinal products and medicinal products that are likely to offer an exceptional therapeutic advancement in the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition. Moreover, the Commission should evaluate the performance of the phased reviews with the aim to further expand the category of medicinal products for which this procedure may apply.

Or. en

Justification

See AM on Article 6 – paragraph 2 – subparagraph 1

Amendment 317

Stanislav Polčák

Proposal for a regulation

Recital 72

Text proposed by the Commission

Amendment

(72) To avoid unnecessary administrative and financial burden both for the pharmaceutical industry and the competent authorities, certain streamlining measures should be introduced. Electronic applications for marketing authorisations and for variations to the terms of the marketing authorisation should be made possible.

deleted

Or. cs

Amendment 318

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Ondřej Knotek, Erik Poulsen

Proposal for a regulation

Recital 73

Text proposed by the Commission

Amendment

(73) To optimise the use of resources for both applicants for marketing authorisations and competent authorities assessing such applications, a single assessment of an active substance master file should be introduced. The outcome of the assessment should be issued through a certificate. To avoid duplication of assessment, the use of an active substance master file certificate should be mandatory for subsequent applications or marketing authorisations for medicinal products for human use containing that active substance from an active substance master file certification holder. The Commission should be empowered to establish the procedure for the single assessment of an active substance master file. To further optimise the use of resources, the Commission should be empowered to extend the certification scheme to additional quality master files, e.g. in case of novel excipients, adjuvants, radiopharmaceutical precursors and active substance intermediates, when the intermediate is a chemical active substance by itself or used in conjugation with a biological substance.

(73) To optimise the use of resources for both applicants for marketing authorisations and competent authorities assessing such applications, a single assessment of an active substance master file should be introduced. The outcome of the assessment should be issued through a certificate. To avoid duplication of assessment, the use of an active substance master file certificate should be mandatory for subsequent applications or marketing authorisations for medicinal products for human use containing that active substance from an active substance master file certification holder. The Commission should be empowered to establish the procedure for the single assessment of an active substance master file. To further optimise the use of resources, the Commission should be empowered to extend the certification scheme to additional master files, such as quality master files for active substances other than chemical active substances or for other substances present or used in the manufacture of a medicinal product e.g. in case of novel excipients, adjuvants, raw materials, viral vectors and other starting materials, growth media, radiopharmaceutical precursors, active substance intermediates and conjugates, or such as platform technology master files for platform technologies used in the manufacturing process of one or more medicinal products.

Or. en

Amendment 319

Susana Solís Pérez, Catherine Amalric

Proposal for a regulation

Recital 73

Text proposed by the Commission

Amendment

(73) To optimise the use of resources for both applicants for marketing authorisations and competent authorities assessing such applications, a single assessment of an active substance master file should be introduced. The outcome of the assessment should be issued through a certificate. To avoid duplication of assessment, the use of an active substance master file certificate should be mandatory for subsequent applications or marketing authorisations for medicinal products for human use containing that active substance from an active substance master file certification holder. The Commission should be empowered to establish the procedure for the single assessment of an active substance master file. To further optimise the use of resources, the Commission should be empowered to extend the certification scheme to additional quality master files, e.g. in case of novel excipients, adjuvants, raw materials, viral vectors and other starting materials, growth media, radiopharmaceutical precursors and active substance intermediates, when the intermediate is a chemical active substance by itself or used in conjugation with a biological substance.

Or. en

Amendment 320

István Ujhelyi

Proposal for a regulation

Recital 73

Text proposed by the Commission

Amendment

(73) To optimise the use of resources for both applicants for marketing authorisations and competent authorities assessing such applications, a single assessment of an active substance master file should be introduced. The outcome of the assessment should be issued through a certificate. To avoid duplication of assessment, the use of an active substance master file certificate should be mandatory for subsequent applications or marketing authorisations for medicinal products for human use containing that active substance from an active substance master file certification holder. The Commission should be empowered to establish the procedure for the single assessment of an active substance master file. To further optimise the use of resources, the Commission should be empowered to extend the certification scheme to additional quality master files, e.g. in case of novel excipients, adjuvants, raw materials, viral vectors and other starting materials, growth media, radiopharmaceutical precursors and active substance intermediates, when the intermediate is a chemical active substance by itself or used in conjugation with a biological substance

Or. en

Amendment 321

Stanislav Polčák

Proposal for a regulation

Recital 74

Text proposed by the Commission

Amendment

(74) To avoid unnecessary administrative and financial burdens for applicants, marketing authorisation holders and competent authorities, certain streamlining measures should be introduced. Electronic application for marketing authorisation and for variations to the terms of the marketing authorisation should be introduced. For generic and biosimilar medicinal products, except in specific cases, risk management plans do not need to be developed and submitted to the competent authorities.

deleted

Or. cs

Amendment 322

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 76

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 323

Tilly Metz

Proposal for a regulation

Recital 76

Text proposed by the Commission

Amendment

(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. The decision of the Commission should be based on the opinion issued by the Agency, detailing how the medicinal product could be effective in treating, preventing or diagnosing the disease or condition directly related to the public health emergency and how the benefits of using the product outweigh the risks. In that effect, and given the limited knowledge of both the benefits and risks, the Agency should only take into account plausible expectations of benefits which should be grounded on the data available at the time of the opinion. 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. en

Amendment 324

Kateřina Konečná

Proposal for a regulation

Recital 76

Text proposed by the Commission

Amendment

(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 a standard or conditional marketing authorisation has been granted for the relevant indication.

Or. en

Amendment 325

Tomislav Sokol

Proposal for a regulation

Recital 76

Text proposed by the Commission

Amendment

(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 a standard or conditional marketing authorisation has been granted for the relevant indication.

Or. en

Amendment 326

Peter Liese

Proposal for a regulation

Recital 77

Text proposed by the Commission

Amendment

(77) The development of antimicrobial resistance is a growing concern and the pipeline of effective antimicrobials is obstructed due to a market failure; 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.

(77) The development of antimicrobial resistance is a growing health problem in the Union and worldwide, thus involving a common responsibilty of all actors concerned. The pipeline of effective antimicrobials is obstructed due to a market failure; it is therefore necessary to maintain the efficacy of existing antimicrobials for as long as possible and to consider a number of 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.

Or. en

Amendment 327

Tiemo Wölken

Proposal for a regulation

Recital 77

Text proposed by the Commission

Amendment

(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. It is equally necessary to support research and development with an aim to develop alternative treatments such as bacteriophages, that are effective against multi-drug resistant bacteria and can be used as alternative or together with antibiotics.

Or. en

Amendment 328

Sirpa Pietikäinen

Proposal for a regulation

Recital 77

Text proposed by the Commission

Amendment

(77) The development of antimicrobial resistance is a growing concern and the pipeline of effective antimicrobials is obstructed due to a market failure whereby access dynamics at Member State level constitute a significant disincentive for companies to invest in the development of novel antibiotics; 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.

Or. en

Amendment 329

Andrey Slabakov

Proposal for a regulation

Recital 77

Text proposed by the Commission

Amendment

(77) The development of antimicrobial resistance is a growing concern and the pipeline of effective antimicrobials is obstructed due to a market failure, whereby antimicrobial R&D is hampered by the low commercial value of antimicrobial medicinal products; 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.

Or. en

Amendment 330

Annalisa Tardino

Proposal for a regulation

Recital 77

Text proposed by the Commission

Amendment

(77) The development of antimicrobial resistance is a growing concern and the pipeline of effective antimicrobials is obstructed due to a market failure; it is therefore necessary to consider new measures to incentivize companies' investments in research and development and 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.

Or. en

Amendment 331

Tilly Metz

Proposal for a regulation

Recital 77

Text proposed by the Commission

Amendment

Or. en

Amendment 332

Tilly Metz

Proposal for a regulation

Recital 78

Text proposed by the Commission

Amendment

(78) To be considered a ‘priority antimicrobial’, a medicinal product should represent a real advancement against antimicrobial resistance and should therefore bring forward non-clinical and clinical data that underpin a significant clinical benefit with respect to antimicrobial resistance. When assessing the conditions for antibiotics, the Agency shall take into account the prioritisation of pathogens as regards the risk of antimicrobial resistance provided for in the ‘WHO priority pathogens list for R&D of new antibiotics’, specifically those listed as priority 1 (critical) or priority 2 (high) or in case there is an equivalent list of priority pathogens adopted at Union level, the Agency should take such Union list into account as a priority. In order to address market failures for the development of antimicrobials, the priority focus should be on the research and development and subsequent production and fair distribution of new antimicrobials. However, addressing AMR will not be solved by R&D alone. To ensure prudent use of existing antibiotics, the Authority should also support the development and procurement of rapid diagnostic tools to ensure appropriate prescriptions.

Or. en

Amendment 333

Tilly Metz

Proposal for a regulation

Recital 78 a (new)

Text proposed by the Commission

Amendment

(78 a) To seriously tackle major ongoing public health challenges, the antimicrobial resistance in particular, while also building on existing resources, the Health Emergency Preparedness and Response Authority (HERA) should be established as a separate structure under the legal personality of the European Centre for Disease Prevention and Control (ECDC) to boost EU’s capability to address health emergencies. The Authority should be responsible for creating, coordinating and implementing the long-term European portfolio of biomedical research and development agenda for medical countermeasures against current and emerging public health threats, as well as providing tools to ensure EU-wide access to these products, including those to support production, procurement, stockpiling and distribution capacity of medical countermeasures and other priority medical products in the EU. The Authority will play a crucial role in addressing health threats globally. The Authority should primarily focus on the fight against antimicrobial resistance and the development of new antimicrobials and medical countermeasures linked to public health emergencies. However, in the future with increasing capacity, the Authority should expand the scope of its agenda, specifically to tackle other areas of unmet medical need such as rare and neglected diseases. The Authority should have adequate resources to fulfil its mandate.

Or. en

Amendment 334

Andrey Slabakov

Proposal for a regulation

Recital 78 a (new)

Text proposed by the Commission

Amendment

(78 a) Addressing Antimicrobial Resistance requires a rethinking of the approach to incentives both at Union and national level. Member States must be willing to re-valuate their commitment to generating pharmaceutical R&D in key areas of priority and in public expenditure directed towards health. The European Commission, the Council and the European Parliament have published a large number of studies and recommendations, addressing the need to refocus on producing novel antimicrobials as soon as possible. These approaches need to be evaluated, consolidated and made operational under a single, comprehensive long-term Action Plan on addressing Antimicrobial Resistance in the Union, which will contain all the necessery measures to stimulate research and developing novel antimicrobials.

Or. en

Amendment 335

Maria Angela Danzì

Proposal for a regulation

Recital 78 a (new)

Text proposed by the Commission

Amendment

(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 EU level is required to meet the public health needs of EU citizens. In particular, there is a stark misalignment between the R&D priorities of the pharmaceutical industry and the public health needs of EU citizens. The market failures in the Union have, in certain instances, also led to excessive prices for medicinal products and led to shortages of medicinal products. This Regulation should, therefore, with a view to achieving synergies and partnerships between the public and private sectors, address market failures of that kind .

Or. it

Amendment 336

Tiemo Wölken

Proposal for a regulation

Recital 78 a (new)

Text proposed by the Commission

Amendment

(78 a) In response to the COVID-19 pandemic, to counteract market failures exacerbated by the public health crisis and to better respond to cross-border health threats, the Commission service HERA was set up under Commission Decision C(2021) 6712. HERA is tasked to strengthen the Union’s ability to prevent, detect and rapidly respond, by ensuring the supply of crisis relevant medical countermeasures, including through their monitoring, procurement and purchase, by activating emergency research and innovation plans, providing emergency funding and financing, and by taking measures concerning the production, availability and supply of such key medical countermeasures.

Or. en

Amendment 337

Maria Angela Danzì

Proposal for a regulation

Recital 78 b (new)

Text proposed by the Commission

Amendment

(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 of oncology and 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 on health.

Or. it

Amendment 338

Tilly Metz

Proposal for a regulation

Recital 78 b (new)

Text proposed by the Commission

Amendment

(78 b) As the Commission’s study on bringing antimicrobial medical countermeasures to the market1a demonstrated, different kinds of push and pull incentives are needed to face this public health emergency. These tools may include market entry rewards, advance purchase agreements, milestone payments, innovation prizes, or subscription payments. In order to maximize the benefits from such public investments, the allocation of these financial and other incentives should respect the following principles and conditions of open science, affordability and EU-wide availability of developed products, delinking revenue from sale volumes of procured products, full transparency of all received funding and purchase agreements, gradient incentive scheme that rewards according to the innovation level, and the development of a stewardship and access plans. These principles and conditions should ensure that public money will be allocated with the objective of a rapid public return on investment for patients.

_________________

1a https://op.europa.eu/en/publication-detail/-/publication/51b2c82c-c21b-11ed-8912-01aa75ed71a1/language-en/format-PDF/source-282347876

Or. en

Amendment 339

Andrey Slabakov

Proposal for a regulation

Recital 78 b (new)

Text proposed by the Commission

Amendment

(78 b) To ensure that the Union has the most efficient and rapid access to innovations in priority antimicrobials, a short-term incentive measure must be put in place to bridge the gap until the Action Plan begins to have effect on the research and development ecosystem and on the pharmaceutical market for antimicrobials. The transferable exclusivity voucher is, therefore, introduced as a short-term tool to incentivise innovation.

Or. en

Amendment 340

Tiemo Wölken

Proposal for a regulation

Recital 78 b (new)

Text proposed by the Commission

Amendment

(78 b) However with the increasing awareness and need for transparency and accountability in expenditure on public health measures and the importance of the missions and responsibilities of HERA it is considered appropriate that it is subject to the same level of oversight and scrutiny requirements as other agencies established under Union legislation such as the ECDC and the EMA.

Or. en

Amendment 341

Maria Angela Danzì

Proposal for a regulation

Recital 78 c (new)

Text proposed by the Commission

Amendment

(78b) 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 at EU of mission-oriented R&D infrastructure which acts in the public interest is a tool to alleviate those market failures.This public law infrastructure must be able to carry out research and development activities concerning novel antimicrobials, and in other sectors where unmet medical needs are common.

What is more, with a view to overcoming market failures of that kind, more preliminary purchasing agreements must be concluded and joint procurement must be carried out, with the aim of providing the EU and its Member States with greater flexibility in line with their needs and of ensuring that medicines are available for all EU residents, irrespective of their Member State of origin.

Or. it

Amendment 342

Tiemo Wölken

Proposal for a regulation

Recital 78 c (new)

Text proposed by the Commission

Amendment

(78 c) The abovementioned market failures highlight the need for paradigm shift in both the Member States and Union approach to generating pharmaceutical R&D in key areas of priority and in public expenditure directed towards health. It is therefore considered that the establishment of a mission oriented R&D and manufacturing infrastructure at Union level which acts in the public interest could alleviate these market failures. The European Medicines Facility (EMF) should therefore be established as an independent agency under this Regulation. The EMF would assume the existing mission and responsibilities of HERA and would be further tasked to research and develop novel antimicrobials as well as other areas of unmet medical need, to respond to health threats and emergencies, to support the Union to overcome market failures, to conduct treatment optimisation studies and to prevent shortages and guarantee security of supply of critical medicinal products.

Or. en

Amendment 343

Tilly Metz

Proposal for a regulation

Recital 78 c (new)

Text proposed by the Commission

Amendment

(78 c) The principle of open science is pivotal to ensure rapid progress in the field of scientific research for priority antimicrobials. Over the past 30 years, the lack of sharing of results, failed trials and ongoing research has created bottlenecks for scientific development and contributes to the current market failure for the placing on the market of new antimicrobials. It is therefore of the utmost importance to have a paradigm shift towards open science, particularly in the area of publicly-funded research, to reduce duplication of research, allow for peer-verification of results and building further evidence based on most recent findings, as to making research and development funding efforts more efficient. Indeed, it would also allow HERA to avoid funding research projects that would lead to similar mistakes being repeated over and over. Open science should serve as the basis for the coordinated research effort.

Or. en

Amendment 344

Tilly Metz

Proposal for a regulation

Recital 78 d (new)

Text proposed by the Commission

Amendment

(78 d) In order to ensure sufficient investment to address AMR, the European Antimicrobial Innovation Fund should be established. The Fund should generate and invest at least 1 billion EUR per year to address antimicrobial resistance. Since the manufacturing, use and disposal of antibiotics and other pharmaceutical substances in humans and animals and their prevalent occurance in the environment is the main cause of AMR and only a handful of developers are actively addressing this emergency, the collective effort, under the One Health Approach, of the industry and public authorities must be in place to solve the crisis. As per the polluter pays principle, companies selling medicines on the EU market, therefore contributing to the growing problem of environmental pollution including antimicrobial resistance, which do not wish to invest in R&D of antimicrobials should financially support, through annual penalty payments, the collective efforts to solving this public health emergency to the European Antimicrobial Innovation Fund. Other EU funding as well as voluntary contributions from philanthropic organisations, international schemes or Member States should represent complementary sources allocated to this Fund.

Or. en

Amendment 345

Maria Angela Danzì

Proposal for a regulation

Recital 78 d (new)

Text proposed by the Commission

Amendment

(78d) To prevent overlapping of competences and with a view to better coordination and efficiency of the use of public resources, the Commission must present a proposal, where necessary, to streamline and restore the competences of the European Medicines Agency (EMA), the Health Emergency Preparedness and Response Authority (HERA) and the European Centre for Disease Prevention and Control (ECDC).

Or. it

Amendment 346

Tiemo Wölken

Proposal for a regulation

Recital 78 e (new)

Text proposed by the Commission

Amendment

(78 e) The increasing prevalence and threat of antimicrobial resistance requires action on different fronts in parallel. In addition to the role of the European Medicines Facility, it is necessary to create a Union push and pull incentive scheme to promote and accelerate the development of novel antimicrobials. In this regard, the Commission should create and define a Union push and pull scheme as well as its funding mechanisms. Given the urgency of need for novel antimicrobials, the Commission should therefore have developed and began the implementation of the scheme by one year after the adoption of the Regulation.

Or. en

Amendment 347

Tiemo Wölken

Proposal for a regulation

Recital 78 f (new)

Text proposed by the Commission

Amendment

(78 f) Practices in procurement procedures for medicines differ between Member States and long-term availability is rarely a primary consideration. The 2014 Procurement Directive encourages a more strategic approach through award criteria, including criteria beyond price. Using the lowest price as the main selection criterion may reduce incentives for the industry to build for long-term supply in the EU. At the same time, vulnerability may be increased when public procurement procedures award contracts to a single company. Where challenges with access to a critical medicine and related affordability may be an issue, Member States can work together to increase buying power: Joint procurement between Member States can act as a powerful tool to improve access, affordability and security of supply, of particular benefit in smaller EU markets. This can improve the negotiating position of Member States to incentivise production capacities, as well as diversifying supply chains. In specific cases, those instruments could also support enhanced predictability through multi-annual contracts.

Or. en

Amendment 348

Tiemo Wölken

Proposal for a regulation

Recital 78 g (new)

Text proposed by the Commission

Amendment

(78 g) Coordination at EU level of medicinal products could offer a strategic frame to enhance security of supply of needed critical medicines through public procurement. This could draw on Commission guidance and common criteria for the procurement of critical medicines, such as green production and prioritisation of supplies in Europe at times of critical shortages. Such procurement efforts should also be based on the principle of the ‘most economically advantageous tender’ (‘MEAT’ criteria), which aims to ensure the best value for money rather than most economically advantageous product. Such an approach could also help in defining adequate supply in relation to critical medicines and, thereby, compensate and incentivise industry, and support the application of these criteria in a coordinated way, at EU level. Predictability of supply would also be helped by medium-term contractual incentives to diversify and attract the next generation of manufacturing investments in Europe.

Or. en

Amendment 349

Maria Angela Danzì

Proposal for a regulation

Recital 79

Text proposed by the Commission

Amendment

(79) The creation of a voucher rewarding the development of priority antimicrobials through an additional year of regulatory data protection has the capacity to provide the needed financial support to developers of priority antimicrobials. However, in order to ensure that the financial reward which is ultimately borne by health systems is mostly absorbed by the developer of the priority antimicrobial and not the buyer of the voucher, the number of available vouchers on the market should be kept to a minimum. It is therefore necessary to establish strict conditions of granting, transfer and use of the voucher and to further give the possibility to the Commission to revoke the voucher under certain circumstances.

deleted

Or. it

Amendment 350

Catherine Amalric, Max Orville

Proposal for a regulation

Recital 79

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 351

Tilly Metz

Proposal for a regulation

Recital 79

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 352

Frédérique Ries, Catherine Amalric, Max Orville

Proposal for a regulation

Recital 79

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 353

Susana Solís Pérez, Ondřej Knotek

Proposal for a regulation

Recital 79

Text proposed by the Commission

Amendment

(79) The creation of a voucher rewarding the development of priority antimicrobials through an additional year of regulatory data protection, in combination with a set of push and pull incentives scheme, constitutes an alternative able to provide the needed financial support to developers of priority antimicrobials. However, in order to ensure that the financial reward which is ultimately borne by health systems is mostly absorbed by the developer of the priority antimicrobial and not the buyer of the voucher, the number of available vouchers on the market should be kept to a minimum. It is therefore necessary to establish strict conditions of granting, transfer and use of the voucher and to further give the possibility to the Commission to revoke the voucher under certain circumstances.

Or. en

Amendment 354

Andrey Slabakov

Proposal for a regulation

Recital 79

Text proposed by the Commission

Amendment

(79) The creation of a voucher rewarding the development of priority antimicrobials, particular narrow-spectrum antibiotics, through an additional year of regulatory data protection has the capacity to provide the needed financial support to developers. However, in order to ensure that the financial reward which is ultimately borne by health systems is mostly absorbed by the developer of the priority antimicrobial and not the buyer of the voucher, the number of available vouchers on the market should be kept to a minimum. It is therefore necessary to establish strict conditions of granting, transfer and use of the voucher and to further give the possibility to the Commission to revoke the voucher under certain circumstances.

Or. en

Amendment 355

Sirpa Pietikäinen

Proposal for a regulation

Recital 79 a (new)

Text proposed by the Commission

Amendment

(79 a) The fight against antimicrobial resistance will only be addressed through push (R&D focused) and pull (access focused) incentives at EU and national level. In order to provide the right signal on which products to incentivise at both EU and national level, the creation of an AMR Designation is needed. Such AMR Designation would allow the EU to identify all products which could contribute to the fight against antimicrobial resistance and would provide the necessary focus to coherently target R&D and access incentives.

Or. en

Amendment 356

Annalisa Tardino

Proposal for a regulation

Recital 79 a (new)

Text proposed by the Commission

Amendment

(79 a) In order to fight against antimicrobial resistance, push and pull incentives are to be considered essential at both Union and national level. In this regard, the creation of an AMR designation is needed to allow the identification of all products which could contribute to this aim and would provide the necessary focus to coherently target research and development and aceess incentives.

Or. en

Amendment 357

Maria Angela Danzì

Proposal for a regulation

Recital 80

Text proposed by the Commission

Amendment

(80) A transferable data exclusivity voucher should only be available to those antimicrobial products that bring a significant clinical benefit with respect to antimicrobial resistance, and which have the characteristics described in this Regulation. It is also necessary to ensure that an undertaking which receives this incentive is in turn capable to supply the medicinal product to patients across the Union in sufficient quantities and to provide information on all funding received for research related to its development in order to provide a full account of the direct financial support given to the medicinal product.

deleted

Or. it

Amendment 358

Tilly Metz

Proposal for a regulation

Recital 80

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 359

Frédérique Ries, Catherine Amalric, Max Orville

Proposal for a regulation

Recital 80

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 360

Catherine Amalric, Max Orville

Proposal for a regulation

Recital 80

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 361

Andrey Slabakov

Proposal for a regulation

Recital 80

Text proposed by the Commission

Amendment

(80) A transferable data exclusivity voucher should only be available to narrow-spectrum antibiotics that bring a significant clinical benefit with respect to antimicrobial resistance, and which have the characteristics described in this Regulation. It is also necessary to ensure that an undertaking which receives this incentive is in turn capable to supply the medicinal product to patients across the Union in sufficient quantities and to provide information on all funding received for research related to its development in order to provide a full account of the direct financial support given to the medicinal product.

Or. en

Amendment 362

Susana Solís Pérez, Ondřej Knotek

Proposal for a regulation

Recital 80

Text proposed by the Commission

Amendment

(80) A transferable data exclusivity voucher and other push and pull incentives schemes to boost the development of priority antimicrobials should only be available to those antimicrobial products that bring a significant clinical benefit with respect to antimicrobial resistance, and which have the characteristics described in this Regulation. It is also necessary to ensure that an undertaking which receives this incentive is in turn capable to supply the medicinal product to patients across the Union in sufficient quantities and to provide information on all funding received for research related to its development in order to provide a full account of the direct financial support given to the medicinal product.

Or. en

Amendment 363

Maria Angela Danzì

Proposal for a regulation

Recital 81

Text proposed by the Commission

Amendment

(81) To ensure a high level of transparency and complete information on the economic effect of the transferable data exclusivity voucher, notably as regards the risk of overcompensation of investment, a developer of a priority antimicrobial is required to provide information on all direct financial support received for research related to the development of the priority antimicrobial. The declaration should include direct financial support received from any source worldwide.

deleted

Or. it

Amendment 364

Catherine Amalric, Max Orville

Proposal for a regulation

Recital 81

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 365

Tilly Metz

Proposal for a regulation

Recital 81

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 366

Frédérique Ries, Catherine Amalric, Max Orville

Proposal for a regulation

Recital 81

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 367

Maria Angela Danzì

Proposal for a regulation

Recital 82

Text proposed by the Commission

Amendment

(82) A transfer of a voucher for a priority antimicrobial may be conducted by sale. The value of the transaction which may be monetary or otherwise agreed between the buyer and the seller, shall be made public so as to inform regulators and the public. The identity of the holder of a voucher that has been granted and not yet used should be publicly known at all times so as to ensure a maximum level of transparency and trust.

deleted

Or. it

Amendment 368

Tilly Metz

Proposal for a regulation

Recital 82

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 369

Frédérique Ries, Catherine Amalric, Max Orville, Billy Kelleher

Proposal for a regulation

Recital 82

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 370

Catherine Amalric, Max Orville

Proposal for a regulation

Recital 82

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 371

Maria Angela Danzì

Proposal for a regulation

Recital 83

Text proposed by the Commission

Amendment

(83) The provisions related to transferable data exclusivity vouchers shall be applicable for a specified period from the entry into force of this Regulation or until a maximum number of vouchers are granted by the Commission in order to limit the total cost of the measure to Member State health systems. The limited application of the measure will also provide the possibility to assess the effect of the measure in addressing the market failure in the development of new antimicrobials addressing antimicrobial resistance and assess the cost on national health systems. Such assessment will provide the necessary knowledge to decide whether to extend the application of the measure.

deleted

Or. it

Amendment 372

Tilly Metz

Proposal for a regulation

Recital 83

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 373

Catherine Amalric, Max Orville

Proposal for a regulation

Recital 83

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 374

Frédérique Ries, Catherine Amalric, Max Orville, Billy Kelleher

Proposal for a regulation

Recital 83

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 375

Andrey Slabakov

Proposal for a regulation

Recital 83

Text proposed by the Commission

Amendment

(83) The provisions related to transferable data exclusivity vouchers shall be applicable for a specified period from the entry into force of this Regulation and until a specified time, as indicated in the Action Plan do address Antimicrobial Resistance in the Union, when the long-term measures foreseen in this Regulation commence to take effect. This will also limit the total cost of the voucher to Member State health systems. The limited application of the measure will also provide the possibility to assess the effect of the measure in addressing the market failure in the development of new antimicrobials addressing antimicrobial resistance and assess the cost on national health systems. Such assessment will provide the necessary knowledge to decide whether it should be extended in some form under the Action Plan.

Or. en

Amendment 376

Maria Angela Danzì

Proposal for a regulation

Recital 84

Text proposed by the Commission

Amendment

(84) The period of application of the provisions on transferable exclusivity vouchers for priority antimicrobials and the total number of vouchers may be extended by the Parliament and the Council upon proposal by the Commission on the basis of the experience acquired.

deleted

Or. it

Amendment 377

Frédérique Ries, Catherine Amalric, Max Orville, Billy Kelleher

Proposal for a regulation

Recital 84

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 378

Tilly Metz

Proposal for a regulation

Recital 84

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 379

Catherine Amalric, Max Orville

Proposal for a regulation

Recital 84

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 380

Tomislav Sokol, Stelios Kympouropoulos

Proposal for a regulation

Recital 84 a (new)

Text proposed by the Commission

Amendment

(84 a) The Transferable Exclusivity Vouchers (TEV) could be complemented by a procurement mechanism, as outlined in the Council Recommendation on “Stepping up actions to combat AMR in a One Health approach”, providing coordinated financial commitment across the EU and Member States. This would provide the predictability that will encourage comprehensive commercialisation of new priority antimicrobials.

Or. en

Amendment 381

Tilly Metz

Proposal for a regulation

Recital 86

Text proposed by the Commission

Amendment

(86) Medicinal products for rare diseases and for children should be subject to the same provisions as any other medicinal product concerning their quality, safety and efficacy, for example for what concerns the marketing authorisation procedures, the pharmacovigilance and quality requirements. However, specific requirements also apply to them. Such requirements, which are currently defined in separate legislations, should be integrated in this Regulation in order to ensure clarity and coherency of all the measures applicable to these medicinal products.

(86) Medicinal products for rare diseases and for children should be subject to the same provisions as any other medicinal product concerning their quality, safety, and efficacy and environmental risk, for example for what concerns the marketing authorisation procedures, the pharmacovigilance and quality requirements. However, specific requirements also apply to them. Such requirements, which are currently defined in separate legislations, should be integrated in this Regulation in order to ensure clarity and coherency of all the measures applicable to these medicinal products.

Or. en

Amendment 382

Tilly Metz

Proposal for a regulation

Recital 87

Text proposed by the Commission

Amendment

(87) Some orphan conditions occur so infrequently that the cost of developing and bringing to the market a medicinal product to diagnose, prevent or treat the condition cannot be recovered by the expected sales of the medicinal product. However, patients suffering from rare conditions should be entitled to the same quality of treatment as other patients; it is therefore necessary to stimulate the research, development and placing on the market of appropriate medications by the pharmaceutical industry.

Or. en

Justification

Should not be exclusively industry

Amendment 383

Susana Solís Pérez, Catherine Amalric

Proposal for a regulation

Recital 87 a (new)

Text proposed by the Commission

Amendment

(87 a) The European Parliament adopted a resolution of 10 July 2020 concerning the EU's post-COVID-19 public health strategy, which called for an EU Action Plan specifically targeting rare diseases.

Or. en

Amendment 384

Kateřina Konečná

Proposal for a regulation

Recital 87 a (new)

Text proposed by the Commission

Amendment

(87 a) The European Parliament adopted a resolution of 10 July 2020 on the EU’s public health strategy post-COVID-19, which requested an EU Action plan for rare diseases.

Or. en

Amendment 385

Andrey Slabakov

Proposal for a regulation

Recital 87 a (new)

Text proposed by the Commission

Amendment

(87 a) The European Parliament adopted a resolution of 10 July 2020 on the EU’s public health strategy post-COVID-19, which requested an EU Action plan for rare diseases

Or. en

Amendment 386

Tomislav Sokol

Proposal for a regulation

Recital 87 a (new)

Text proposed by the Commission

Amendment

(87 a) The European Parliament resolution of 10 July 2020 on the EU’s public health strategy post-COVID-19, called for an EU Action plan for rare diseases.

Or. en

Amendment 387

Andrey Slabakov

Proposal for a regulation

Recital 88

Text proposed by the Commission

Amendment

(88) Regulation (EC) No 141/2000 of the European Parliament and of the Council55 has proved to be successful in boosting developments of orphan medicinal products in the Union; therefore an action at Union level remains preferable to uncoordinated measures by the Member States which may result in distortions of competition and barriers to intra-Union trade. The Union should not neglect or disregard the success of the Regulation since its entry into force in 2000; it should build upon its success, in order to ensure an even greater degree of innovation under this Regulation;

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55 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p. 1).

Or. en

Amendment 388

Tomislav Sokol

Proposal for a regulation

Recital 88

Text proposed by the Commission

Amendment

(88) Regulation (EC) No 141/2000 of the European Parliament and of the Council55 has proved to be successful in boosting developments of orphan medicinal products in the Union; therefore an action at Union level remains preferable to uncoordinated measures by the Member States which may result in distortions of competition and barriers to intra-Union trade. The Union should not neglect or disregard the success of the Regulation since its entry into force in 2000, and build on its success driving and ensuring a similar degree of innovation under this Regulation.

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55 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p. 1).

Or. en

Amendment 389

Billy Kelleher, Catherine Amalric, Ondřej Knotek

Proposal for a regulation

Recital 88

Text proposed by the Commission

Amendment

(88) Regulation (EC) No 141/2000 of the European Parliament and of the Council55 has proved to be successful in boosting developments of orphan medicinal products in the Union; therefore an action at Union level remains preferable to uncoordinated measures by the Member States which may result in distortions of competition and barriers to intra-Union trade. The Union should not neglect or disregard the success of the Regulation since its entry into force in 2000, and build on its success driving and ensuring a similar degree of innovation under this Regulation.

_________________

55 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p. 1).

Or. en

Amendment 390

István Ujhelyi

Proposal for a regulation

Recital 88

Text proposed by the Commission

Amendment

(88) Regulation (EC) No 141/2000 of the European Parliament and of the Council has proved to be successful in boosting developments of orphan medicinal products in the European Union; therefore an action at Union level remains preferable to uncoordinated measures by the Member States which may result in distortions of competition and barriers to intra-EU trade. The Union should not neglect or disregard the success of the Regulation since its entry into force in 2000, and build on its success driving and ensuring a similar degree of innovation under this Regulation.

_________________

55 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p. 1).

Or. en

Amendment 391

Tilly Metz

Proposal for a regulation

Recital 88

Text proposed by the Commission

Amendment

(88) Regulation (EC) No 141/2000 of the European Parliament and of the Council55 has proved to be successful in boosting developments of orphan medicinal products in the Union for certain conditions; nevertheless, 95% of rare diseases are still without authorised treatment and the treatments available for the 5% are not necessarily transformative or curative1a, therefore an action at Union level remains preferable to uncoordinated measures by the Member States which may result in distortions of competition and barriers to intra-Union trade.

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1a https://research-and-innovation.ec.europa.eu/system/files/2022-02/ec_rtd_he-partnerships-rare-diseases.pdf

55 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p. 1).

Or. en

Amendment 392

Tilly Metz

Proposal for a regulation

Recital 90

Text proposed by the Commission

Amendment

(90) Objective criteria for the orphan designation based on the prevalence of the life-threatening or chronically debilitating condition for which diagnosis, prevention or treatment is sought and the existence of no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Union should be maintained; a prevalence of not more than five affected persons per 10 000 is generally regarded as the appropriate threshold. The orphan designation criterion on the basis of return on investment has been abolished, since it has never been used.

Or. en

Amendment 393

Tilly Metz

Proposal for a regulation

Recital 90 a (new)

Text proposed by the Commission

Amendment

(90 a) The aim of intellectual property and regulatory incentives is to benefit society and promote innovation in areas of public health and unmet medical needs, such as rare diseases. It is of utmost importance that such incentives are not misused or abused, nor pose threats to affordability and patient access to treatments. In particular, the practice of artificially subdividing diseases to create subgroups of patients in order to fall under the orphan medicine prevalence criterion should be prohibited.

Or. en

Amendment 394

Tilly Metz

Proposal for a regulation

Recital 91

Text proposed by the Commission

Amendment

(91) The criterion for orphan designation based on prevalence of a disease may, however, not be appropriate to identify rare diseases in all cases. For example, for conditions which have a short duration and high mortality, measuring the number of people that acquired the disease during a specific time period would better reflect if it is rare within the meaning of this Regulation than measuring the number of people who are ‘affected by it’ in a specific moment of time. With the aim to better identify only those diseases which are rare, the Commission should be empowered to set up specific designation criteria for certain conditions if the one provided for are not appropriate due to scientific reasons and on the basis of a recommendation of the Agency.

deleted

Or. en

Amendment 395

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 91

Text proposed by the Commission

Amendment

deleted

Or. en

Justification

The amendment reflects the proposed changes to Article 63.

Amendment 396

Tilly Metz

Proposal for a regulation

Recital 92

Text proposed by the Commission

Amendment

(92) With the aim to better identify only those diseases which are rare, the Commission should be empowered to supplement the designation criteria by a delegated act if they are not appropriate for certain conditions due to scientific reasons and on the recommendation of the Agency. In addition, the designation criteria require implementing measures to be adopted by the Commission.

deleted

Or. en

Amendment 397

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 92

Text proposed by the Commission

Amendment

deleted

Or. en

Justification

changes to art 63

Amendment 398

Andrey Slabakov

Proposal for a regulation

Recital 92 a (new)

Text proposed by the Commission

Amendment

(92 a) Significant benefit should remain the main determining factor for a therapy's eligibility for orphan status when assessing an orphan medicinal product that meets the incidence threshold.

Or. en

Amendment 399

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Billy Kelleher, Erik Poulsen

Proposal for a regulation

Recital 92 a (new)

Text proposed by the Commission

Amendment

(92 a) Significant benefit should remain the main determining factor for eligibility for orphan status when assessing an orphan medicinal product that meets the incidence threshold.

Or. en

Amendment 400

István Ujhelyi

Proposal for a regulation

Recital 92 a (new)

Text proposed by the Commission

Amendment

(92 a) What constitutes significant benefit in a patient population can change over time, therefore, the concept should remain sufficiently flexible to ensure a future-proof regulatory framework while ensuring predictability through additional guidance.

Or. en

Amendment 401

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Billy Kelleher, Erik Poulsen, Dacian Cioloş

Proposal for a regulation

Recital 92 b (new)

Text proposed by the Commission

Amendment

(92 b) What constitutes significant benefit in a patient population can change over time, therefore, the concept should remain sufficiently flexible to ensure a future-proof regulatory framework while ensuring predictability through additional guidance developped in consultation with patient organisations.

Or. en

Amendment 402

Andrey Slabakov

Proposal for a regulation

Recital 92 b (new)

Text proposed by the Commission

Amendment

(92 b) What constitutes significant benefit in a patient population can change over time. Therefore, the concept must retain sufficient flexibility to ensure a future-proof regulatory framework, while ensuring predictability through additional guidance.

Or. en

Amendment 403

Anders Vistisen

on behalf of the ID Group

Proposal for a regulation

Recital 93

Text proposed by the Commission

Amendment

(93) If a satisfactory method of diagnosis, prevention or treatment of the condition in question has already been authorised in the Union, the orphan medicinal product will have to be of significant benefit to those affected by that condition. In this context, a medicinal product authorised in one Member State is generally deemed as being authorised in the Union. It is not necessary for it to have Union authorisation or to be authorised in all Member States to be considered as a satisfactory method. In addition, commonly used methods of diagnosis, prevention or treatment that are not subject to a marketing authorisation may be considered satisfactory if there is scientific evidence of their efficacy and safety. In certain cases, medicinal products prepared for an individual patient in a pharmacy according to a medical prescription, or according to the prescriptions of a pharmacopoeia and intended to be supplied directly to patients served by the pharmacy, may be considered as satisfactory treatment if they are well known and safe and this is a general practice for the relevant patient population in the Union.

Or. en

Amendment 404

Susana Solís Pérez

Proposal for a regulation

Recital 93

Text proposed by the Commission

Amendment

Or. en

Amendment 405

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Billy Kelleher, Erik Poulsen

Proposal for a regulation

Recital 93

Text proposed by the Commission

Amendment

Or. en

Amendment 406

István Ujhelyi

Proposal for a regulation

Recital 93

Text proposed by the Commission

Amendment

Or. en

Amendment 407

Stelios Kympouropoulos

Proposal for a regulation

Recital 93

Text proposed by the Commission

Amendment

Or. en

Amendment 408

Tilly Metz

Proposal for a regulation

Recital 93

Text proposed by the Commission

Amendment

(93) If a satisfactory method of diagnosis, prevention or treatment of the condition in question has already been authorised in the Union, the orphan medicinal product will have to be of significant benefit to those affected by that condition. In this context, a medicinal product authorised in one Member State is generally deemed as being authorised in the Union. It is not necessary for it to have Union authorisation or to be authorised in all Member States to be considered as a satisfactory method. In addition, commonly used methods of diagnosis, prevention or treatment that are not subject to a marketing authorisation may be considered satisfactory if there is scientific evidence of their efficacy and safety. In certain cases, medicinal products prepared for an individual patient in a pharmacy according to a medical prescription, or according to the prescriptions of a pharmacopoeia and intended to be supplied directly to patients served by the pharmacy, should also be considered as satisfactory treatment if they are well known and safe and this is a general practice for the relevant patient population in the Union.

Or. en

Amendment 409

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 94

Text proposed by the Commission

Amendment

(94) The competence to designate a medicinal product as an orphan medicinal product, in the form of a decision, is accorded to the Agency. This is expected to facilitate and expedite the designation procedure, while ensuring high level of scientific expertise.

(94) The competence to designate a medicinal product as an orphan medicinal product, in the form of a decision, is accorded to the Agency. The designation shall be valid from the day of the adoption of the decision by the Agency. This is expected to facilitate and expedite the designation procedure, while ensuring high level of scientific expertise. The orphan designation may be withdrawn at the request of the orphan medicine sponsor.

Or. en

Justification

to reflect changes in art 66

Amendment 410

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Recital 95

Text proposed by the Commission

Amendment

(95) In order to incite faster authorisation of designated orphan medicinal products, the validity of orphan designation has been set at seven years, with the possibility of extension by the Agency under certain specified conditions; the orphan designation may be withdrawn at the request of the orphan medicine sponsor.

deleted

Or. en

Justification

to reflect changes in art 66

Amendment 411

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Ondřej Knotek, Billy Kelleher, Erik Poulsen, Dacian Cioloş

Proposal for a regulation

Recital 96 a (new)

Text proposed by the Commission

Amendment

(96 a) The regulatory pathway can be uncertain and lack flexibility towards the unique challenges of orphan medicinal products, both in the way developers are required to meet evidentiary standards and in the interactions between developers and the regulatory actors. Therefore, the Agency should develop a dedicated and tailored procedure for early engagement with developers of orphan medicinal products with a view to ensuring that more orphan medicinal product candidates are successful on the regulatory pathway, while managing resources in an efficient way.

Or. en

Amendment 412

Tilly Metz

Proposal for a regulation

Recital 99

Text proposed by the Commission

Amendment

(99) A vast percentage of rare diseases remains without treatment with research and development clustered in the areas where profit is better assured. Therefore, there is a need to target those areas where research is mostly needed and where investments are most risky.

Or. en

Amendment 413

Tomislav Sokol

Proposal for a regulation

Recital 100

Text proposed by the Commission

Amendment

(100) Orphan medicinal products addressing a high unmet medical need prevent, diagnose or treat conditions where either no other method of prevention, diagnosis or treatment exists or, if such method already exists, they would bring exceptional therapeutic advancement. In both cases, the criterion of meaningful reduction in disease morbidity or mortality for the relevant patient population should ensure that only most effective medicinal products are covered. The Agency should draw up scientific guidelines on the category of ‘orphan medicinal products addressing a high unmet medical need’.

deleted

Or. en

Amendment 414

Tilly Metz

Proposal for a regulation

Recital 100

Text proposed by the Commission

Amendment

(100) Orphan medicinal products addressing a high unmet medical need prevent, diagnose or treat conditions where either no other method of prevention, diagnosis or treatment exists or, if such method already exists, they would bring exceptional therapeutic advancement. In both cases, the criterion of substantial reduction in disease morbidity or mortality for the relevant patient population should ensure that only most effective medicinal products are covered. The Agency should draw up scientific guidelines on the category of ‘orphan medicinal products addressing a high unmet medical need’.

Or. en

Amendment 415

Maria Angela Danzì

Proposal for a regulation

Recital 102

Text proposed by the Commission

Amendment

(102) In order to incentivise research and development of orphan medicinal products addressing high unmet needs, to ensure market predictability and to ensure a fair distribution of incentives, a modulation of market exclusivity has been introduced; orphan medicinal products addressing high unmet medical needs benefit from the longest market exclusivity, while market exclusivity for well-established use orphan medicinal products, requiring less investment, is the shortest. In order to ensure increased predictability for developers, the possibility to review the eligibility criteria for market exclusivity after six years after the marketing authorisation has been abolished.

(102) In order to incentivise investment and innovation, research and development of orphan medicinal products for which no other therapies exist or, where they already exist, would be a significant benefit for the target population, a modulation of market exclusivity has been introduced. This modulation is based on the science and principles that guide research, with incentives based on practical obstacles, unique attributes and the development needs of new therapies that address patients' needs; the Regulation provides for four main archetypes of incentives, each addressing unique needs and knowledge gaps in research. Market exclusivity for well-established use orphan medicinal products, requiring less investment, is the shortest. In order to ensure increased predictability for developers, the possibility to review the eligibility criteria for market exclusivity after six years after the marketing authorisation has been abolished.

Or. it

Amendment 416

Andrey Slabakov

Proposal for a regulation

Recital 102

Text proposed by the Commission

Amendment

(102) In order to incentivize investment and innovation, research and development of orphan medicines where either no other treatment exists or, if other treatments already exist, they would constitute a significant benefit to the target population, a modulation of market exclusivity has been introduced. Such modulation is science-driven and informed by the principles that guide research, with incentives based on the concrete barriers, unique attributes, and needs for development of novel therapies that address patient needs; four main incentive archetypes are foreseen by the Regulation, each addressing unique needs and knowledge gaps in research; market exclusivity for well-established use orphan medicines, requiring less investment, is the shortest. In order to ensure increased predictability for developers, the possibility to review the eligibility criteria for market exclusivity six years after the marketing authorisation has been abolished.

Or. en