54 Amendments of Ondřej KNOTEK related to 2023/0131(COD)
Amendment 204 #
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 creatingaiming to create an attractive environment for research, development and production of medicines in the Union, along with a modern framework that makes innovative and established medicinal products available to patients and healthcare systems at affordable prices, while ensuring security of supply and addressing environmental concerns.
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 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 244 #
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 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. The ad-hoc working group on Advanced Therapies should consult the SoHo Coordination Board on borderline cases.
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
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, their caregivers 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.
Amendment 265 #
Proposal for a regulation
Recital 37
Recital 37
(37) Scientific committees like the CAT have been instrumental to ensure expertise and capacity building in an emerging technological field. However, after more than 15 years, advanced therapy medicinal products are now more common. The full integration of their assessment in the work of the CHMP will facilitate the assessment of medicinal products within the same therapeutic class, independent of the technology on which they are based. It will also ensure that all biological medicinal products are assessed by the same committee. The ad-hoc working group on Advanced Therapy Medicinal Products (ATMPs) should provide guidance on the classification of ATMPs and borderline cases to the CHMP.
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 270 #
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, academia, industry, associations representing payers, 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 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 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 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 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 389 #
Proposal for a regulation
Recital 88
Recital 88
(88) Regulation (EC) No 141/2000 of the European Parliament and of the Council55 has proved to be successful in boosting developments of orphan medicinal products in the Union; therefore an action at Union level remains preferable to uncoordinated measures by the Member States which may result in distortions of competition and barriers to intra-Union trade. The Union should not neglect or disregard the success of the Regulation since its entry into force in 2000, and build on its success driving and ensuring a similar degree of innovation under this Regulation. _________________ 55 Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products (OJ L 18, 22.1.2000, p. 1).
Amendment 411 #
Proposal for a regulation
Recital 96 a (new)
Recital 96 a (new)
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 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 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 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 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 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 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 600 #
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 the improvement of quality of life of certain patient's groups, 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 668 #
Proposal for a regulation
Article 15 – paragraph 1 – point d
Article 15 – paragraph 1 – point d
(d) the environmental risk assessment is incomplete or insufficiently substantiated by the applicant or if the risks identified in the environmental risk assessment have not been sufficiently addressed by the applicant, with the exception of medicinal products authorised before 30 october 2005 to avoid restricting patients’ access to existing treatments;
Amendment 766 #
Proposal for a regulation
Article 26 – paragraph 4 – subparagraph 2
Article 26 – paragraph 4 – subparagraph 2
In the preparation of the opinion, the Committee for Medicinal Products for Human Use may request information and data from marketing authorisation holders and from developers and may engage with them in preliminary discussions. The Committee may also make use of health data generated outside of clinical studies, including real world data, where available, taking into account the reliability of those data.
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 1009 #
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 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 1019 #
Proposal for a regulation
Article 60 – paragraph 1 – point a a (new)
Article 60 – paragraph 1 – point a a (new)
(a a) are innovative Advanced Therapy Medicinal Products (ATMPs);
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 1030 #
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 1041 #
Proposal for a regulation
Article 61 – paragraph 1 – subparagraph 1
Article 61 – paragraph 1 – subparagraph 1
For products under development which may fall within the categories of medicinal products to be authorised by the Union listed in Annex I, a developer or a competent authority of the Member States may submit a duly substantiated request to the Agency for a scientific recommendation with a view to determining on scientific grounds whether the concerned product is potentially a ‘medicinal product’, including an ‘advanced therapy medicinal product’ as defined in Article 4 (7), (29) and (30) of [revised Directive 2001/83/EC] and Article 2 of Regulation (EC) No 1394/2007 of the European Parliament and of the Council71 . _________________ 71 Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products and amending Directive 2001/83/EC and Regulation (EC) No 726/2004 (OJ L 324, 10.12.2007, p. 121).
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 1125 #
Proposal for a regulation
Article 70
Article 70
Amendment 1140 #
Proposal for a regulation
Article 70 – paragraph 1 – point b
Article 70 – paragraph 1 – point b
(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or, mortality, severity or long term side effects for the relevant patient population.
Amendment 1152 #
Proposal for a regulation
Article 70 – paragraph 3
Article 70 – paragraph 3
3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and, the authorities or, bodies and stakeholders referred to in Article 162.
Amendment 1158 #
Proposal for a regulation
Article 71 – paragraph 2 – point a
Article 71 – paragraph 2 – point a
(a) nine years for orphan medicinal products other than those referred to in points (b), (c) and (ca);
Amendment 1159 #
Proposal for a regulation
Article 71 – paragraph 2 – point a
Article 71 – paragraph 2 – point a
(a) nine years for orphan medicinal products other than those referred to in points (b), (c) and (ca);
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 1194 #
Proposal for a regulation
Article 71 – paragraph 2 – point b a (new)
Article 71 – paragraph 2 – point b a (new)
(b a) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70 in the paediatric population;
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 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 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 1408 #
Proposal for a regulation
Article 113 – paragraph 2 – subparagraph 1
Article 113 – paragraph 2 – subparagraph 1
The regulatory sandbox shall set out a regulatory framework, including scientific requirements, for the development and, where appropriate clinical trials and placing on the market of a product referred to in paragraph 1 under the conditions set out in this Chapter. The regulatory sandbox may allow targeted derogations to this Regulation, [revised Directive 2001/83/EC] or Regulation (EC) 1394/2007 under the conditions set out in Article 114, while balancing patient safety and innovation in regulatory process.
Amendment 1418 #
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 mayshall engage with them in preliminary discussions.
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.