Activities of Susana SOLÍS PÉREZ related to 2023/0131(COD)
Shadow opinions (1)
OPINION on the proposal for a regulation of the European Parliament and of the Council laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006
Amendments (271)
Amendment 40 #
Proposal for a regulation
Recital 2
Recital 2
(2) The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by creatingsupporting 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.
Amendment 43 #
Proposal for a regulation
Recital 3
Recital 3
(3) Addressing unequal patient access of medicinal products has become a key priority of the Pharmaceutical Strategy for Europe as has been highlighted by the Council and the European Parliament. Member States and the 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.
Amendment 44 #
Proposal for a regulation
Recital 5 a (new)
Recital 5 a (new)
(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.
Amendment 48 #
Proposal for a regulation
Recital 20
Recital 20
(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.
Amendment 52 #
Proposal for a regulation
Recital 30
Recital 30
(30) The Agency should be empowered to give scientific recommendations on whether a product under development, which could potentially fall under the mandatory scope of the centralised procedure, meets the scientific criteria to be a medicinal product such as an advanced therapy medicinal products (ATMPs). 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. For Advanced Therapy Medicinal Products (ATMPs), the European Medicines Agency's (EMA) working group dedicated to ATMPs should seek input from the Substances of Human Origin (SoHO) Coordination Board when dealing with cases that are not clearly defined.
Amendment 53 #
Proposal for a regulation
Recital 30 a (new)
Recital 30 a (new)
(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.
Amendment 57 #
Proposal for a regulation
Recital 36
Recital 36
(36) The expertise of the Committee for Advanced Therapies (CAT), the Committee for Orphan Medicinal Products (COMP), the Paediatric Committee (PDCO) and Committee for Herbal Medicinal Products (HMPC) is retained through working groups, 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.
Amendment 59 #
Proposal for a regulation
Recital 39
Recital 39
(39) To allow for a more informative decision making and for exchange of information and pooling of knowledge on general issues of scientific or technical nature related to the tasks of the Agency regarding medicinal products for human use, in particular to scientific guidelines on unmet medical needs and the design of clinical trials, or other studies and the generation of evidence along the life cycle of medicinal product, the Agency should be able to have recourse to a consultation process of authorities or bodies active along the life cycle of medicinal products. These authorities could be, as appropriate, representatives from Heads of Medicines Agencies, the Clinical Trial Coordination and Advisory Group, the SoHO Coordination Board, the Coordination Group on Health Technology Assessment, Medical Devices Coordination Group, medical devices national competent authorities, national competent authorities for pricing and reimbursement of medicines, national insurance funds or healthcare payers. The Agency should also be able to extend the consultation mechanism to consumers, patients and their caregivers, healthcare professionals, industry, associations representing payers, academia, or other stakeholders, as relevant.
Amendment 68 #
Proposal for a regulation
Recital 53 a (new)
Recital 53 a (new)
(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.
Amendment 69 #
Proposal for a regulation
Recital 60
Recital 60
(60) Regulatory decision-making on the development, authorisation and supervision of medicinal products may be supported by access and analysis of health data, including real world data, where appropriate, i.e. health data generated outside of clinical studies 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.
Amendment 72 #
Proposal for a regulation
Recital 73
Recital 73
(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, adjuvantsmaster 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 and, active substance intermediates, when the intermediate is a chemical active substance by itself or used in conjugation with a biological substance and conjugates, or such as platform technology master files for platform technologies used in the manufacturing process of one or more medicinal products.
Amendment 73 #
Proposal for a regulation
Recital 77
Recital 77
(77) The development of antimicrobial resistance is a growing concern and the pipeline of effective antimicrobials is obstructed due to a market failure; 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. The Union should thus establish a mechanism of AMR Designation to identify medicinal products that can contribute to the fight against AMR and to support the targetting of R&D and access incentives.
Amendment 81 #
Proposal for a regulation
Recital 79
Recital 79
(79) The creation of a voucher rewarding the development of priority antimicrobials through an additional year of regulatory data protection has the capacity, 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.
Amendment 85 #
Proposal for a regulation
Recital 80
Recital 80
(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.
Amendment 95 #
Proposal for a regulation
Recital 87 a (new)
Recital 87 a (new)
(87 a) Recalls the European Parliament's resolution from 10 July 2020 concerning the EU's post-COVID-19 public health strategy, which called for an EU Action Plan specifically targeting rare diseases.
Amendment 98 #
Proposal for a regulation
Recital 92 a (new)
Recital 92 a (new)
(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.
Amendment 99 #
Proposal for a regulation
Recital 92 b (new)
Recital 92 b (new)
(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.
Amendment 100 #
Proposal for a regulation
Recital 93
Recital 93
(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. This provision is applicable solely to medicinal products that are not classified as advanced therapy medicinal products and do not contain complex active substances.
Amendment 101 #
Proposal for a regulation
Recital 96 a (new)
Recital 96 a (new)
(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.
Amendment 105 #
Proposal for a regulation
Recital 102
Recital 102
(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; pioneering orphan medicinal products addressing high unmet medical needareas where there is a total lack of approved treatments 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.
Amendment 108 #
Proposal for a regulation
Recital 104
Recital 104
(104) To maximise the potential benefit of clinical research, continued exploration of new indications should be encouraged. To reward research into and development of new therapeutic indications, an additional period of one year of market exclusivity is provided for a new therapeutic indication (with a maximum of two indications). To continue incentivising innovation, especially in underserved areas, while also allowing generic entry, any subsequent new orphan marketing authorisations granted to the marketing authorisation holder should receive three years of market exclusivity bound to the indication, not the active substance. This will allow generic competition in the first two orphan indications, while allowing for continued research for those patients who could still benefit.
Amendment 109 #
Proposal for a regulation
Recital 105 a (new)
Recital 105 a (new)
(105 a)) One of the overarching goals of this Regulation is to help meet the medical needs of patients with rare diseases, to improve the affordability of orphan medicinal products and the patient access to orphan medicinal products across the Union, and to encourage innovation in areas of need. While other Union programmes and policies also contribute to these goals, people living with a rare disease continue to face common challenges that are many and multifactorial, including delayed diagnoses, lack of available transformative treatments, and difficulties to access treatments where they live, reflecting the fragmentation of the market across the Member States. The European added value to addressing the needs of people living with a rare disease being exceptionally high due to the rarity of patients, experts, data, and resources, it is appropriate for the Commission to develop, to complement this Regulation, a dedicated framework for rare diseases to bridge relevant legislation, policies and programmes, and support national strategies with a view to better meet the unmet needs of people living with rare diseases and their carers. This framework should be needs driven and goals based, and developped in consultation with the Member States and patient organisations as well as, where relevant, other interested parties.
Amendment 112 #
Proposal for a regulation
Recital 126
Recital 126
(126) It is necessary to take measures for the supervision of medicinal products authorised by the Union, and in particular for the intensive supervision of undesirable effects of these medicinal products, and the collection of real-world data within the framework of Union pharmacovigilance activities, so as to ensure the rapid withdrawal from the market of any medicinal product presenting a negative benefit-risk balance under normal conditions of use.
Amendment 113 #
Proposal for a regulation
Recital 129
Recital 129
(129) Scientific and technological progresses in data analytics and data infrastructure are essential for the development, authorisation and supervision of medicinal products. The digital transformation has affected regulatory decision-making, making it more data- driven and multiplying the possibilities to access evidence and real-world data, across the life cycle of a medicinal product. This Regulation recognises the Agency’s experience and capacity to access and analyse data submitted independently from the marketing authorisation applicant or marketing authorisation holder. On this basis, the Agency should take initiative to update the summary of product characteristics in case new efficacy or safety data has an impact on the benefit- risk balance of a medicinal product. In such case, the Agency and the marketing authorisation holder should collaborate to determine the particulars of any such update.
Amendment 123 #
Proposal for a regulation
Recital 134
Recital 134
(134) In the area of medicinal products, a high level of protection of inter alia citizens, consumers, health, as well as legal certainty, a level playing field and fair competition always need to be ensured and existing levels of protection need to be respected. Whenever possible, priority should be given to the use of non-animal approaches.
Amendment 127 #
Proposal for a regulation
Recital 136
Recital 136
(136) Shortages of medicinal products represent a growing threat to public health, with potential serious risks to the health of patients in the Union and impacts on the right of patients to access appropriate medical treatment, including longer delays or interruptions in care or therapy, longer periods of hospitalisation, increased risks of exposure to falsified medicinal products, medication errors, adverse effects resulting from the substitution of unavailable medicinal products with alternative ones, significant psychological distress for patients and increased costs for healthcare systems. The root causes of shortages are multifactorial, with challenges identified along the entire pharmaceutical value chain, from quality and manufacturing problems. In particular, shortages of medicinal products can result from supply chain disruptions and vulnerabilities affecting the supply of key ingredients and components. Therefore, all marketing authorisation holders should have shortage prevention plans in place, to prevent shortages. The Agency should provide guidance to marketing authorisation holders on approaches to streamline the implementation of those plans.
Amendment 132 #
Proposal for a regulation
Recital 138
Recital 138
(138) The national competent authorities and the Agency should be empowered to monitor shortages of medicinal products that are authorised through both national and centralised procedures, based on notifications of marketing authorisation holders. The Agency should be empowered to monitor shortages of medicinal products that are authorised through the centralised procedure, also based on notifications of marketing authorisation holders in a centralised, digitalised and automated system. When critical shortages are identified, both national competent authorities and the Agency should work in a coordinated manner to manage those critical shortages, whether the medicinal product concerned by the critical shortage is covered by a centralised marketing authorisation or a national marketing authorisation. Marketing authorisation holders and other relevant entities must provide the relevant information to inform the monitoring. Wholesale distributors and other persons or legal entities, including patient organisations or health care professionals, may also report a shortage of a given medicinal product marketed in the Member State concerned to the competent authority. The Executive Steering Group on Shortages and Safety of Medicinal Products (‘the Medicines Shortages Steering Group’ (MSSG)) already established within the Agency pursuant to Regulation (EU) 2022/123 of the European Parliament and of the Council56 , should adopt a list of critical shortages of medicinal products and ensure monitoring of those shortages by the Agency. The MSSG should also adopt a list of critical medicinal products authorised in accordance with [revised Directive 2001/83/EC] or this Regulation to ensure monitoring of the supply of those products. The MSSG may provide recommendations on measures to be taken by marketing authorisation holders, the Member States, the Commission and other entities to resolve any critical shortage or to ensure the security of supply of those critical medicinal products to the market. Implementing acts can be adopted by the Commission to ensure that appropriate measures, including the establishment or maintenance of contingency stocks, are taken by marketing authorisation holders, wholesale distributors or other relevant entities. The MSSG should develop in coordination with the Agency a Voluntary Solidarity Mechanism to allow Member States where stocks of important medicines are critically low and where other available options have been exhausted to send out on a voluntary basis a notification, to which other Members States may respond on a voluntary basis to provide temporary relief. This mechanism should leverage existing structures, including the European Shortages Monitoring Platform (‘ESMP’), established by Regulation (EU) 2022/123, and should invite manufacturers and wholesalers to participate where relevant. _________________ 56 Regulation (EU) 2022/123 of the European Parliament and of the Council of 25 January 2022 on a reinforced role for the European Medicines Agency in crisis preparedness and management for medicinal products and medical devices (OJ L 20, 31.1.2022, p. 1).
Amendment 137 #
Proposal for a regulation
Recital 138 a (new)
Recital 138 a (new)
(138 a)To avoid that measures foreseen or taken by a Member State to prevent or mitigate a shortage at national level when responding to the legitimate needs of its citizen increase the risk of shortages in another Member State, the Agency should assess those measures with regards to their potential or actual impact on the availability and security of supply in other Member States and at European level, and inform of its assessment the Member States and the MSSG.
Amendment 138 #
Proposal for a regulation
Recital 138 b (new)
Recital 138 b (new)
(138 b)) One of the aims of this Regulation is to set out a framework for the activities to be deployed by the Member States and the Agency to improve the Union's capacity to react efficiently and in a coordinated manner to support shortage management and security of supply of medicinal products, in particular critical medicinal products, to EU citizens, at all times. Those shortages are a persistent problem that has been increasingly affecting the health and lives of Union citizens for decades and the root causes are multifactorial. Therefore, this Regulation should be a first step towards improving the Union response to that persistent problem. The Commission should subsequently expand that framework to continue addressing the causes of shortages of medicinal products, and better prevent and mitigate their effects.
Amendment 139 #
Proposal for a regulation
Recital 138 c (new)
Recital 138 c (new)
(138 c)To complement this Regulation and as a first step to a more structural, long term approach to reduce Union dependencies for critical medicines and ingredients, particularly for products where there are only a few supplying manufacturers or countries, the Commission should propose by (OP: 24 months after the date of entry into force of this Regulation) a legislative initiative for an EU Critical Medicines Act for supporting the European green, digital manufacturing of key medicines, active pharmaceutical ingredients, and intermediate pharmaceutical ingredients for which the Union is dependent on one country or a limited number of manufacturers
Amendment 140 #
Proposal for a regulation
Recital 138 d (new)
Recital 138 d (new)
(138 d)It is appropriate for the Commission to build upon the Communication addressing medicine shortages in the European Union of 24 October 2023 and the many tools which can be used to promote a coordinated industrial approach, bringing together public and private actors from the European health and industrial ecosystem.
Amendment 141 #
Proposal for a regulation
Recital 145
Recital 145
(145) Experience shows that, in clinical trials with investigational medicinal products containing or consisting of GMOs, including some advanced therapy medicinal products, the procedure to achieve compliance with the requirements of Directives 2001/18/EC and 2009/41/EC as regards the environmental risk assessment and consent by the competent authority of a Member State is complex and can take a significant amount of time.
Amendment 144 #
Proposal for a regulation
Article 1 – paragraph 1
Article 1 – paragraph 1
This Regulation lays down Union procedures for the authorisation, supervision and pharmacovigilance of medicinal products for human use at Union level, establishes rules and procedures at Union and at Member State level relating to the monitoring and management of shortages and critical shortages and security of supply of medicinal products, and lays down the governance provisions of the European Medicines Agency (‘the Agency’) established by Regulation (EC) No 726/2004 which shall carry out the tasks relating to medicinal products for human use that are laid down in this Regulation, Regulation (EU) No 2019/6 and other relevant Union legal acts.
Amendment 146 #
Proposal for a regulation
Article 2 – paragraph 2 – point 4
Article 2 – paragraph 2 – point 4
(4) ‘orphan medicineal product sponsor’ means any legal or natural person, established in the Union, who submitted an application for or has been granted an orphan designation by a decision referred to in Article 64(4);
Amendment 152 #
Proposal for a regulation
Article 2 – paragraph 2 – point 11 a (new)
Article 2 – paragraph 2 – point 11 a (new)
(11 a) ‘supply’ means the total volume of stock of a given medicinal product or medical device that is placed on the market by a marketing authorisation holder or a manufacturer;
Amendment 153 #
Proposal for a regulation
Article 2 – paragraph 2 – point 11 b (new)
Article 2 – paragraph 2 – point 11 b (new)
(11 b) ‘demand’ means the request for a medicinal product or a medical device by a healthcare professional or patient in response to clinical need; the demand is satisfactorily met when the medicinal product or the medical device is acquired in appropriate time and in sufficient quantity to allow continuity of the best care of patients;
Amendment 154 #
Proposal for a regulation
Article 2 – paragraph 2 – point 11 c (new)
Article 2 – paragraph 2 – point 11 c (new)
(11 c) 'adverse reaction' means a response to a medicinal product that is noxious and unintended, and includes medication errors and uses outside of the terms of the marketing authorisation, including the misuse and abuse of the medication product
Amendment 155 #
Proposal for a regulation
Article 2 – paragraph 2 – point 12
Article 2 – paragraph 2 – point 12
(12) ‘shortage’ means a situation in which the supply of a medicinal product that is authorised and placed on the market in a Member State or of a CE- marked medical device does not meet the demand for that medicinal product in that Member State.or medical device at a national level, whatever the cause
Amendment 158 #
Proposal for a regulation
Article 4 – title
Article 4 – title
Member State authorisation of generics of centrally authorisedspecific categories of medicinal products
Amendment 161 #
Proposal for a regulation
Article 4 – paragraph 1 – point a
Article 4 – paragraph 1 – point a
(a) the application for marketing authorisation is submitted in accordance with Article 9, 10, 13 of [revised Directive 2001/83/EC], or for active substances used in fixed dose combination medicinal products that have previously been used in the composition of authorised medicinal products;
Amendment 164 #
Proposal for a regulation
Article 4 – paragraph 2
Article 4 – paragraph 2
Point (b), first subparagraph, shall not apply to those parts of summary of product characteristics and package leaflet referring to indications, posologies, pharmaceutical forms, methods or routes of administration or any other way in which the medicinal product may be used which were still covered by a patent or a supplementary protection certificate for medicinal products at the time when the generic medicinal product was marketed and where the applicant for the generic medicinal product has requested not to include this information in their marketing authorisation.
Amendment 165 #
Proposal for a regulation
Article 5 – paragraph 5
Article 5 – paragraph 5
5. Within 20 days of receipt of an application, the Agency shall check whether all the information and documentation required in accordance with Article 6 have been submitted, that the application does not contain critical deficiencies as defined in guidelines established under paragraph 7 that may prevent the evaluation of the medicinal product and decide whether the application is valid.
Amendment 166 #
Proposal for a regulation
Article 6 – paragraph 1 – subparagraph 2
Article 6 – paragraph 1 – subparagraph 2
The documentation shall include a declaration to the effect that clinical trials carried out outside the Union meet the ethical requirements of Regulation (EU) No 536/2014. Those particulars and documentation shall take account of the unique, Union nature of the authorisation requested and, otherwise than in exceptional cases relating to the application of the law on trademarks pursuant to Regulation (EU) 2017/1001 of the European Parliament and of the Council66 , shall include the use of a single name for the medicinal product. The use of a single name does not exclude: (a) the use of additional qualifiers where necessary to identify different presentations of the medicinal product concerned. (b) The use identified versions of the summary of product characteristics as referred to in article 62 of [Revised Directive] in situations where elements of the product information are still covered by patent law or supplementary protection certificates for medicinal products.the use of additional qualifiers where necessary to identify different presentations of the medicinal product concerned. _________________ 66 Regulation (EU) 2017/1001 of the European Parliament and of the Council of 14 June 2017 on the European Union trade mark (OJ L 154, 16.6.2017, p. 1).
Amendment 167 #
Proposal for a regulation
Article 6 – paragraph 2 – subparagraph 1
Article 6 – paragraph 2 – subparagraph 1
For medicinal products that are likely to offer an exceptional therapeutic advancement in the diagnosis, prevention or treatment, including with regard to the quality of life of a relevant patient population or subpopulation, of a life- threatening, seriously debilitating or serious and chronic condition in the Union, the Agency may, following the advice of the Committee for Medicinal Products for Human Use regarding the maturity of the data related to the development, offer to the applicant a phased review of complete data packages for individual modules of particulars and documentation as referred to in paragraph 1.
Amendment 169 #
Proposal for a regulation
Article 6 – paragraph 4
Article 6 – paragraph 4
4. Where appropriate, the application may include an active substance master file certificate or an application for an active substance master file or any other quality master file certificate or application as referred to in Article 25 and Article 26 of [revised Directive 2001/83/EC].
Amendment 171 #
Proposal for a regulation
Article 6 – paragraph 5 – subparagraph 2
Article 6 – paragraph 5 – subparagraph 2
The marketing authorisation applicant shall not carry out animal tests in case scientifically satisfactory non-animal testing methods are available. When scientifically reliable non-animal testing alternatives are unavailable, applicants utilizing animal testing must adhere to the principles of replacement, reduction, and refinement of animal testing for scientific purposes. This adherence must be in accordance with Directive 2010/63/EU for any animal study carried out to support their application.
Amendment 172 #
Proposal for a regulation
Article 6 – paragraph 7 – subparagraph 2
Article 6 – paragraph 7 – subparagraph 2
If the Committee for Medicinal Products for Human Use accepts the request, the time-limit laid down in Article 6(6), first subparagraph, shall be reduced to 1520 days.
Amendment 173 #
Proposal for a regulation
Article 6 a (new)
Article 6 a (new)
Article6a Parallel application for one or more new indications (1) During the scientific assessment of an initial marketing authorisation application of a medicinal product by the Agency, the applicant may submit to the Agency a separate parallel application for one or more new indications concerning the same medicinal product. (2) The parallel application shall be assessed by the Agency as a marketing authorisation application in accordance with this Regulation, subject to the following: (a) To the extent the Committee for Medicinal Products for Human Use can assess the parallel application within the timeframe applicable to the initial marketing authorisation application, leading to the same outcome for both applications it shall group the applications and issue a single opinion. (b) To the extent the said Committee cannot issue an opinion on the parallel application within the timeframe applicable to the initial marketing authorisation application the parallel application shall be converted to a Type II variation application in accordance with Commission Regulation (EC) No 1234/2008. (c) If the initial marketing authorisation application is withdrawn or receives a negative opinion from the Committee for Medicinal Products for Human Use, the Committee will pursue the assessment of the parallel application as a standalone marketing authorisation application.
Amendment 177 #
Proposal for a regulation
Article 10 – paragraph 2
Article 10 – paragraph 2
2. Where within 90 days of the validation of the marketing authorisation application and during the assessment the Committee for Medicinal Products for Human Use considers that the submitted data are not of sufficient quality or maturity to complete the assessment, the assessment can be terminated. The Committee for Medicinal Products for Human Use shall summarise the deficiencies in writing. On this basis, the Agency shall inform the applicant accordingly and set a reasonable time limit to address the deficiencies. The application shall be suspended until the applicant addresses the deficiencies. If the applicant fails to address those deficiencies within the time limit set by the Agency, the application shall be considered as withdrawnrefused.
Amendment 178 #
Proposal for a regulation
Article 12 – paragraph 4 – point h
Article 12 – paragraph 4 – point h
(h) where appropriate, details of any recommended obligation to conduct any other post-authorisation studies, including post-authorisation treatment optimisation studies, to improve the safe and effective use of the medicinal product;
Amendment 189 #
Proposal for a regulation
Article 16 – paragraph 3 – subparagraph 1
Article 16 – paragraph 3 – subparagraph 1
The Agency shall immediately publish the assessment report on the medicinal product for human use and the reasons for its opinion in favour of granting marketing authorisation, after deletion of any information of a commercially confidential nature and following consultation of patients’ organisations.
Amendment 199 #
Proposal for a regulation
Article 19 – paragraph 3
Article 19 – paragraph 3
3. Conditional marketing authorisations or a new conditional therapeutic indication granted pursuant to this Article shall be subject to specific obligations. Those specific obligations and, where appropriate, the time limit for compliance shall be specified in the conditions to the marketing authorisation. Those specific obligations shall be reviewed annually by the Agency for the first three years after granting the authorisation and every two years thereafter.
Amendment 200 #
Proposal for a regulation
Article 19 – paragraph 4
Article 19 – paragraph 4
4. As part of the specific obligations referred to in paragraph 3, the marketing authorisation holder of a conditional marketing authorisation granted pursuant to this Article shall be required to complete ongoing studies, or to conduct new studies, with a view to confirming that the benefit- risk balance is favourable. The Agency will establish and publish specific deadlines and criteria for meeting these conditions, making them accessible to the public.
Amendment 206 #
Proposal for a regulation
Recital 2
Recital 2
(2) The Pharmaceutical Strategy for Europe marks a turning point with the addition of further key objectives and by creatingsupporting 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.
Amendment 211 #
Proposal for a regulation
Article 24 – paragraph 1 – subparagraph 2 – point f a (new)
Article 24 – paragraph 1 – subparagraph 2 – point f a (new)
(f a) a decision driven by commercial considerations, while safeguarding any information that is of a commercially confidential nature.
Amendment 212 #
Proposal for a regulation
Recital 3
Recital 3
(3) Addressing unequal patient access of medicinal products has become a key priority of the Pharmaceutical Strategy for Europe as has been highlighted by the Council and the European Parliament. Member States and the 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.
Amendment 214 #
Proposal for a regulation
Article 24 – paragraph 4
Article 24 – paragraph 4
4. Where the marketing authorisation holder intends to permanently withdraw the marketing authorisation for a critical medicinal product, the marketing authorisation holder shall, prior to the notification referred to in paragraph 1, offer, on reasonable terms, to transfer the marketing authorisation to a third party that has declared its intention to place that critical medicinal product on the market, or to use the pharmaceutical non-clinical and clinical documentation contained in the file of the medicinal product for the purposes of submitting an application in accordance with Article 14 of [revised Directive 2001/83/EC]. When a marketing authorization is transferred to a new holder, said change shall be reported to the Agency within a 30-day timeframe and include the financial details of the transaction between the transferring parties. This information is then to be made accessible to the public by the Agency.
Amendment 215 #
Proposal for a regulation
Article 25 – paragraph 1 – subparagraph 2 – point a
Article 25 – paragraph 1 – subparagraph 2 – point a
(a) if one of its indications or, posologies, pharmaceutical forms, methods or routes of administration or any other element is protected by a patent or a supplementary protection certificate in one or more Member States;
Amendment 216 #
Proposal for a regulation
Article 25 – paragraph 1 – subparagraph 3
Article 25 – paragraph 1 – subparagraph 3
As soon as the relevant patent or supplementary protection certificate referred to in point (a) expires, the marketing authorisation holder shall withdraw the initial or duplicate marketing authorisation or where appropriate vary the term of the marketing authorisation to include the relevant SmPC information for which the corresponding patent(s) or supplementary protection certificate(s) has(ve) expired.
Amendment 217 #
Proposal for a regulation
Article 26 – paragraph 3
Article 26 – paragraph 3
3. When applying paragraph 1, the Member State shall notify the Agency, which shall make the notification publicly available.
Amendment 218 #
Proposal for a regulation
Recital 5 a (new)
Recital 5 a (new)
(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.
Amendment 219 #
Proposal for a regulation
Article 26 – paragraph 6
Article 26 – paragraph 6
6. The Agency shall keep an up-to- date list of the opinions adopted in accordance with paragraph 4 and shall publish it in a centralized database for compassionate use programs on its website.
Amendment 221 #
Proposal for a regulation
Article 29 – paragraph 1
Article 29 – paragraph 1
Without prejudice to the law on the protection of industrial and commercial property, medicinal products for human use which have been authorised in accordance with this Regulation shall benefit from the periods of regulatory protection set out in Chapter VII of [revised Directive 2001/83/EC]. The granting of periods of regulatory protection shall be published and updated where appropriate by the Agency in a designated registry.
Amendment 222 #
Proposal for a regulation
Article 30 – paragraph 1
Article 30 – paragraph 1
During a public health emergency, the Commission may grant a temporary emergency marketing authorisation (‘TEMA’) or a new temporary emergency therapeutic indication, including when grouped with an extension of an existing marketing authorisation under this Regulation, for medicinal products intended for the treatment, prevention or medical diagnosis of a serious or life- threatening disease or condition which are directly related to the public health emergency, prior to the submission of the complete quality, non-clinical, clinical data and environmental data and information.
Amendment 223 #
Proposal for a regulation
Article 31 – paragraph 1 – introductory part
Article 31 – paragraph 1 – introductory part
A temporary emergency marketing authorisation or a temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, may be granted only after the recognition of a public health emergency at Union level in accordance with Article 23 of Regulation (EU) 2022/2371 of the European Parliament and of the Council67 and where the following requirements are met: _________________ 67 Regulation (EU) 2022/2371 of the European Parliament and of the Council of 23 November 2022 on serious cross- border threats to health and repealing Decision No 1082/2013/EU (OJ L 314, 6.12.2022, p. 26).
Amendment 224 #
Proposal for a regulation
Article 31 – paragraph 1 – point a
Article 31 – paragraph 1 – point a
(a) there is no other satisfactory method of treatment, prevention or diagnosis authorised or sufficiently available in the Union or, if such method is already available, the temporary emergency marketing authorisation of the medicinal productr temporary emergency therapeutic indication of the medicinal product, including when grouped with an extension of the marketing authorisation, will contribute to address the public health emergency;
Amendment 227 #
Proposal for a regulation
Article 32 – paragraph 3
Article 32 – paragraph 3
3. The Agency shall transmit without undue delay to the Commission the scientific opinion and its updates and any recommendations on the temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation.
Amendment 228 #
Proposal for a regulation
Article 33 – paragraph 2
Article 33 – paragraph 2
2. On the basis of the scientific opinion of the Agency referred to in paragraph 1, the Commission shall set specific conditions with respect to the temporary emergency marketing authorisation, in particular the conditions for manufacturing, use, supply and safety monitoring and the compliance with related good manufacturing, and pharmacovigilance practices. If necessary, the conditions may specify the batches of the medicinal product concerned by the temporary emergency marketing authorisation, after consultation with the applicant or marketing authorisation holder.
Amendment 229 #
Proposal for a regulation
Article 33 – paragraph 4
Article 33 – paragraph 4
4. Those specific conditions and, where appropriate, the time limit for compliance shall be specified in the conditions to the marketing authorisation and shall be reviewed annually by the Agency, in consultation with the applicant or marketing authorisation holder.
Amendment 230 #
Proposal for a regulation
Recital 20
Recital 20
(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.
Amendment 230 #
Proposal for a regulation
Article 34 – paragraph 1
Article 34 – paragraph 1
The temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, shall cease to be valid when the Commission terminates the recognition of a public health emergency in accordance with Article 23(2) and (4) of Regulation (EU) 2022/2371.
Amendment 231 #
Proposal for a regulation
Article 34 – paragraph 1 a (new)
Article 34 – paragraph 1 a (new)
When the temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, ceases to be valid in accordance with paragraph 1 of this Article, to avoid any disruption in supply of the medicinal product concerned, the Agency may set a transitional period after consultation with the marketing authorisation holder.
Amendment 232 #
Proposal for a regulation
Article 35 – paragraph 2 a (new)
Article 35 – paragraph 2 a (new)
Where the Commission adopts any such implementing act, the provisions of Article 34, paragraph 1a shall apply.
Amendment 233 #
Proposal for a regulation
Article 36 – paragraph 2
Article 36 – paragraph 2
For the purpose of regulatory data protection, the temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, and any subsequent marketing authorisation, as referred to in subparagraph 1, shall be considered as part of the same global marketing authorisation.
Amendment 243 #
Proposal for a regulation
Recital 30
Recital 30
(30) The Agency should be empowered to give scientific recommendations on whether a product under development, which could potentially fall under the mandatory scope of the centralised procedure, meets the scientific criteria to be a medicinal product such as an advanced therapy medicinal products (ATMPs). 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. For Advanced Therapy Medicinal Products (ATMPs), the European Medicines Agency's (EMA) working group dedicated to ATMPs should seek input from the Substances of Human Origin (SoHO) Coordination Board when dealing with cases that are not clearly defined.
Amendment 248 #
Proposal for a regulation
Recital 30 a (new)
Recital 30 a (new)
(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.
Amendment 261 #
Proposal for a regulation
Recital 36
Recital 36
(36) The expertise of the Committee for Advanced Therapies (CAT), the Committee for Orphan Medicinal Products (COMP), the Paediatric Committee (PDCO) and Committee for Herbal Medicinal Products (HMPC) is retained through working groups, 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.
Amendment 263 #
Proposal for a regulation
Article 40 a (new)
Article 40 a (new)
Article 40a Procedure for AMR designation 1 An antimicrobial medicinal product developed to address priority pathogens shall be granted an AMR designation where the antimicrobial medicinal sponsor can demonstrate that the following requirements are met: a) the product is intended for the diagnosis, prevention or treatment of a pathogen included in the ‘WHO priority pathogens list for R&D of new antibiotics’, or an equivalent list established at Union level; b) there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Union or, where such method exists, that the medicinal product would be of significant benefit to those affected by that condition. 2. The antimicrobial medicinal product sponsor shall submit an application for the designation of the orphan medicinal product to the Agency at any stage of the development of the medicinal product before the application for marketing authorisation referred to in Articles 5 and 6 is submitted. 3. The application of the antimicrobial medicinal sponsor shall be accompanied by the following particulars and documentation: a) name or corporate name and permanent address of the sponsor; b) active substances of the medicinal product; c) proposed condition for which it is intended or the proposed therapeutic indication; d) justification that the criteria laid down in paragraph 1 of this Article are met, and a description of the stage of development, including the expected therapeutic indication. 4. The Agency shall adopt a decision granting or refusing the orphan designation based on the criteria referred to in paragraph 1 of this Article within 90 days of the receipt of a valid application. 5. Designated antimicrobial medicinal products shall be considered as addressing an unmet medical need as referred to in Article 83 of [Proposal for a Directive on the Union code relating to medicinal products for human use].
Amendment 264 #
Proposal for a regulation
Article 40 a (new)
Article 40 a (new)
Article40a Push and pull incentives scheme to boost the development of priority antimicrobials 1. The Commission shall establish a Union push and pull incentives scheme to promote and urgently accelerate the development of novel antimicrobials, as well as promote increased access to existing and newly developed antimicrobials. Member States shall be encouraged to participate in the Union level scheme. 2. The Commission is empowered to adopt delegated acts in accordance with Article 175 to supplement this Regulation by further defining the scheme and its funding, which shall include i.a. the following incentives: (a) research grants under Union funds; (b) milestone prizes for novel antimicrobial developers; (c) voluntary joint procurement with subscription payment mechanisms or market entry rewards that delink or partially delink revenues and sales; 3. The Union push and pull incentives scheme shall be coordinated and managed by the Commission. 4. By ... [one year after the date of entry into force of this Regulation], the Commission shall have developed, and commenced the implementation of the Union push and pull incentives scheme. 5. By ... [7 years after the date of entry into force of this Regulation], the Commission shall present a report to the European Parliament and to the Council reviewing the application of the scheme laid down in this Article.
Amendment 269 #
Proposal for a regulation
Recital 39
Recital 39
(39) To allow for a more informative decision making and for exchange of information and pooling of knowledge on general issues of scientific or technical nature related to the tasks of the Agency regarding medicinal products for human use, in particular to scientific guidelines on unmet medical needs and the design of clinical trials, or other studies and the generation of evidence along the life cycle of medicinal product, the Agency should be able to have recourse to a consultation process of authorities or bodies active along the life cycle of medicinal products. These authorities could be, as appropriate, representatives from Heads of Medicines Agencies, the Clinical Trial Coordination and Advisory Group, the SoHO Coordination Board, the Coordination Group on Health Technology Assessment, Medical Devices Coordination Group, medical devices national competent authorities, national competent authorities for pricing and reimbursement of medicines, national insurance funds or healthcare payers. The Agency should also be able to extend the consultation mechanism to consumers, patients and their caregivers, healthcare professionals, industry, associations representing payers, academia, or other stakeholders, as relevant.
Amendment 272 #
Proposal for a regulation
Recital 39
Recital 39
(39) To allow for a more informative decision making and for exchange of information and pooling of knowledge on general issues of scientific or technical nature related to the tasks of the Agency regarding medicinal products for human use, in particular to scientific guidelines on unmet medical needs and the design of clinical trials, or other studies and the generation of evidence along the life cycle of medicinal product, the Agency should be able to have recourse to a consultation process of authorities or bodies active along the life cycle of medicinal products. These authorities could be, as appropriate, representatives from Heads of Medicines Agencies, the Clinical Trial Coordination and Advisory Group, the SoHO Coordination Board, the Coordination Group on Health Technology Assessment, Medical Devices Coordination Group, medical devices national competent authorities, national competent authorities for pricing and reimbursement of medicines, national insurance funds or healthcare payers. The Agency should also be able to extend the consultation mechanism to consumers, patients and their caregivers, healthcare professionals, industry, associations representing payers, or other stakeholders, as relevant.
Amendment 278 #
Proposal for a regulation
Article 48 – paragraph 1 – subparagraph 1
Article 48 – paragraph 1 – subparagraph 1
An entity not engaged in an economic activity (‘not-for-profit entity’) may submit to the Agency or to a competent authority of the Member State substantive non- clinical or clinical evidence for a new therapeutic indication that is expected to fulfil an unmet medical need.
Amendment 279 #
Proposal for a regulation
Article 48 – paragraph 1 – subparagraph 2
Article 48 – paragraph 1 – subparagraph 2
The Agency may, at the request of a Member State, the Commission, or on its own initiative and on the basis of all available evidence make a scientific evaluation of the benefit-risk of the use of a medicinal product with a new therapeutic indication that concerns an unmet medical need.
Amendment 280 #
Proposal for a regulation
Article 48 – paragraph 3
Article 48 – paragraph 3
3. Article 81(2), point (c) of [revised Directive 2001/83/EC] shall notmay apply for variations under this Article.
Amendment 281 #
Proposal for a regulation
Article 56 – paragraph 1
Article 56 – paragraph 1
Where the Agency concludes that a holder of a marketing authorisation, or a new therapeutic indication, including when grouped with an extension of the marketing authorisation, granted in accordance with Article 19, including a new therapeutic indication granted referred to Article 19, failed to comply with the obligations laid down in the marketing authorisation, the Agency shall inform the Commission accordingly.
Amendment 282 #
Proposal for a regulation
Article 58 – paragraph 1 – subparagraph 2
Article 58 – paragraph 1 – subparagraph 2
Such advice can also be requested for medicinal products referred to in Articles 83 and 84 of [revised Directive 2001/83/EC] and for medicinal products used with an in vitro diagnostic medical device.
Amendment 283 #
Proposal for a regulation
Article 58 – paragraph 4 a (new)
Article 58 – paragraph 4 a (new)
4 a. Disclosed conflicts of interest and the mitigating actions implemented by the concerned individual(s) must be documented in the abridged minutes of the meetings, following the stipulations of Article 147(2).
Amendment 284 #
Proposal for a regulation
Article 58 – paragraph 4 b (new)
Article 58 – paragraph 4 b (new)
4 b. When granting scientific advice, the Agency shall ensure to the greatest extent possible that there is a separation between those responsible for providing scientific advice to a medicine developer and those subsequently involved in evaluating a marketing authorisation application for the same medicinal product. The Agency shall ensure that at least one of the two rapporteurs for a marketing authorisation application should not have taken part in any pre- submission activities concerning the medicinal product. The reasons for any exceptions shall be documented and published with the European Public Assessment Report.
Amendment 285 #
Proposal for a regulation
Recital 46
Recital 46
(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 ofgiving 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).
Amendment 286 #
Proposal for a regulation
Article 60 – paragraph 1 – introductory part
Article 60 – paragraph 1 – introductory part
1. The Agency mayshall offer enhanced scientific and regulatory support, including as applicable consultation with other bodies as referred to in Articles 58 and 59 and accelerated assessment mechanisms, for certain medicinal products that, based on preliminary evidence submitted by the developer fulfil at least one of the following conditions:
Amendment 287 #
Proposal for a regulation
Article 60 – paragraph 1 – point a
Article 60 – paragraph 1 – point a
(a) are likely to address an unmet medical need as referred to in Article 83(1) and 83(2) of [revised Directive 2001/83/EC];
Amendment 290 #
Proposal for a regulation
Article 60 – paragraph 1 – point b
Article 60 – paragraph 1 – point b
(b) are orphan medicinal products and are likely to address a high unmet medical need as referred to in Article 70(1);innovative advanced therapy medicinal products or orphan medicinal products
Amendment 291 #
Proposal for a regulation
Article 60 – paragraph 1 – point c
Article 60 – paragraph 1 – point c
(c) provide an exceptional therapeutic advancement or are expected to be of major interest from the point of view of public health, in particular as regards therapeutic innovation, taking into account the early stage of development, or antimicrobials with any of the characteristics mentioned in Article 40(3).
Amendment 292 #
Proposal for a regulation
Article 60 – paragraph 1 – point c a (new)
Article 60 – paragraph 1 – point c a (new)
(c a) The Agency's working group on advanced therapy medicinal products is tasked with evaluating which products fulfill the criteria of innovative advanced therapy medicinal products as set out in point (b) of this article. The determinations made by the working group should take into account the progressive development inherent to advanced therapy medicinal products.
Amendment 293 #
Proposal for a regulation
Article 61 – paragraph 2 – subparagraph 1
Article 61 – paragraph 2 – subparagraph 1
When forming the recommendation referred to in paragraph 1, the Agency shall consult, where appropriate, relevant advisory or regulatory bodies established in other Union legal acts in related fields. In the case of products which are based on substances of human origin, the Agency shall consult the Substances of Human Origin (SoHO) Coordination Board as established in Regulation (EU) No [reference to be added after adoption cf. COM(2022)338 final], and classify all products that are significantly manipulated or utilized in a non- homologous manner, as either a medicinal product or an advanced therapy medicinal product, whichever is applicable.
Amendment 295 #
Proposal for a regulation
Recital 53 a (new)
Recital 53 a (new)
(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.
Amendment 306 #
Proposal for a regulation
Recital 60
Recital 60
(60) Regulatory decision-making on the development, authorisation and supervision of medicinal products may be supported by access and analysis of health data, including real world data, where appropriate, i.e. health data generated outside of clinical studies 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.
Amendment 318 #
Proposal for a regulation
Recital 73
Recital 73
(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, adjuvantsmaster 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 and, active substance intermediates, when the intermediate is a chemical active substance by itself or used in conjugation with a biological substance and conjugates, or such as platform technology master files for platform technologies used in the manufacturing process of one or more medicinal products.
Amendment 319 #
Proposal for a regulation
Recital 73
Recital 73
(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.
Amendment 323 #
Proposal for a regulation
Article 68 – paragraph 1 – introductory part
Article 68 – paragraph 1 – introductory part
1. The orphan medicine sponsor mayshall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:
Amendment 331 #
Proposal for a regulation
Article 70
Article 70
Amendment 353 #
Proposal for a regulation
Recital 79
Recital 79
(79) The creation of a voucher rewarding the development of priority antimicrobials through an additional year of regulatory data protection has the capacity, 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.
Amendment 353 #
Proposal for a regulation
Article 71 – paragraph 2 – point b
Article 71 – paragraph 2 – point b
(b) twelven years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;where no satisfactory treatment has been approved in the Union for the indication in question.
Amendment 357 #
Proposal for a regulation
Article 71 – paragraph 2 – point b a (new)
Article 71 – paragraph 2 – point b a (new)
(b a) (b a) Ten years for orphan medicinal products where one of the following criteria applies: i. fewer than three orphan medicinal products have been approved in the Union for the indication in question; ii. despite medicinal products being authorised for the indication in question, none has been approved in the Union for the relevant subpopulation that is covered by the therapeutic indication of the new medicinal product; iii. an orphan medicinal product has been approved in the Union for the indication, but the new orphan medical product will represent a new mechanism of action or technology, and will result in significant reduction in disease morbidity or mortality for the relevant patient population, or a major contribution to the quality of life of the relevant population.
Amendment 362 #
Proposal for a regulation
Recital 80
Recital 80
(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.
Amendment 367 #
Proposal for a regulation
Article 71 – paragraph 3
Article 71 – paragraph 3
3. Where a marketing authorisation holder holds more than one orphan marketing authorisations for the same active substance, other than in cases foreseen in Article 72 (2), subparagraph 2, those authorisations shall not benefit from separate market exclusivity periods. The duration of the market exclusivity shall start from the date when the first orphan marketing authorisation was granted in the Union.
Amendment 377 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 1
Article 72 – paragraph 1 – subparagraph 1
Amendment 383 #
Proposal for a regulation
Recital 87 a (new)
Recital 87 a (new)
(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.
Amendment 385 #
Proposal for a regulation
Article 72 – paragraph 2 – subparagraph 1
Article 72 – paragraph 2 – subparagraph 1
The period of market exclusivity shall be prolonged by an additional 12 months for orphan medicinal products referred to in Article 71(2), points (a), (b) and (ba), if at least two years before the end of the exclusivity period, the orphan marketing authorisation holder obtains a marketing authorisation for one or more new therapeutic indications for a different orphan condition.
Amendment 389 #
Proposal for a regulation
Article 72 – paragraph 2 – subparagraph 2
Article 72 – paragraph 2 – subparagraph 2
Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions. Any subsequent marketing authorisation obtained by the marketing authorisation holder for one or more new therapeutic indications for different orphan conditions shall receive a separate market exclusivity of three years, starting from the expiration date of the market exclusivity for the entire product under Article 71, where relevant as extended under paragraphs 1 and 2 of this article.
Amendment 391 #
Proposal for a regulation
Article 72 – paragraph 2 – subparagraph 2 a (new)
Article 72 – paragraph 2 – subparagraph 2 a (new)
The period of market exclusivity shall be prolonged by an additional 12 months for orphan medicinal products referred to in Article 71(2), points (a), (b), and (ba) if the orphan medicinal product is not authorised for the indication in question in any third country at the time of granting the authorisation.
Amendment 398 #
Proposal for a regulation
Article 73 a (new)
Article 73 a (new)
Article73a European Framework for Rare Diseases By (OP: 24 months after the date of entry into force of this Regulation), the Commission shall, following appropriate consultation with the Member States, patient organisations and, where relevant, other interested parties, propose a needs driven and goals based European Framework for Rare Diseases with a view to bridge relevant Union legislation, policies and programmes, and support national strategies to better meet the unmet needs of people living with rare diseases, and their carers.
Amendment 399 #
Proposal for a regulation
Recital 92 a (new)
Recital 92 a (new)
(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.
Amendment 399 #
Proposal for a regulation
Article 75 – paragraph 1 – point b
Article 75 – paragraph 1 – point b
(b) that the disease or condition for which the specific medicinal product or class is intended occurs only in adult populations, unless wthen the product is directed at a molecular target thatre is medical plausibility that the product, due to its mechanism of action and on the basis of existing scientific data, is responsible forcould address an unmet medical need in a different disease or condition in the same therapeutic area in children than the one for which the specific medicinal product or class of medicinal products is intended for in the adult population;
Amendment 400 #
Proposal for a regulation
Article 75 – paragraph 1 – point c
Article 75 – paragraph 1 – point c
(c) that the specific medicinal product is likely to not represent a significant therapeutic benefit over existing methods of diagnosis, prevention or treatments for paediatric patients.
Amendment 401 #
Proposal for a regulation
Recital 92 b (new)
Recital 92 b (new)
(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.
Amendment 401 #
Proposal for a regulation
Article 75 – paragraph 1 a (new)
Article 75 – paragraph 1 a (new)
1 a. The Agency shall, after consultation with the Commission and relevant interested parties, draw up guidelines for the application of this Article.
Amendment 403 #
Proposal for a regulation
Article 81 – paragraph 3
Article 81 – paragraph 3
3. The length of the deferral shall be specified in a decision of the Agency and shall not exceed five yearsbe substantiated by scientific and technical reasoning or by considerations pertaining to public health.
Amendment 404 #
Proposal for a regulation
Recital 93
Recital 93
(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. This provision is applicable solely to medicinal products that are not classified as advanced therapy medicinal products and do not contain complex active substances.
Amendment 405 #
(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. This only applies to medicinal products that are not advanced therapy medicinal products and that do not otherwise contain complex active substances.
Amendment 405 #
Proposal for a regulation
Article 87 – paragraph 2 a (new)
Article 87 – paragraph 2 a (new)
2 a. Within the timelines for adoption of a decision foreseen in Articles 77, 78, 80, 81, 82 and 84, the Agency shall transmit its scientific conclusions to the applicant.
Amendment 406 #
Proposal for a regulation
Article 87 – paragraph 2 b (new)
Article 87 – paragraph 2 b (new)
2 b. Within 20 days following receipt of the scientific conclusions, the applicant may submit to the Agency a written request, citing detailed grounds, for a re- examination. Within 30 days following receipt of a request for re-examination pursuant to paragraph 4, the Agency shall confirm or revise its previous scientific conclusions. The Agency shall be able to question the applicant directly. The applicant may also offer to be questioned. Where the Agency considers it necessary, it may consult the Committee for Medicinal Products for Human Use or the appropriate working parties when re- examining the above mentioned scientific conclusions. If, within the 15-day period referred to in paragraph 4, the applicant does not request re-examination, the scientific conclusions shall become definitive. The Agency shall adopt a decision within a period not exceeding 15 days following the date on which the scientific conclusions have become definitive. This decision shall be communicated to the applicant in writing and shall annex the final scientific conclusions.
Amendment 407 #
Proposal for a regulation
Article 88 – paragraph 1
Article 88 – paragraph 1
Where a paediatric investigation plan, agreed in accordance with the provisions of Article 77, paragraphs 1, 2 and 4, is discontinued, the applicant shall notify the Agency of its intention to discontinue the conduct of the paediatric investigation plan and provide the reasons for such discontinuation no lessas soon as possible or no later than six months beforeafter the discontinuation.
Amendment 408 #
Proposal for a regulation
Article 91 – paragraph 3
Article 91 – paragraph 3
3. When products are authorised in accordance with the provisions of this Regulation, the Commission may update the summary of product characteristics and package leaflet, including dosage accuracy, and may vary the marketing authorisation accordingly.
Amendment 411 #
Proposal for a regulation
Recital 96 a (new)
Recital 96 a (new)
Amendment 413 #
Proposal for a regulation
Article 113 – paragraph 1 – point b a (new)
Article 113 – paragraph 1 – point b a (new)
(b a) The creation of a regulatory sandbox is intended to fulfill the following objectives: a) Enabling competent authorities to offer advice to potential marketing authorization applicants to ensure adherence to this Regulation, or other pertinent EU legislation as applicable. b) Assisting prospective marketing authorization applicants in the experimentation and advancement of innovative medicinal products or product categories. c) Facilitating regulatory learning within a managed setting.
Amendment 416 #
Proposal for a regulation
Article 113 – paragraph 3
Article 113 – paragraph 3
3. The Agency shall monitor the field of emerging medicinal products and may request information and data from marketing authorisation holders, developers, independent experts and researchers, and representatives of healthcare professionals and of patients and may engage with them in preliminary discussions. The Agency may establish a dialogue framework with regulatory bodies both within and outside the Union to aid in its oversight role. Additionally, the Agency is tasked with creating and routinely revising a roster of nascent medicinal or health products that could be considered for a regulatory sandbox environment.
Amendment 419 #
Proposal for a regulation
Recital 102
Recital 102
(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; pioneering orphan medicinal products addressing high unmet medical needareas where there is a total lack of approved treatments 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.
Amendment 419 #
Proposal for a regulation
Article 113 – paragraph 5
Article 113 – paragraph 5
5. The Agency shall be responsible for developing a sandbox plan based on data submitted by developers of eligible products and following appropriate consultations including, where relevant, HTA bodies, patients and their caregivers, clinicians, sponsors, developers and academia . The plan shall set out clinical, scientific and regulatory justification for a sandbox, including the identification of the requirements of this Regulation, [revised Directive 2001/83/EC] and Regulation (EC) 1394/2007 that cannot be complied with and a proposal for alternative or mitigation measures, where appropriate. The plan shall also include a proposed timeline for the duration of the sandbox. Where appropriate, the Agency shall also propose measures in order to mitigate any possible distortion of market conditions as a consequence of establishing a regulatory.
Amendment 428 #
Proposal for a regulation
Recital 104
Recital 104
(104) To maximise the potential benefit of clinical research, continued exploration of new indications should be encouraged. To reward research into and development of new therapeutic indications, an additional period of one year of market exclusivity is provided for a new therapeutic indication (with a maximum of two indications). To continue incentivising innovation, especially in underserved areas, while also allowing generic entry, any subsequent new orphan marketing authorisations granted to the marketing authorisation holder should receive three years of market exclusivity bound to the indication, not the active substance. This will allow generic competition in the first two orphan indications, while allowing for continued research for those patients who could still benefit.
Amendment 433 #
Proposal for a regulation
Recital 105 a (new)
Recital 105 a (new)
(105 a)One of the overarching goals of this Regulation is to help meeting the medical needs of patients with rare diseases, to improve the affordability of orphan medicinal products and the patient access to orphan medicinal products across the Union, and to encourage innovation in areas of need. While other Union programmes and policies also contribute to these goals, people living with a rare disease continue to face common challenges that are many and multifactorial, including delayed diagnoses, lack of available transformative treatments, and difficulties to access treatments where they live, reflecting the fragmentation of the market across the Member States. The European added value to addressing the needs of people living with a rare disease being exceptionally high due to the rarity of patients, experts, data, and resources, it is appropriate for the Commission to develop, to complement this Regulation, a dedicated framework for rare diseases to bridge relevant legislation, policies and programmes, and support national strategies with a view to better meet the unmet needs of people living with rare diseases and their carers. This framework should be needs driven and goals based, and developped in consultation with the Member States and patient organisations as well as, where relevant, other interested parties.
Amendment 438 #
(126) It is necessary to take measures for the supervision of medicinal products authorised by the Union, and in particular for the intensive supervision of undesirable effects of these medicinal products, and the collection of real-world data within the framework of Union pharmacovigilance activities, so as to ensure the rapid withdrawal from the market of any medicinal product presenting a negative benefit-risk balance under normal conditions of use.
Amendment 440 #
Proposal for a regulation
Recital 129
Recital 129
(129) Scientific and technological progresses in data analytics and data infrastructure are essential for the development, authorisation and supervision of medicinal products. The digital transformation has affected regulatory decision-making, making it more data- driven and multiplying the possibilities to access evidence and real-world data, across the life cycle of a medicinal product. This Regulation recognises the Agency’s experience and capacity to access and analyse data submitted independently from the marketing authorisation applicant or marketing authorisation holder. On this basis, the Agency should take initiative to update the summary of product characteristics in case new efficacy or safety data has an impact on the benefit- risk balance of a medicinal product. In such case, the Agency and the marketing authorisation holder should collaborate to determine the particulars of any such update.
Amendment 440 #
Proposal for a regulation
Article 118 – paragraph 1 – subparagraph 1 a (new)
Article 118 – paragraph 1 – subparagraph 1 a (new)
On the basis of the information provided pursuant to article 121(2), point (f), the Agency should monitor and assess any actions foreseen or taken by a Member State to mitigate a shortage at national level with regards to their impact on the availability and supply of medicinal products at European level.
Amendment 450 #
Proposal for a regulation
Article 120 – paragraph 2
Article 120 – paragraph 2
2. For the purposes of Article 118(1), where relevant, upon request from the competent authority concerned as defined in Article 116(1), entities including other marketing authorisation holders as defined in Article 116(1), importers and manufacturers of medicinal products or active substances and relevant suppliers of these, wholesale distributors, stakeholder representative associations or other persons or legal entities that are authorised or entitled to supply medicinal products to the public shall provide any information requested in a timely manner. Commercially sensitive information shall only be available to the relevant authorities.
Amendment 452 #
Proposal for a regulation
Article 121 – paragraph 1 – point b a (new)
Article 121 – paragraph 1 – point b a (new)
(b a) Create a system that is user- friendly and straightforward, allowing patients and patient organizations to report shortages of medicinal products.
Amendment 456 #
Proposal for a regulation
Article 121 – paragraph 2 – point f
Article 121 – paragraph 2 – point f
(f) inform the Agency within one week of any actions foreseen or taken by that Member State to mitigate the shortage at national level.
Amendment 457 #
Proposal for a regulation
Recital 134
Recital 134
(134) In the area of medicinal products, a high level of protection of inter alia citizens, consumers, health, as well as legal certainty, a level playing field and fair competition always need to be ensured and existing levels of protection need to be respected. Whenever possible, priority should be given to the use of non-animal approaches.
Amendment 457 #
Proposal for a regulation
Article 121 – paragraph 2 a (new)
Article 121 – paragraph 2 a (new)
2 a. After the expansion of the ESMP referred to in article 122 (6) and for the purpose of articles 118 (1), and 121 (2), point (a), competent authorities of the Member States shall set up national IT systems which are interoperable with the ESMP and allow for the automated exchange of information with the ESMP while avoiding duplication of reporting.
Amendment 459 #
Proposal for a regulation
Article 121 – paragraph 5 – point d
Article 121 – paragraph 5 – point d
(d) inform the Agency in a timely manner of any actions foreseen or taken by that Member State in accordance with points (b) and (c) and report within one week on any other actions taken to mitigate or resolve the critical shortage in the Member State, as well as the results of these actions.
Amendment 461 #
Proposal for a regulation
Article 122 – paragraph 1 a (new)
Article 122 – paragraph 1 a (new)
1 a. For the purpose of article 118 (1a new) and based on the information provided pursuant to articles 121 (1), point (d), and 121 (2), the Agency shall assess the actions taken or foreseen by a Member State to mitigate a shortage at national level with regards to any potential or actual negative impacts of these actions on the availability and security of supply in another Member State and at European level. The Agency shall inform the Member State in question of its assessment in a timely manner and the MSSG and the Member States potentially or actually impacted through the single point of contact working party referred to in Article 3(6) of Regulation (EU) 2022/123. The Agency shall also inform the Commission of its assessment.
Amendment 463 #
Proposal for a regulation
Article 122 – paragraph 4 – introductory part
Article 122 – paragraph 4 – introductory part
4. For the purposes of fulfilling the tasks referred to in Articles 118(1), 123 and 124, the Agency shall ensure the following, in consultation with patient organisations and the working party referred to in Article 121(1), point (c):
Amendment 470 #
Proposal for a regulation
Recital 136
Recital 136
(136) Shortages of medicinal products represent a growing threat to public health, with potential serious risks to the health of patients in the Union and impacts on the right of patients to access appropriate medical treatment, including longer delays or interruptions in care or therapy, longer periods of hospitalisation, increased risks of exposure to falsified medicinal products, medication errors, adverse effects resulting from the substitution of unavailable medicinal products with alternative ones, significant psychological distress for patients and increased costs for healthcare systems. The root causes of shortages are multifactorial, with challenges identified along the entire pharmaceutical value chain, from quality and manufacturing problems. In particular, shortages of medicinal products can result from supply chain disruptions and vulnerabilities affecting the supply of key ingredients and components. Therefore, all marketing authorisation holders should have shortage prevention plans in place, to prevent shortages. The Agency should provide guidance to marketing authorisation holders on approaches to streamline the implementation of those plans.
Amendment 477 #
Proposal for a regulation
Article 126 – paragraph 2 a (new)
Article 126 – paragraph 2 a (new)
2 a. The Commission shall take the appropriate steps to address any concerns raised by the asseessment of the Agency referred to in Article 122 (1a new).
Amendment 480 #
Proposal for a regulation
Recital 138
Recital 138
(138) The national competent authorities and the Agency should be empowered to monitor shortages of medicinal products that are authorised through both national and centralised procedures, based on notifications of marketing authorisation holders. The Agency should be empowered to monitor shortages of medicinal products that are authorised through the centralised procedure, also based on notifications of marketing authorisation holders in a centralised, digitalised and automated system. When critical shortages are identified, both national competent authorities and the Agency should work in a coordinated manner to manage those critical shortages, whether the medicinal product concerned by the critical shortage is covered by a centralised marketing authorisation or a national marketing authorisation. Marketing authorisation holders and other relevant entities must provide the relevant information to inform the monitoring. Wholesale distributors and other persons or legal entities, including patient organisations or health care professionals, may also report a shortage of a given medicinal product marketed in the Member State concerned to the competent authority. The Executive Steering Group on Shortages and Safety of Medicinal Products (‘the Medicines Shortages Steering Group’ (MSSG)) already established within the Agency pursuant to Regulation (EU) 2022/123 of the European Parliament and of the Council56 , should adopt a list of critical shortages of medicinal products and ensure monitoring of those shortages by the Agency. The MSSG should also adopt a list of critical medicinal products authorised in accordance with [revised Directive 2001/83/EC] or this Regulation to ensure monitoring of the supply of those products. The MSSG may provide recommendations on measures to be taken by marketing authorisation holders, the Member States, the Commission and other entities to resolve any critical shortage or to ensure the security of supply of those critical medicinal products to the market. Implementing acts can be adopted by the Commission to ensure that appropriate measures, including the establishment or maintenance of contingency stocks, are taken by marketing authorisation holders, wholesale distributors or other relevant entities. _________________ 56 Regulation (EU) 2022/123 of the European Parliament and of the Council of 25 January 2022 on a reinforced role for the European Medicines Agency in crisis preparedness and management for medicinal products and medical devices (OJ L 20, 31.1.2022, p. 1).
Amendment 482 #
Proposal for a regulation
Article 129 – paragraph 1
Article 129 – paragraph 1
For the purposes of Article 127(4) and Article 130(2), point (c), and Article 130(4), point (c), where relevant, upon request from the competent authority concerned as defined in Article 116(1), entities including other marketing authorisation holders as defined in Article 116(1), importers and manufacturers of medicinal products or active substances and relevant suppliers of these, wholesale distributors, stakeholder representative associations or other persons or legal entities that are authorised or entitled to supply medicinal products to the public shall provide any information requested in a timely manner. Commercially sensitive information shall only be available to the relevant authorities.
Amendment 484 #
Proposal for a regulation
Recital 138
Recital 138
(138) The national competent authorities should be empowered to monitor shortages of medicinal products that are authorised through both national and centralised procedures, based on notifications of marketing authorisation holders. The Agency should be empowered to monitor shortages of medicinal products that are authorised through the centralised procedure, also based on notifications of marketing authorisation holders. When critical shortages are identified, both national competent authorities and the Agency should work in a coordinated manner to manage those critical shortages, whether the medicinal product concerned by the critical shortage is covered by a centralised marketing authorisation or a national marketing authorisation. Marketing authorisation holders and other relevant entities must provide the relevant information to inform the monitoring. Wholesale distributors and other persons or legal entities, including patient organisations or health care professionals, may also report a shortage of a given medicinal product marketed in the Member State concerned to the competent authority. The Executive Steering Group on Shortages and Safety of Medicinal Products (‘the Medicines Shortages Steering Group’ (MSSG)) already established within the Agency pursuant to Regulation (EU) 2022/123 of the European Parliament and of the Council56 , should adopt a list of critical shortages of medicinal products and ensure monitoring of those shortages by the Agency. The MSSG should also adopt a list of critical medicinal products authorised in accordance with [revised Directive 2001/83/EC] or this Regulation to ensure monitoring of the supply of those products. The MSSG may provide recommendations on measures to be taken by marketing authorisation holders, the Member States, the Commission and other entities to resolve any critical shortage or to ensure the security of supply of those critical medicinal products to the market. Implementing acts can be adopted by the Commission to ensure that appropriate measures, including the establishment or maintenance of contingency stocks, are taken by marketing authorisation holders, wholesale distributors or other relevant entities. The MSSG should develop in coordination with the Agency a Voluntary Solidarity Mechanism to allow Member States where stocks of important medicines are critically low and where other available options have been exhausted to send out on a voluntary basis a notification, to which other Members States may respond on a voluntary basis to provide temporary relief. This mechanism should leverage existing structures, including the European Shortages Monitoring Platform (‘ESMP’), established by Regulation (EU) 2022/123, and should invite manufacturers and wholesalers to participate where relevant. _________________ 56 Regulation (EU) 2022/123 of the European Parliament and of the Council of 25 January 2022 on a reinforced role for the European Medicines Agency in crisis preparedness and management for medicinal products and medical devices (OJ L 20, 31.1.2022, p. 1).
Amendment 487 #
Proposal for a regulation
Article 130 – paragraph 1 – subparagraph 1 – introductory part
Article 130 – paragraph 1 – subparagraph 1 – introductory part
The Agency shall, in collaboration with patient organisations and the working party referred to in Article 121(1), point (c), ensure the following:
Amendment 489 #
Proposal for a regulation
Recital 138 a (new)
Recital 138 a (new)
(138 a)To avoid that measures foreseen or taken by a Member State to prevent or mitigate a shortage at national level when responding to the legitimate needs of its citizen increase the risk of shortages in another Member State, the Agency should assess those measures with regards to their potential or actual impact on the availability and security of supply in other Member States and at European level, and inform of its assessment the Member States and the MSSG.
Amendment 490 #
Proposal for a regulation
Recital 138 b (new)
Recital 138 b (new)
(138 b)One of the aims of this Regulation is to set out a framework for the activities to be deployed by the Member States and the Agency to improve the Union's capacity to react efficiently and in a coordinated manner to support shortage management and security of supply of medicinal products, in particular critical medicinal products, to EU citizens, at all times. Those shortages are a persistent problem that has been increasingly affecting the health and lives of Union citizens for decades and the root causes are multifactorial. Therefore, this Regulation should be a first step towards improving the Union response to that persistent problem. The Commission should subsequently expand that framework to continue addressing the causes of shortages of medicinal products, and better prevent and mitigate their effects.
Amendment 491 #
Proposal for a regulation
Recital 138 c (new)
Recital 138 c (new)
(138 c) To complement this Regulation and as a first step to a more structural, long term approach to reduce Union dependencies for critical medicines and ingredients, particularly for products where there are only a few supplying manufacturers or countries, the Commission should propose by (OP: 24 months after the date of entry into force of this Regulation) a legislative initiative for an EU Critical Medicines Act for supporting the European green, digital manufacturing of key medicines, active pharmaceutical ingredients, and intermediate pharmaceutical ingredients for which the Union is dependent on one country or a limited number of manufacturers.
Amendment 491 #
Proposal for a regulation
Article 130 – paragraph 6 a (new)
Article 130 – paragraph 6 a (new)
6 a. Following the request by a Member State to launch the Voluntary Solidarity Mechanism referred to in article 132(5), the Agency shall provide assistance to the MSSG and may: (a) Confirm that the conditions are met to launch the Voluntary Solidarity Mechanism; (b) Notify the members of the MSSG of the launch of the Voluntary Solidarity Mechanism; (c) Request from the members of the MSSG relevant information within a specific time-limit; (d) Put the issuing country in contact with those Member States able to support them; (e) Organise meetings with the issuing Member States, the donating party and other relevant concerned parties; (f) request the activation of the Union Civil Protection Mechanism to coordinate and logistically support the voluntary transfer of medicines.
Amendment 492 #
Proposal for a regulation
Recital 138 d (new)
Recital 138 d (new)
(138 d)It is appropriate for the Commission to build upon the Communication addressing medicine shortages in the European Union of 24 October 2023 and the many tools which can be used to promote a coordinated industrial approach, bringing together public and private actors from the European health and industrial ecosystem.
Amendment 497 #
Proposal for a regulation
Article 132 – paragraph 1
Article 132 – paragraph 1
1. Following the adoption of the Union list of critical medicinal products pursuant to Article 131(3), in consultation with the Agency and the working party referred to in Article 121(1), point (c), the MSSG may provide recommendations, in accordance with the methods referred to in Article 130(1), point (d), on appropriate security of supply measures to marketing authorisation holders as defined in Article 116(1), the Member States, the Commission or other entities. Such measures may include recommendations on diversification of suppliers and inventory management.
Amendment 505 #
Proposal for a regulation
Article 142 – paragraph 1 – point j a (new)
Article 142 – paragraph 1 – point j a (new)
(j a) an ad hoc working group on Advanced Therapy Medicinal Products;
Amendment 506 #
Proposal for a regulation
Article 142 – paragraph 1 – point j b (new)
Article 142 – paragraph 1 – point j b (new)
(j b) an ad hoc working group on Orphan Medicinal Products;
Amendment 507 #
Proposal for a regulation
Recital 145
Recital 145
(145) Experience shows that, in clinical trials with investigational medicinal products containing or consisting of GMOs, including some advanced therapy medicinal products, the procedure to achieve compliance with the requirements of Directives 2001/18/EC and 2009/41/EC as regards the environmental risk assessment and consent by the competent authority of a Member State is complex and can take a significant amount of time.
Amendment 507 #
Proposal for a regulation
Article 142 – paragraph 1 – point j c (new)
Article 142 – paragraph 1 – point j c (new)
(j c) an ad hoc working group on Paediatric Products;
Amendment 508 #
Proposal for a regulation
Recital 145
Recital 145
(145) Experience shows that, in clinical trials with investigational medicinal products containing or consisting of GMOs, including some advanced therapy medicinal products, the procedure to achieve compliance with the requirements of Directives 2001/18/EC and 2009/41/EC as regards the environmental risk assessment and consent by the competent authority of a Member State is complex and can take a significant amount of time.
Amendment 508 #
Proposal for a regulation
Article 142 – paragraph 1 – point k a (new)
Article 142 – paragraph 1 – point k a (new)
(k a) establish a reservoir of specialists drawn from Member States and key interested parties to serve on ad hoc working groups; members will be chosen for their pertinent expertise corresponding to the varied ad hoc working groups' needs, enabling them to contribute to regulatory support and scientific advisory processes, while ensuring they are not subject to any conflicts of interest.
Amendment 509 #
Proposal for a regulation
Article 146 – paragraph 4 a (new)
Article 146 – paragraph 4 a (new)
4 a. representatives from patients' organizations serving as members or alternate members on scientific committees shall receive remuneration from the Agency's budget, following the financial regulations that apply to the Agency
Amendment 512 #
Proposal for a regulation
Article 150 – paragraph 2 – subparagraph 1
Article 150 – paragraph 2 – subparagraph 1
The Committee for Human Medicinal Products shall establish for the evaluation of specific types of medicinal products or treatments, working parties with scientific expertise in the fields of pharmaceutical quality, methodologies, non-clinical and, clinical evaluations, rare diseases and pediatric medicines, mandating the participation of patient representatives and patient experts.
Amendment 516 #
Proposal for a regulation
Article 152 – paragraph 1 – subparagraph 1
Article 152 – paragraph 1 – subparagraph 1
Where, in accordance with this Regulation, any of the Committees referred to in Article 142 is required to evaluate a medicinal product for human use, it shall appoint one of its members to act as rapporteur, taking into account existing expertise in the Member State. The Committee concerned may appoint a second member to act as co-rapporteur who may represent the patient or healthcare professional community.
Amendment 517 #
Proposal for a regulation
Article 1 – paragraph 1
Article 1 – paragraph 1
This Regulation lays down Union procedures for the authorisation, supervision and pharmacovigilance of medicinal products for human use at Union level, establishes rules and procedures at Union and at Member State level relating to the monitoring and management of shortages and critical shortages and security of supply of medicinal products, and lays down the governance provisions of the European Medicines Agency (‘the Agency’) established by Regulation (EC) No 726/2004 which shall carry out the tasks relating to medicinal products for human use that are laid down in this Regulation, Regulation (EU) No 2019/6 and other relevant Union legal acts.
Amendment 518 #
Proposal for a regulation
Article 153 – paragraph 1
Article 153 – paragraph 1
At the request of the Commission, the Agency shall, in respect of authorised medicinal products for human use, collect any available information on methods that Member States' competent authorities use to determine the added therapeutic value that any new medicinal product for human use provides. The Agency shall jointly develop guidelines for assessing added therapeutic value with patient organizations.
Amendment 519 #
Proposal for a regulation
Article 162 – paragraph 2
Article 162 – paragraph 2
2. The Agency mayshall extend the consultation process to patients, medicine developers, healthcare professionals, industries or other relevant stakeholders, as relevant. It may do so through the creation of a Regulatory Stakeholder Network that streamlines the consultation process.
Amendment 521 #
Proposal for a regulation
Article 2 – paragraph 2 – point 4
Article 2 – paragraph 2 – point 4
(4) ‘orphan medicineal product sponsor’ means any legal or natural person, established in the Union, who submitted an application for or has been granted an orphan designation by a decision referred to in Article 64(4);
Amendment 524 #
Proposal for a regulation
Article 166 – paragraph 2
Article 166 – paragraph 2
2. The Agency may consider and decide upon additional evidence available, independently from the data submitted by the marketing authorisation applicant or marketing authorisation holder. On that basis, the summary of product characteristics shall be updated if the additional evidence has Upon evaluation of the totality of evidence, including the data submitted by the marketing authorisation applicant or marketing authorisation holder, if the Agency considers that a change to the summary of product characteristics may be appropriate due to the impact of such evidence on the benefit-risk balance of the medicinal product, the Agency shall share its proposal with the applicant or marketing authorisation holder without delay. The applicant or marketing authorisation holder shall be provided with all the additional evidence including any relevant study documentation upon which the Agency proposal is based, and impact on the benefit-risk balance of a medicinal productn compliance with applicable requirements set forth under Regulation (EU) 2016/679. The applicant or marketing authorisation holder shall be invited to submit to the Agency their position on the evidence provided and, if necessary, shall be offered the opportunity of an explanation to discuss any proposed update to the summary of product characteristics as may be appropriate. The Agency shall without undue delay inform the applicant or the marketing authorisation holder of its opinion. Where the applicant or marketing authorisation holder disagrees with the Agency’s opinion, the applicant or marketing authorisation holder may request by written notice to the Agency a re-examination of the opinion. In that case, the applicant or marketing authorisation holder shall provide the Agency with the detailed grounds for the request within 30 days after receipt of the opinion. Within 30 days following receipt of the grounds for the request, the Agency shall re-examine its opinion and shall issue a final opinion. The reasons for the conclusion reached shall be annexed to the final opinion. Where the final opinion of the Agency confirms the need for a change to the summary of product characteristics, the summary of product characteristics shall be updated.
Amendment 531 #
Proposal for a regulation
Article 2 – paragraph 2 – point 7 a (new)
Article 2 – paragraph 2 – point 7 a (new)
(7 a) 'adverse reaction' means a response to a medicinal product that is noxious and unintended, and includes medication errors and uses outside of the terms of the marketing authorisation, including the misuse and abuse of the medication product;
Amendment 549 #
Proposal for a regulation
Article 2 – paragraph 2 – point 11 a (new)
Article 2 – paragraph 2 – point 11 a (new)
(11 a) ‘demand’ means the request for a medicinal product or a medical device by a healthcare professional or patient in response to clinical need; the demand is satisfactorily met when the medicinal product or the medical device is acquired in appropriate time and in sufficient quantity to allow continuity of the best care of patients;
Amendment 559 #
Proposal for a regulation
Article 2 – paragraph 2 – point 12
Article 2 – paragraph 2 – point 12
(12) ‘shortage’ means a situation in which the supply of a medicinal product that is authorised and placed on the market in a Member State or of a CE-marked medical device does not meet the demand for that medicinal product in that Member State.or medical device at a national level, whatever the cause
Amendment 564 #
Proposal for a regulation
Article 2 – paragraph 2 – point 12 a (new)
Article 2 – paragraph 2 – point 12 a (new)
(12 a) ‘supply’ means the total volume of stock of a given medicinal product or medical device that is placed on the market by a marketing authorisation holder or a manufacturer;
Amendment 577 #
Proposal for a regulation
Article 4 – title
Article 4 – title
Member State authorisation of generics of centrally authorisedspecific categories of medicinal products
Amendment 583 #
Proposal for a regulation
Article 4 – paragraph 1 – point a
Article 4 – paragraph 1 – point a
(a) the application for marketing authorisation is submitted in accordance with Article 9, 10, 13 of [revised Directive 2001/83/EC], or for active substances used in fixed dose combination medicinal products that have previously been used in the composition of authorised medicinal products;
Amendment 585 #
Proposal for a regulation
Article 4 – paragraph 2
Article 4 – paragraph 2
Point (b), first subparagraph, shall not apply to those parts of summary of product characteristics and package leaflet referring to indications, posologies, pharmaceutical forms, methods or routes of administration or any other way in which the medicinal product may be used which were still covered by a patent or a supplementary protection certificate for medicinal products at the time when the generic medicinal product was marketed and where the applicant for the generic medicinal product has requested not to include this information in their marketing authorisation.
Amendment 589 #
Proposal for a regulation
Article 5 – paragraph 5
Article 5 – paragraph 5
5. Within 20 days of receipt of an application, the Agency shall check whether all the information and documentation required in accordance with Article 6 have been submitted, that the application does not contain critical deficiencies as defined in guidelines established under paragraph 7 that may prevent the evaluation of the medicinal product and decide whether the application is valid.
Amendment 591 #
Proposal for a regulation
Article 6 – paragraph 1 – subparagraph 2
Article 6 – paragraph 1 – subparagraph 2
The documentation shall include a declaration to the effect that clinical trials carried out outside the Union meet the ethical requirements of Regulation (EU) No 536/2014. Those particulars and documentation shall take account of the unique, Union nature of the authorisation requested and, otherwise than in exceptional cases relating to the application of the law on trademarks pursuant to Regulation (EU) 2017/1001 of the European Parliament and of the Council66 , shall include the use of a single name for the medicinal product. The use of a single name does not exclude: (a) the use of additional qualifiers where necessary to identify different presentations of the medicinal product concerned. (b) the use of identified versions of the summary of product characteristics as referred to in article 62 of [Revised Directive] in situations where elements of the product information are still covered by patent law or supplementary protection certificates for medicinal products. (c) the use of additional qualifiers where necessary to identify different presentations of the medicinal product concerned. _________________ 66 Regulation (EU) 2017/1001 of the European Parliament and of the Council of 14 June 2017 on the European Union trade mark (OJ L 154, 16.6.2017, p. 1).
Amendment 599 #
Proposal for a regulation
Article 6 – paragraph 2 – subparagraph 1
Article 6 – paragraph 2 – subparagraph 1
For medicinal products that are likely to offer an exceptional therapeutic advancement in the diagnosis, prevention or treatment, including with regard to the quality of life of a relevant patient population or subpopulation, of a life- threatening, seriously debilitating or serious and chronic condition in the Union, the Agency may, following the advice of the Committee for Medicinal Products for Human Use regarding the maturity of the data related to the development, offer to the applicant a phased review of complete data packages for individual modules of particulars and documentation as referred to in paragraph 1.
Amendment 607 #
Proposal for a regulation
Article 6 – paragraph 4
Article 6 – paragraph 4
4. Where appropriate, the application may include an active substance master file certificate or an application for an active substance master file or any other quality master file certificate or application as referred to in Article 25 and Article 26 of [revised Directive 2001/83/EC].
Amendment 608 #
Proposal for a regulation
Article 6 – paragraph 5 – subparagraph 2
Article 6 – paragraph 5 – subparagraph 2
The marketing authorisation applicant shall not carry out animal tests in case scientifically satisfactory non-animal testing methods are available. When scientifically reliable non-animal testing alternatives are unavailable, applicants utilizing animal testing must adhere to the principles of replacement, reduction, and refinement of animal testing for scientific purposes. This adherence must be in accordance with Directive 2010/63/EU for any animal study carried out to support their application.
Amendment 616 #
Proposal for a regulation
Article 6 – paragraph 7 – subparagraph 2
Article 6 – paragraph 7 – subparagraph 2
If the Committee for Medicinal Products for Human Use accepts the request, the time-limit laid down in Article 6(6), first subparagraph, shall be reduced to 1520 days.
Amendment 621 #
Proposal for a regulation
Article 6 a (new)
Article 6 a (new)
Article6a Parallel application for one or more new indications (1) During the scientific assessment of an initial marketing authorisation application of a medicinal product by the Agency, the applicant may submit to the Agency a separate parallel application for one or more new indications concerning the same medicinal product. (2) The parallel application shall be assessed by the Agency as a marketing authorisation application in accordance with this Regulation, subject to the following: (a) To the extent the Committee for Medicinal Products for Human Use can assess the parallel application within the timeframe applicable to the initial marketing authorisation application, leading to the same outcome for both applications it shall group the applications and issue a single opinion. (b) To the extent the said Committee cannot issue an opinion on the parallel application within the timeframe applicable to the initial marketing authorisation application the parallel application shall be converted to a Type II variation application in accordance with Commission Regulation (EC) No 1234/2008. (c) If the initial marketing authorisation application is withdrawn or receives a negative opinion from the Committee for Medicinal Products for Human Use, the Committee will pursue the assessment of the parallel application as a standalone marketing authorisation application.
Amendment 636 #
Proposal for a regulation
Article 10 – paragraph 2
Article 10 – paragraph 2
2. Where within 90 days of the validation of the marketing authorisation application and during the assessment the Committee for Medicinal Products for Human Use considers that the submitted data are not of sufficient quality or maturity to complete the assessment, the assessment can be terminated. The Committee for Medicinal Products for Human Use shall summarise the deficiencies in writing. On this basis, the Agency shall inform the applicant accordingly and set a reasonable time limit to address the deficiencies. The application shall be suspended until the applicant addresses the deficiencies. If the applicant fails to address those deficiencies within the time limit set by the Agency, the application shall be considered as withdrawnrefused.
Amendment 643 #
Proposal for a regulation
Article 12 – paragraph 4 – point h
Article 12 – paragraph 4 – point h
(h) where appropriate, details of any recommended obligation to conduct any other post-authorisation studies, including post-authorisation treatment optimisation studies, to improve the safe and effective use of the medicinal product;
Amendment 676 #
Proposal for a regulation
Article 16 – paragraph 3 – subparagraph 1
Article 16 – paragraph 3 – subparagraph 1
The Agency shall immediately publish the assessment report on the medicinal product for human use and the reasons for its opinion in favour of granting marketing authorisation, after deletion of any information of a commercially confidential nature and following consultation of patients’ organisations.
Amendment 677 #
Proposal for a regulation
Article 16 – paragraph 3 – subparagraph 1
Article 16 – paragraph 3 – subparagraph 1
The Agency shall immediately publish the assessment report on the medicinal product for human use and the reasons for its opinion in favour of granting marketing authorisation, after deletion of any information of a commercially confidential nature and subsequent consultation of patient organisations.
Amendment 718 #
Proposal for a regulation
Article 19 – paragraph 3
Article 19 – paragraph 3
3. Conditional marketing authorisations or a new conditional therapeutic indication granted pursuant to this Article shall be subject to specific obligations. Those specific obligations and, where appropriate, the time limit for compliance shall be specified in the conditions to the marketing authorisation. Those specific obligations shall be reviewed annually by the Agency for the first three years after granting the authorisation and every two years thereafter.
Amendment 724 #
Proposal for a regulation
Article 19 – paragraph 4
Article 19 – paragraph 4
4. As part of the specific obligations referred to in paragraph 3, the marketing authorisation holder of a conditional marketing authorisation granted pursuant to this Article shall be required to complete ongoing studies, or to conduct new studies, with a view to confirming that the benefit- risk balance is favourable. After consultation with the marketing authorisation holder, the Agency shall lay down deadlines and criteria for the fulfilment of these conditions, and make them publicly available.
Amendment 726 #
Proposal for a regulation
Article 19 – paragraph 4
Article 19 – paragraph 4
4. As part of the specific obligations referred to in paragraph 3, the marketing authorisation holder of a conditional marketing authorisation granted pursuant to this Article shall be required to complete ongoing studies, or to conduct new studies, with a view to confirming that the benefit- risk balance is favourable. The Agency shall establish and publish specific deadlines and criteria for meeting these conditions, making them accessible to the public.
Amendment 744 #
Proposal for a regulation
Article 24 – paragraph 1 – subparagraph 2 – point f a (new)
Article 24 – paragraph 1 – subparagraph 2 – point f a (new)
(f a) a decision driven by commercial considerations, while safeguarding any information that is of a commercially confidential nature.
Amendment 752 #
Proposal for a regulation
Article 24 – paragraph 4
Article 24 – paragraph 4
4. Where the marketing authorisation holder intends to permanently withdraw the marketing authorisation for a critical medicinal product, the marketing authorisation holder shall, prior to the notification referred to in paragraph 1, offer, on reasonable terms, to transfer the marketing authorisation to a third party that has declared its intention to place that critical medicinal product on the market, or to use the pharmaceutical non-clinical and clinical documentation contained in the file of the medicinal product for the purposes of submitting an application in accordance with Article 14 of [revised Directive 2001/83/EC]. When a marketing authorization is transferred to a new holder, such change shall be reported to the Agency within a 30-day timeframe and include the financial details of the transaction between the transferring parties. This information shall be made accessible to the public by the Agency.
Amendment 756 #
Proposal for a regulation
Article 25 – paragraph 1 – subparagraph 2 – point a
Article 25 – paragraph 1 – subparagraph 2 – point a
(a) if one of its indications or, posologies, pharmaceutical forms, methods or routes of administration or any other element is protected by a patent or a supplementary protection certificate in one or more Member States;
Amendment 757 #
Proposal for a regulation
Article 25 – paragraph 1 – subparagraph 3
Article 25 – paragraph 1 – subparagraph 3
As soon as the relevant patent or supplementary protection certificate referred to in point (a) expires, the marketing authorisation holder shall withdraw the initial or duplicate marketing authorisation or where appropriate vary the term of the marketing authorisation to include the relevant SmPC information for which the corresponding patent(s) or supplementary protection certificate(s) has(ve) expired.
Amendment 762 #
Proposal for a regulation
Article 26 – paragraph 3
Article 26 – paragraph 3
3. When applying paragraph 1, the Member State shall notify the Agency, which shall make the notification publicly available.
Amendment 767 #
Proposal for a regulation
Article 26 – paragraph 6
Article 26 – paragraph 6
6. The Agency shall keep an up-to- date list of the opinions adopted in accordance with paragraph 4 and shall publish it in a centralized database for compassionate use programs on its website.
Amendment 770 #
Proposal for a regulation
Article 26 – paragraph 6
Article 26 – paragraph 6
6. The Agency shall keep an up-to- date list of the opinions adopted in accordance with paragraph 4 and shall publish it in a central repository for compassionate use on its website.
Amendment 775 #
Proposal for a regulation
Article 29 – paragraph 1
Article 29 – paragraph 1
Without prejudice to the law on the protection of industrial and commercial property, medicinal products for human use which have been authorised in accordance with this Regulation shall benefit from the periods of regulatory protection set out in Chapter VII of [revised Directive 2001/83/EC]. The granting of periods of regulatory protection shall be published and updated where appropriate by the Agency in a designated registry.
Amendment 782 #
Proposal for a regulation
Article 30 – paragraph 1
Article 30 – paragraph 1
During a public health emergency, the Commission may grant a temporary emergency marketing authorisation (‘TEMA’) or a new temporary emergency therapeutic indication, including when grouped with an extension of an existing marketing authorisation under this Regulation, for medicinal products intended for the treatment, prevention or medical diagnosis of a serious or life- threatening disease or condition which are directly related to the public health emergency, prior to the submission of the complete quality, non-clinical, clinical data and environmental data and information.
Amendment 786 #
Proposal for a regulation
Article 31 – paragraph 1 – introductory part
Article 31 – paragraph 1 – introductory part
A temporary emergency marketing authorisation or a temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, may be granted only after the recognition of a public health emergency at Union level in accordance with Article 23 of Regulation (EU) 2022/2371 of the European Parliament and of the Council67 and where the following requirements are met: _________________ 67 Regulation (EU) 2022/2371 of the European Parliament and of the Council of 23 November 2022 on serious cross-border threats to health and repealing Decision No 1082/2013/EU (OJ L 314, 6.12.2022, p. 26).
Amendment 789 #
Proposal for a regulation
Article 31 – paragraph 1 – point a
Article 31 – paragraph 1 – point a
(a) there is no other satisfactory method of treatment, prevention or diagnosis authorised or sufficiently available in the Union or, if such method is already available, the temporary emergency marketing authorisation of the medicinal productr temporary emergency therapeutic indication of the medicinal product, including when grouped with an extension of the marketing authorisation, will contribute to address the public health emergency;
Amendment 799 #
Proposal for a regulation
Article 32 – paragraph 3
Article 32 – paragraph 3
3. The Agency shall transmit without undue delay to the Commission the scientific opinion and its updates and any recommendations on the temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation.
Amendment 801 #
Proposal for a regulation
Article 33 – paragraph 2
Article 33 – paragraph 2
2. On the basis of the scientific opinion of the Agency referred to in paragraph 1, the Commission shall set specific conditions with respect to the temporary emergency marketing authorisation, in particular the conditions for manufacturing, use, supply and safety monitoring and the compliance with related good manufacturing, and pharmacovigilance practices. If necessary, the conditions may specify the batches of the medicinal product concerned by the temporary emergency marketing authorisation, after consultation with the applicant or marketing authorisation holder.
Amendment 803 #
Proposal for a regulation
Article 33 – paragraph 4
Article 33 – paragraph 4
4. Those specific conditions and, where appropriate, the time limit for compliance shall be specified in the conditions to the marketing authorisation and shall be reviewed annually by the Agency, in consultation with the applicant or marketing authorisation holder.
Amendment 805 #
Proposal for a regulation
Article 34 – paragraph 1
Article 34 – paragraph 1
The temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, shall cease to be valid when the Commission terminates the recognition of a public health emergency in accordance with Article 23(2) and (4) of Regulation (EU) 2022/2371.
Amendment 809 #
Proposal for a regulation
Article 34 – paragraph 1 a (new)
Article 34 – paragraph 1 a (new)
1a. When the temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, ceases to be valid in accordance with paragraph 1 of this Article, the Agency may set a transitional period after consultation with the marketing authorisation holder in order to avoid any disruption in supply of the medicinal product concerned.
Amendment 814 #
Proposal for a regulation
Article 35 – paragraph 2 a (new)
Article 35 – paragraph 2 a (new)
2a. Where the Commission adopts any such implementing act, the provisions of Article 34, paragraph 1a shall apply.
Amendment 817 #
Proposal for a regulation
Article 36 – paragraph 2
Article 36 – paragraph 2
For the purpose of regulatory data protection, the temporary emergency marketing authorisation or temporary emergency therapeutic indication, including when grouped with an extension of the marketing authorisation, and any subsequent marketing authorisation, as referred to in subparagraph 1, shall be considered as part of the same global marketing authorisation.
Amendment 888 #
Proposal for a regulation
Article 40 a (new)
Article 40 a (new)
Article40a Push and pull incentives scheme to boost the development of priority antimicrobials 1. The Commission shall establish a Union push and pull incentives scheme to promote and urgently accelerate the development of novel antimicrobials, as well as promote increased access to existing and newly developed antimicrobials. Member States shall be encouraged to participate in the Union level scheme. 2. The Commission is empowered to adopt delegated acts in accordance with Article 175 to supplement this Regulation by further defining the scheme and its funding, which shall include i.a. the following incentives: (a) research grants under Union funds; (b) milestone prizes for novel antimicrobial developers; (c) voluntary joint procurement with subscription payment mechanisms or market entry rewards that delink or partially delink revenues and sales; 3. The Union push and pull incentives scheme shall be coordinated and managed by the Commission. 4. By ... [one year after the date of entry into force of this Regulation], the Commission shall have developed, and commenced the implementation of the Union push and pull incentives scheme. 5. By ... [7 years after the date of entry into force of this Regulation], the Commission shall present a report to the European Parliament and to the Council reviewing the application of the scheme laid down in this Article.
Amendment 962 #
Proposal for a regulation
Article 48 – paragraph 1 – subparagraph 1
Article 48 – paragraph 1 – subparagraph 1
An entity not engaged in an economic activity (‘not-for-profit entity’) may submit to the Agency or to a competent authority of the Member State substantive non- clinical or clinical evidence for a new therapeutic indication that is expected to fulfil an unmet medical need.
Amendment 963 #
Proposal for a regulation
Article 48 – paragraph 1 – subparagraph 1
Article 48 – paragraph 1 – subparagraph 1
An entity not engaged in an economic activity (‘not-for-profit entity’) may submit to the Agency or to a competent authority of the Member State substantive non- clinical or clinical evidence for a new therapeutic indication that is expected to fulfil an unmet medical need.
Amendment 968 #
Proposal for a regulation
Article 48 – paragraph 1 – subparagraph 2
Article 48 – paragraph 1 – subparagraph 2
The Agency may, at the request of a Member State, the Commission, or on its own initiative and on the basis of all available evidence make a scientific evaluation of the benefit-risk of the use of a medicinal product with a new therapeutic indication that concerns an unmet medical need.
Amendment 970 #
Proposal for a regulation
Article 48 – paragraph 1 – subparagraph 2
Article 48 – paragraph 1 – subparagraph 2
The Agency may, at the request of a Member State, the Commission, or on its own initiative and on the basis of all available evidence make a scientific evaluation of the benefit-risk of the use of a medicinal product with a new therapeutic indication that concerns an unmet medical need.
Amendment 975 #
Proposal for a regulation
Article 48 – paragraph 3
Article 48 – paragraph 3
3. Article 81(2), point (c) of [revised Directive 2001/83/EC] shall notmay apply for variations under this Article.
Amendment 979 #
Proposal for a regulation
Article 56 – paragraph 1
Article 56 – paragraph 1
Where the Agency concludes that a holder of a marketing authorisation, or a new therapeutic indication, including when grouped with an extension of the marketing authorisation, granted in accordance with Article 19, including a new therapeutic indication granted referred to Article 19, failed to comply with the obligations laid down in the marketing authorisation, the Agency shall inform the Commission accordingly.
Amendment 984 #
Proposal for a regulation
Article 58 – paragraph 1 – subparagraph 2
Article 58 – paragraph 1 – subparagraph 2
Such advice can also be requested for medicinal products referred to in Articles 83 and 84 of [revised Directive 2001/83/EC], and for medicinal products used with an in vitro diagnostic medical device.
Amendment 986 #
Proposal for a regulation
Article 58 – paragraph 1 – subparagraph 2
Article 58 – paragraph 1 – subparagraph 2
Such advice can also be requested for medicinal products referred to in Articles 83 and 84 of [revised Directive 2001/83/EC] and for medicinal products used with an in vitro diagnostic medical device.
Amendment 996 #
Proposal for a regulation
Article 58 – paragraph 4 a (new)
Article 58 – paragraph 4 a (new)
4 a. When granting scientific advice, the Agency shall ensure to the greatest extent possible that there is a separation between those responsible for providing scientific advice to a medicine developer and those subsequently involved in evaluating a marketing authorisation application for the same medicinal product. The Agency shall ensure that at least one of the two rapporteurs for a marketing authorisation application should not have taken part in any pre- submission activities concerning the medicinal product. The reasons for any exceptions shall be documented and published with the European Public Assessment Report.
Amendment 1000 #
Proposal for a regulation
Article 58 – paragraph 4 a (new)
Article 58 – paragraph 4 a (new)
4 a. Disclosed conflicts of interest and the mitigating actions implemented by the concerned individual(s) must be documented in the abridged minutes of the meetings, following the stipulations of Article 147(2).
Amendment 1010 #
Proposal for a regulation
Article 60 – paragraph 1 – introductory part
Article 60 – paragraph 1 – introductory part
1. The Agency mayshall offer enhanced scientific and regulatory support, including as applicable consultation with other bodies as referred to in Articles 58 and 59 and accelerated assessment mechanisms, for certain medicinal products that, based on preliminary evidence submitted by the developer fulfil the following conditions:
Amendment 1012 #
Proposal for a regulation
Article 60 – paragraph 1 – introductory part
Article 60 – paragraph 1 – introductory part
1. The Agency may offer enhanced scientific and regulatory support, including as applicable consultation with other bodies as referred to in Articles 58 and 59 and accelerated assessment mechanisms, for certain medicinal products that, based on preliminary evidence submitted by the developer fulfil at least one of the following conditions:
Amendment 1022 #
Proposal for a regulation
Article 60 – paragraph 1 – point b
Article 60 – paragraph 1 – point b
(b) are orphan medicinal products and are likely to address a high unmetlisted on the register of designated orphan medicinal need aproducts referred to in Aarticle 70(1); 67 (1) of this Regulation;
Amendment 1026 #
Proposal for a regulation
Article 60 – paragraph 1 – point b
Article 60 – paragraph 1 – point b
(b) are orphan medicinal products and are likely to address a high unmet medical need as referred to in Article 70(1);innovative advanced therapy medicinal products or orphan medicinal products
Amendment 1032 #
Proposal for a regulation
Article 60 – paragraph 1 – point c
Article 60 – paragraph 1 – point c
(c) provide an exceptional therapeutic advancement or are expected to be of major interest from the point of view of public health, in particular as regards therapeutic innovation, taking into account the early stage of development, or antimicrobials with any of the characteristics mentioned in Article 40(3).
Amendment 1033 #
Proposal for a regulation
Article 60 – paragraph 1 – point c a (new)
Article 60 – paragraph 1 – point c a (new)
(c a) The Agency's working group on advanced therapy medicinal products is tasked with evaluating which products fulfill the criteria of innovative advanced therapy medicinal products as set out in point (b) of this article. The determinations made by the working group shall take into account the progressive development inherent to advanced therapy medicinal products.
Amendment 1043 #
Proposal for a regulation
Article 61 – paragraph 2 – subparagraph 1
Article 61 – paragraph 2 – subparagraph 1
When forming the recommendation referred to in paragraph 1, the Agency shall consult, where appropriate, relevant advisory or regulatory bodies established in other Union legal acts in related fields. In the case of products which are based on substances of human origin, the Agency shall consult the Substances of Human Origin (SoHO) Coordination Board as established in Regulation (EU) No [reference to be added after adoption cf. COM(2022)338 final], and classify all products that are significantly manipulated or utilized in a non- homologous manner, as either a medicinal product or an advanced therapy medicinal product, whichever is applicable.
Amendment 1098 #
Proposal for a regulation
Article 68 – paragraph 1 – introductory part
Article 68 – paragraph 1 – introductory part
1. The orphan medicine sponsor mayshall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:
Amendment 1099 #
Proposal for a regulation
Article 68 – paragraph 1 – introductory part
Article 68 – paragraph 1 – introductory part
1. The orphan medicine sponsor mayshall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:
Amendment 1125 #
Proposal for a regulation
Article 70
Article 70
Amendment 1182 #
Proposal for a regulation
Article 71 – paragraph 2 – point b
Article 71 – paragraph 2 – point b
(b) twelven years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;where no satisfactory treatment has been approved in the Union for the indication in question.
Amendment 1192 #
Proposal for a regulation
Article 71 – paragraph 2 – point b a (new)
Article 71 – paragraph 2 – point b a (new)
Amendment 1209 #
Proposal for a regulation
Article 71 – paragraph 3
Article 71 – paragraph 3
3. Where a marketing authorisation holder holds more than one orphan marketing authorisations for the same active substance, other than in cases foreseen in Article 72, paragraph 2, subparagraph 2, those authorisations shall not benefit from separate market exclusivity periods. The duration of the market exclusivity shall start from the date when the first orphan marketing authorisation was granted in the Union.
Amendment 1222 #
Proposal for a regulation
Article 72 – paragraph 1
Article 72 – paragraph 1
Amendment 1251 #
Proposal for a regulation
Article 72 – paragraph 2 – subparagraph 1
Article 72 – paragraph 2 – subparagraph 1
The period of market exclusivity shall be prolonged by an additional 12 months for orphan medicinal products referred to in Article 71(2), points (a), (b) and (ba), if at least two years before the end of the exclusivity period, the orphan marketing authorisation holder obtains a marketing authorisation for one or more new therapeutic indications for a different orphan condition.
Amendment 1259 #
Proposal for a regulation
Article 72 – paragraph 2 – subparagraph 2 a (new)
Article 72 – paragraph 2 – subparagraph 2 a (new)
Any subsequent marketing authorisation obtained by the marketing authorisation holder for one or more new therapeutic indications for different orphan conditions shall receive a separate market exclusivity of three years, starting from the expiration date of the market exclusivity for the entire product under Article 71, where relevant as extended under paragraphs 1 and 2 of this article.
Amendment 1264 #
Proposal for a regulation
Article 72 – paragraph 2 a (new)
Article 72 – paragraph 2 a (new)
2 a. The period of market exclusivity shall be prolonged by an additional 12 months for orphan medicinal products referred to in Article 71(2), points (a), (b), and (ba) if the orphan medicinal product is not authorised for the indication in question in any third country at the time of granting the authorisation.
Amendment 1282 #
Proposal for a regulation
Article 73 a (new)
Article 73 a (new)
Article 73a European Framework for Rare Diseases By (OP: 24 months after the date of entry into force of this Regulation), the Commission shall, following appropriate consultation with the Member States, patient organisations and, where relevant, other interested parties, propose a needs driven and goals based European Framework for Rare Diseases with a view to bridge relevant Union legislation, policies and programmes, and support national strategies to better meet the unmet needs of people living with rare diseases, and their carers.
Amendment 1288 #
Proposal for a regulation
Article 75 – paragraph 1 – point b
Article 75 – paragraph 1 – point b
(b) that the disease or condition for which the specific medicinal product or class is intended occurs only in adult populations, unless wthen the product is directed at a molecular target thatre is medical plausibility that the product, due to its mechanism of action and on the basis of existing scientific data, is responsible forcould address an unmet medical need in a different disease or condition in the same therapeutic area in children than the one for which the specific medicinal product or class of medicinal products is intended for in the adult population;
Amendment 1293 #
Proposal for a regulation
Article 75 – paragraph 1 – point c
Article 75 – paragraph 1 – point c
(c) that the specific medicinal product is likely to not represent a significant therapeutic benefit over existing methods of diagnosis, prevention or treatments for paediatric patients.
Amendment 1294 #
Proposal for a regulation
Article 75 – paragraph 1 a (new)
Article 75 – paragraph 1 a (new)
1 a. The Agency shall, after consultation with the Commission and relevant interested parties, draw up guidelines for the application of this Article.
Amendment 1302 #
Proposal for a regulation
Article 76 – paragraph 1
Article 76 – paragraph 1
1. A paediatric investigation plan or an application for waiver shall be submitted to the Agency with a request for agreement, except in duly justified cases, before the initiation of confirmatory safety and efficacy clinical studies so as to ensure that a decision on use in the paediatric population of the medicinal product concerned can be given at the time of the marketing authorisation or other application concerned.
Amendment 1321 #
Proposal for a regulation
Article 81 – paragraph 3
Article 81 – paragraph 3
3. The length of the deferral shall be specified in a decision of the Agency and shall not exceed five yearsbe substantiated by scientific and technical reasoning or by considerations pertaining to public health.
Amendment 1341 #
Proposal for a regulation
Article 87 – paragraph 2 a (new)
Article 87 – paragraph 2 a (new)
2 a. Within the timelines for adoption of a decision foreseen in Articles 77, 78, 80, 81, 82 and 84, the Agency shall transmit its scientific conclusions to the applicant.
Amendment 1344 #
Proposal for a regulation
Article 87 – paragraph 2 b (new)
Article 87 – paragraph 2 b (new)
2 b. Within 20 days following receipt of the scientific conclusions, the applicant may submit to the Agency a written request, citing detailed grounds, for a re- examination. Within 30 days following receipt of a request for re-examination pursuant to paragraph 2b, the Agency shall confirm or revise its previous scientific conclusions.The Agency shall be able to question the applicant directly.The applicant may also offer to be questioned.Where the Agency considers it necessary, it may consult the Committee for Medicinal Products for Human Use or the appropriate working parties when re- examining the above mentioned scientific conclusions. If, within the 15-day period referred to in paragraph 4, the applicant does not request re-examination, the scientific conclusions shall become definitive. The Agency shall adopt a decision within a period not exceeding 15 days following the date on which the scientific conclusions have become definitive. This decision shall be communicated to the applicant in writing and shall annex the final scientific conclusions.
Amendment 1354 #
Proposal for a regulation
Article 88 – paragraph 1
Article 88 – paragraph 1
Where a paediatric investigation plan, agreed in accordance with the provisions of Article 77, paragraphs 1, 2 and 4, is discontinued, the applicant shall notify the Agency of its intention to discontinue the conduct of the paediatric investigation plan and provide the reasons for such discontinuation no less than six months before the discontinuation or as soon as possible.
Amendment 1359 #
Proposal for a regulation
Article 91 – paragraph 3
Article 91 – paragraph 3
3. When products are authorised in accordance with the provisions of this Regulation, the Commission may update the summary of product characteristics and package leaflet, including dosage accuracy, and may vary the marketing authorisation accordingly.
Amendment 1368 #
Proposal for a regulation
Article 101 – paragraph 1 – subparagraph 3
Article 101 – paragraph 1 – subparagraph 3
The Eudravigilance database shall contain information on suspected adverse reactions in human beings arising from use of the medicinal product within the terms of the marketing authorisation as well as from uses outside the terms of the marketing authorisation, including medication errors, and on those occurring in the course of post-authorisation studies with the medicinal product or associated with occupational exposure.
Amendment 1390 #
Proposal for a regulation
Article 111 – paragraph 1
Article 111 – paragraph 1
The Agency and the Member States shall cooperate to continuously develop pharmacovigilance systems capable of achieving, including those that record adverse reactions including medication errors, processes and standards for medication safety, and implement digital technologies, with a view to achieve high standards of public health protection for all medicinal products, regardless of the routes of marketing authorisation, including the use of collaborative approaches, to maximise use of resources available within the Union.
Amendment 1399 #
Proposal for a regulation
Article 113 – paragraph 1 – point a
Article 113 – paragraph 1 – point a
(a) it is not possible to adequately develop the medicinal product or category of products in compliance with the requirements applicable to medicinal products due to scientific or regulatory challenges arising from characteristics or methods related to the product;
Amendment 1404 #
Proposal for a regulation
Article 113 – paragraph 1 a (new)
Article 113 – paragraph 1 a (new)
1 a. The creation of a regulatory sandbox is intended to fulfill the following objectives: a) enabling competent authorities to offer advice to potential marketing authorization applicants to ensure adherence to this Regulation, or other pertinent EU legislation as applicable: b) assisting prospective marketing authorization applicants in the experimentation and advancement of innovative medicinal products or product categories; c) facilitating regulatory learning within a managed setting.
Amendment 1413 #
Proposal for a regulation
Article 113 – paragraph 3
Article 113 – paragraph 3
3. The Agency shall monitor the field of emerging medicinal products and may request information and data from marketing authorisation holders, developers, independent experts and researchers, and representatives of healthcare professionals and of patients and may engage with them in preliminary discussions. The Agency may establish a dialogue framework with regulatory bodies both within and outside the Union to aid in its oversight role. Additionally, the Agency is tasked with creating and routinely revising a roster of nascent medicinal or health products that could be considered for a regulatory sandbox environment.
Amendment 1430 #
Proposal for a regulation
Article 113 – paragraph 5
Article 113 – paragraph 5
5. The Agency shall be responsible for developing a sandbox plan based on data submitted by developers of eligible products and following appropriate consultations, including, where relevant, of HTA bodies, patients and their caregivers, clinicians, sponsors, developers and academia. The plan shall set out clinical, scientific and regulatory justification for a sandbox, including the identification of the requirements of this Regulation, [revised Directive 2001/83/EC] and Regulation (EC) 1394/2007 that cannot be complied with and a proposal for alternative or mitigation measures, where appropriate. The plan shall also include a proposed timeline for the duration of the sandbox. Where appropriate, the Agency shall also propose measures in order to mitigate any possible distortion of market conditions as a consequence of establishing a regulatory.
Amendment 1431 #
Proposal for a regulation
Article 113 – paragraph 5
Article 113 – paragraph 5
5. The Agency shall be responsible for developing a sandbox plan based on data submitted by developers of eligible products and following appropriate consultations including, where relevant, HTA bodies, patients and their caregivers, clinicians, sponsors, developers and academia . The plan shall set out clinical, scientific and regulatory justification for a sandbox, including the identification of the requirements of this Regulation, [revised Directive 2001/83/EC] and Regulation (EC) 1394/2007 that cannot be complied with and a proposal for alternative or mitigation measures, where appropriate. The plan shall also include a proposed timeline for the duration of the sandbox. Where appropriate, the Agency shall also propose measures in order to mitigate any possible distortion of market conditions as a consequence of establishing a regulatory.
Amendment 1512 #
Proposal for a regulation
Article 118 – paragraph 1 a (new)
Article 118 – paragraph 1 a (new)
Amendment 1515 #
Proposal for a regulation
Article 118 – paragraph 2
Article 118 – paragraph 2
2. For the purposes of paragraph 1, the competent authority concerned as defined in Article 116(1) may request any additional information from the marketing authorisation holder as defined in Article 116(1). In particular, it may request the marketing authorisation holder to submit a shortage mitigation plan in accordance with Article 119(2), a risk assessment of impact of suspension, cessation or withdrawal in accordance with Article 119(3), or the shortage prevention plan referred to in Article 117, where this has not already been requested following paragraph 9 of article 127. The competent authority concerned mayshall set a deadline for the submission of the information requested.
Amendment 1533 #
Proposal for a regulation
Article 120 – paragraph 2
Article 120 – paragraph 2
2. For the purposes of Article 118(1), where relevant, upon request from the competent authority concerned as defined in Article 116(1), entities including other marketing authorisation holders as defined in Article 116(1), importers and manufacturers of medicinal products or active substances and relevant suppliers of these, wholesale distributors, stakeholder representative associations or other persons or legal entities that are authorised or entitled to supply medicinal products to the public shall provide any information requested in a timely manner. Commercially sensitive information shall only be available to the relevant authorities.
Amendment 1542 #
Proposal for a regulation
Article 121 – paragraph 1 – point b a (new)
Article 121 – paragraph 1 – point b a (new)
(b a) create a system that is user- friendly and straightforward, allowing patients and patient organizations to report shortages of medicinal products.
Amendment 1543 #
Proposal for a regulation
Article 121 – paragraph 1 – point b a (new)
Article 121 – paragraph 1 – point b a (new)
(b a) establish an accessible and easily understandable system for patients and patient organisations to report shortages of medicinal products;
Amendment 1559 #
Proposal for a regulation
Article 121 – paragraph 2 – point f
Article 121 – paragraph 2 – point f
(f) inform the Agency within one week of any actions foreseen or taken by that Member State to mitigate the shortage at national level.
Amendment 1560 #
Proposal for a regulation
Article 121 – paragraph 2 a (new)
Article 121 – paragraph 2 a (new)
2 a. After the expansion of the ESMP referred to in article 122 (6) and for the purpose of articles 118 (1), and 121 (2), point (a), competent authorities of the Member States shall set up national IT systems which are interoperable with the ESMP and allow for the automated exchange of information with the ESMP while avoiding duplication of reporting.
Amendment 1562 #
Proposal for a regulation
Article 121 – paragraph 5 – point d
Article 121 – paragraph 5 – point d
(d) inform the Agency in a timely manner of any actions foreseen or taken by that Member State in accordance with points (b) and (c) and report within one week on any other actions taken to mitigate or resolve the critical shortage in the Member State, as well as the results of these actions.
Amendment 1567 #
Proposal for a regulation
Article 122 – paragraph 1
Article 122 – paragraph 1
1. For the purposes of Article 118(1) and 118(1a)(new), the Agency may request additional information from the competent authority of the Member State, through the working party referred to in Article 121(1), point (c). The Agency may set a deadline for the submission of the information requested.
Amendment 1569 #
Proposal for a regulation
Article 122 – paragraph 1 a (new)
Article 122 – paragraph 1 a (new)
1 a. For the purpose of Article 118 (1a) (new) and based on the information provided pursuant to articles 121 (1), point (d), and 121 (2), the Agency shall assess the actions taken or foreseen by a Member State to mitigate a shortage at national level with regards to any potential or actual negative impacts of these actions on the availability and security of supply in another Member State and at European level. The Agency shall inform the Member State in question of its assessment in a timely manner and the MSSG and the Member States potentially or actually impacted through the single point of contact working party referred to in Article 3(6) of Regulation (EU) 2022/123. The Agency shall also inform the Commission of its assessment.
Amendment 1575 #
Proposal for a regulation
Article 122 – paragraph 4 – introductory part
Article 122 – paragraph 4 – introductory part
4. For the purposes of fulfilling the tasks referred to in Articles 118(1), 123 and 124, the Agency shall ensure the following, in consultation with patients' organisations and the working party referred to in Article 121(1), point (c):
Amendment 1631 #
Proposal for a regulation
Article 126 – paragraph 2 a (new)
Article 126 – paragraph 2 a (new)
2 a. The Commission shall take the appropriate steps to address any concerns raised by the assessment of the Agency referred to in Article 122 (1a) (new).
Amendment 1647 #
Proposal for a regulation
Article 129 – paragraph 1
Article 129 – paragraph 1
For the purposes of Article 127(4) and Article 130(2), point (c), and Article 130(4), point (c), where relevant, upon request from the competent authority concerned as defined in Article 116(1), entities including other marketing authorisation holders as defined in Article 116(1), importers and manufacturers of medicinal products or active substances and relevant suppliers of these, wholesale distributors, stakeholder representative associations or other persons or legal entities that are authorised or entitled to supply medicinal products to the public shall provide any information requested in a timely manner. Commercially sensitive information shall only be available to the relevant authorities.
Amendment 1654 #
Proposal for a regulation
Article 130 – paragraph 1 – subparagraph 1 – introductory part
Article 130 – paragraph 1 – subparagraph 1 – introductory part
The Agency shall, in collaboration with patient organisations and the working party referred to in Article 121(1), point (c), ensure the following:
Amendment 1655 #
Proposal for a regulation
Article 130 – paragraph 1 – subparagraph 1 – introductory part
Article 130 – paragraph 1 – subparagraph 1 – introductory part
The Agency shall, in collaboration with patient organisations and the working party referred to in Article 121(1), point (c), ensure the following:
Amendment 1670 #
Proposal for a regulation
Article 130 – paragraph 6 a (new)
Article 130 – paragraph 6 a (new)
Amendment 1687 #
Proposal for a regulation
Article 132 – paragraph 1
Article 132 – paragraph 1
1. Following the adoption of the Union list of critical medicinal products pursuant to Article 131(3), in consultation with the Agency and the working party referred to in Article 121(1), point (c), the MSSG may provide recommendations, in accordance with the methods referred to in Article 130(1), point (d), on appropriate security of supply measures to marketing authorisation holders as defined in Article 116(1), the Member States, the Commission or other entities. Such measures may include recommendations on diversification of suppliers and, inventory management, on pricing mechanisms, tender practices and cost-containment measures, and on the use of regulatory flexibilities.
Amendment 1691 #
Proposal for a regulation
Article 132 – paragraph 1
Article 132 – paragraph 1
1. Following the adoption of the Union list of critical medicinal products pursuant to Article 131(3), in consultation with the Agency and the working party referred to in Article 121(1), point (c), the MSSG may provide recommendations, in accordance with the methods referred to in Article 130(1), point (d), on appropriate security of supply measures to marketing authorisation holders as defined in Article 116(1), the Member States, the Commission or other entities. Such measures may include recommendations on diversification of suppliers and inventory management.
Amendment 1693 #
Proposal for a regulation
Article 132 – paragraph 1 b (new)
Article 132 – paragraph 1 b (new)
1 b. The MSSG shall set up a Voluntary Solidarity Mechanism to allow Member States to request assistance in obtaining stocks of a medicine during critical shortages. The MSSG shall specify the procedures and criteria to launch the Voluntary Solidarity Mechanism in consultation with the Member States, the Agency and the Commission.