120 Amendments of Pernille WEISS related to 2023/0131(COD)
Amendment 58 #
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. Information regarding the composition and work of the committees and working groups should be publically available.
Amendment 60 #
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, their caregivers, healthcare professionals, acdemia, industry, associations representing payers, or other stakeholders, as relevant.
Amendment 147 #
Proposal for a regulation
Article 2 – paragraph 2 – point 7
Article 2 – paragraph 2 – point 7
(7) ‘significant benefit’ means a clinically relevant advantage or a major contribution to patient care of an orphan medicinal product if such an advantage or contribution benefits a substantial part of the target population;
Amendment 151 #
Proposal for a regulation
Article 2 – paragraph 2 – point 10
Article 2 – paragraph 2 – point 10
(10) ‘regulatory sandbox’ means a regulatory framework during which it is possible to develop, validate and test in a controlled environment innovative or adapted regulatory solutions that facilitate the development and authorisation of innovative products which are likely to fall in the scope of this Regulation, pursuant to a specific plan and for a limited time under regulatory supervision.
Amendment 184 #
Proposal for a regulation
Article 15 – paragraph 1 – point d
Article 15 – paragraph 1 – point d
Amendment 188 #
Proposal for a regulation
Article 15 – paragraph 1 a (new)
Article 15 – paragraph 1 a (new)
1 a. The marketing authorisation may furthermore be refused if, after verification of the particulars and documentation submitted in accordance with Article 6, the view is taken that 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.
Amendment 218 #
Proposal for a regulation
Article 26 – paragraph 4 – subparagraph 1
Article 26 – paragraph 4 – subparagraph 1
When compassionate use is envisaged by a Member State, or by the applicant or sponsor, the Committee for Medicinal Products for Human Use, after consulting the manufacturer or the applicant, may adopt opinions on the conditions for use, the conditions for distribution and the patients targeted. The opinions shall be updated where necessary.
Amendment 220 #
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 on its website in an accessible, searchable data format.
Amendment 242 #
Proposal for a regulation
Article 40 – paragraph 2
Article 40 – paragraph 2
2. The voucher referred to in paragraph 1 shall give the right to its holder to an additional 12 monthsperiod of data protection for one authorised medicinal product as set out according to paragraph 3 of this Article.
Amendment 244 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – introductory part
Article 40 – paragraph 3 – subparagraph 1 – introductory part
An antimicrobial shall be considered ‘priority antimicrobial’ if preclinical and clinical data underpin a significant clinical benefit with respect to antimicrobial resistance and it has at least one of the following characteristics:.
Amendment 247 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – point a
Article 40 – paragraph 3 – subparagraph 1 – point a
Amendment 251 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – point b
Article 40 – paragraph 3 – subparagraph 1 – point b
Amendment 252 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – point c
Article 40 – paragraph 3 – subparagraph 1 – point c
Amendment 255 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 2
Article 40 – paragraph 3 – subparagraph 2
In the scientific assessment of the criteriapriority antimicrobials as referred to in the first subparagraph, and in the cathe Agency shall develop a set of antibiotics, the Agency shallcriteria takeing into account the ‘WHO priority pathogens list for R&D of new antibiotics’, or an equivalent list established at Union level; health system benefits, including with regard to safety and ease of administration; and pharmacological benefits, including novelty of the product.
Amendment 258 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 2 a (new)
Article 40 – paragraph 3 – subparagraph 2 a (new)
The Agency shall develop a weighting of each criteria and a corresponding scoring system allowing priority antimicrobials to be designated one of three categories of vouchers according to its expected impact on combatting antimicrobial resistance. The agency shall assign each voucher category a corresponding financial value which will be payed to the applicant following the auction process set out in Article 41.
Amendment 261 #
Proposal for a regulation
Article 40 – paragraph 4 – subparagraph 1 – point b
Article 40 – paragraph 4 – subparagraph 1 – point b
(b) provide information on all direct EU financial support received for research related to the development of the priority antimicrobial.
Amendment 262 #
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. Information regarding the composition and work of the committees and working groups should be publically available.
Amendment 264 #
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 267 #
Proposal for a regulation
Article 41 – paragraph 1 – subparagraph 1
Article 41 – paragraph 1 – subparagraph 1
A voucher may be used to extend the data protection fgranted by the Commission shall be subject to a public auction by the Agency. The financial value to be payed to the applicant by the winner of the auction is predetermined by the voucher category a period of 12 months of the priority antimicrobial or anothers set out in Article 40. Thus, those wishing to participate in the auction shall bid for the shortest data protection extension for which they are willing to pay the financial amount of the voucher category to the applicant. The voucher may be used to extend the data protection of a medicinal product authorised in accordance with this Regulation ofor the same or different marketing authorisation holderperiod of the winning bid.
Amendment 268 #
Proposal for a regulation
Article 41 – paragraph 1 – subparagraph 2
Article 41 – paragraph 1 – subparagraph 2
A voucher shall only be used once and in relation to a single centrally authorised medicinal product and only if that product is within its first fourleast two years of regulatory data protection is remaining for that product.
Amendment 269 #
Proposal for a regulation
Article 41 – paragraph 3
Article 41 – paragraph 3
3. AWhen an auction is completed, and the voucher mayhas been transferred to another marketing authorisation holder andthe auction winner, the voucher shall not be transferred further.
Amendment 270 #
Proposal for a regulation
Article 41 – paragraph 4
Article 41 – paragraph 4
4. AThe Agency shall market ing authorisation holder to whom a voucher is transferred shall notify the Agency of the transfer within 30 days, stating the value of the transaction between the two parties. The Agency shall make this informationformation regarding the voucher category value and the lenght of data exclusivity extension of the winning bid publicly available.
Amendment 271 #
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, their caregivers, healthcare professionals, acdemia, industry, associations representing payers, or other stakeholders, as relevant.
Amendment 277 #
Proposal for a regulation
Article 43 – paragraph 1
Article 43 – paragraph 1
This Chapter shall apply untilbe subject to a review by the Commission by [Note to OP: insert the date of 15 years after the date of entry into force of this Regulation] or until the date when the Commission has granted a total of 10 vouchers in accordance with this Chapter, whichever date is the earliest.
Amendment 285 #
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 and new indications of medicinal products, that, based on preliminary evidence submitted by the developer fulfil one or more of the following conditions:
Amendment 289 #
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)or advanced therapy medicinal products;
Amendment 296 #
Proposal for a regulation
Recital 53 a (new)
Recital 53 a (new)
(53 a) Several care pathways should be explored to make therapies available in all Member States, including by advancing provisions for access to cross border care such as Directive 2011/24/EU and Regulation (EC) No 883/2004. This is particularly important for the advanced therapy medicinal products (ATMPs), as their unique characteristics result in significant infrastructural complexities and system barriers, which can substantially limit their continuously supply.
Amendment 297 #
Proposal for a regulation
Article 63 – paragraph 2
Article 63 – paragraph 2
Amendment 298 #
Proposal for a regulation
Article 63 – paragraph 3
Article 63 – paragraph 3
3. The Commission shall adopt the necessary provisions for implementing this Article by means of implementing acts in accordance with the procedure laid down in Article 173(2) in order to further specify the requirements referred to in paragraph 1.
Amendment 307 #
Proposal for a regulation
Article 64 – paragraph 4 – subparagraph 1
Article 64 – paragraph 4 – subparagraph 1
The Agency shall adopt a decision granting or refusing the orphan designation based on the criteria referred to in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) within 90 days of the receipt of a valid application. The application is considered valid if it includes all the particulars and documentation referred to in paragraph 2.
Amendment 329 #
Proposal for a regulation
Article 68 – paragraph 2 – subparagraph 1 (new)
Article 68 – paragraph 2 – subparagraph 1 (new)
For the purpose of paragraph 2, the definitions set out in Article 58a paragraph 1 of [revised Directive 2001/83/EC] shall apply.
Amendment 332 #
Proposal for a regulation
Article 70 – title
Article 70 – title
Breakthrough Designated Orphan medicinal products addressing a high unmet medical need
Amendment 333 #
Proposal for a regulation
Article 70 – paragraph 1 – introductory part
Article 70 – paragraph 1 – introductory part
1. An orphan medicinal product shall be considered as addressing a high unmetdesignated as a breakthrough orphan medicinal needproduct where it fulfilscan be demonstrated at the moment of designation that the one of the following requirements are met:
Amendment 334 #
Proposal for a regulation
Article 70 – paragraph 1 – point a
Article 70 – paragraph 1 – point a
(a) there is no medicinal product authorised in the Union for such condition orwhere, despite medicinal products being authorised for such condition in the Union, the applicant demonstrates that the orphan medicinal product, in addition to having a significant benefit, will bring exceptional therapeutic advancementexists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Union, or;
Amendment 337 #
Proposal for a regulation
Article 70 – paragraph 1 – point b
Article 70 – paragraph 1 – point b
(b) twhere, despite medicinal products being authorised for such condition in the Union, the applicant demonstrates that the orphan medicinal product uses a new and unique mechanism of action and the use of the orphan medicinal product results in a meaningful prevention of or reduction in disease morbidity or mortality or a major contribution to patient care for the relevant patient population.
Amendment 340 #
Proposal for a regulation
Article 70 – paragraph 2
Article 70 – paragraph 2
Amendment 341 #
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, as well as the stakeholders such as representatives of patients' organisations in the relevant disease areas, healthcare professionals, orphan medicinal product sponsors, representatives of pharmaceutical industry and other relevant stakeholders referred to in Article 162.
Amendment 346 #
Proposal for a regulation
Article 71 – paragraph 2 – point a
Article 71 – paragraph 2 – point a
(a) nineten years for orphan medicinal products other than those referred to in points (b) and (c);
Amendment 355 #
Proposal for a regulation
Article 71 – paragraph 2 – point b
Article 71 – paragraph 2 – point b
(b) twelven years for breakthrough orphan medicinal products addressing a high unmet medical need as referred to in Article 70;
Amendment 363 #
Proposal for a regulation
Article 71 – paragraph 2 – point c
Article 71 – paragraph 2 – point c
(c) fivesix years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].
Amendment 365 #
Proposal for a regulation
Article 71 – paragraph 2 – point c a (new)
Article 71 – paragraph 2 – point c a (new)
(c a) twelve years for orphan medicinal products addressing requirements set in Article 70, paragraph 1 point (a) and (b) in the paediatric population.
Amendment 368 #
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, 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, provided that a market exclusivity period was still in force when the application for marketing authorisation was granted in the Unionsubmitted.
Amendment 376 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 1
Article 72 – paragraph 1 – subparagraph 1
Amendment 382 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 2
Article 72 – paragraph 1 – subparagraph 2
Amendment 386 #
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 124 months for orphan medicinal products referred to in Article 71(2), points (a) and (b), 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 390 #
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.
Amendment 393 #
Proposal for a regulation
Article 72 – paragraph 3
Article 72 – paragraph 3
3. The orphan medicinal products which benefit from the prolongatUpon request from the applicant, the periond of market exclusivity referred to in the paragraph 2 shall not benefit from the additional period of data protection referred to in Article 81(2), point (d), of [revised Directive 2001/83/EC]for orphan medicinal products referred to in Article 71(2), points (a) and (b) shall be prolonged by an additional 24 months where an application for orphan marketing authorisation is submitted in respect of a designated orphan medicinal product and that application includes the results of all studies conducted in compliance with an agreed paediatric investigation plan.
Amendment 415 #
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 or other relevant Union legislation under the conditions set out in Article 114. By [OP please insert the date = 12 months after the date of entering into force of this Regulation], the Commission shall make an assessment of other relevant Union legislation, and where appropriate draw up a list for which this Article shall apply.
Amendment 417 #
Proposal for a regulation
Article 113 – paragraph 4 – subparagraph 1
Article 113 – paragraph 4 – subparagraph 1
Where the Agency considers it appropriate to set up a regulatory sandbox for medicinal products which are likely to fall under the scope of this Regulation or other relevant Union legislation, it shall provide a recommendation to the Commission. The Agency shall list eligible products or category of products in that recommendation and shall include the sandbox plan referred to in paragraph 1.
Amendment 418 #
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, with academia, HTA bodies, patients and their caregivers, healthcare professionals, sponsors, or developers. 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 and other relevant Union legislation 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 420 #
Proposal for a regulation
Article 113 – paragraph 7 – point c
Article 113 – paragraph 7 – point c
(c) include as part of the sandbox plan the requirements of this Regulation and, of [revised Directive 2001/83/EC] or of other relevant Union legislation that cannot be complied with and shall include appropriate measures to mitigate potential risks to health and to the environment.
Amendment 438 #
Proposal for a regulation
Article 117 – paragraph 2
Article 117 – paragraph 2
2. The Agency, in collaboration with the working party referred to in Article 121(1), point (c), patients´ organisations and healthcare professionals and other interested parties shall draw up guidance to marketing authorisation holders as defined in Article 116(1) to put in place the shortage prevention plan.
Amendment 448 #
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 accessible by the relevant competent authorities.
Amendment 454 #
Proposal for a regulation
Article 121 – paragraph 2 – point f
Article 121 – paragraph 2 – point f
(f) as soon as possible inform the Agency of any actions foreseen or taken by that Member State to mitigate the shortage at national level.
Amendment 460 #
Proposal for a regulation
Article 121 – paragraph 5 – point d
Article 121 – paragraph 5 – point d
(d) as soon as possible inform the Agency of any actions foreseen or taken by that Member State in accordance with points (b) and (c) and report 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 467 #
Proposal for a regulation
Article 122 – paragraph 6 a (new)
Article 122 – paragraph 6 a (new)
6 a. The Agency shall assess measures notified by competent authorities of the Member States under Article 121 with regard to possible effects on the availability of medicines in other Member States, and shall where relevant report its findings to the Commission.
Amendment 476 #
Proposal for a regulation
Article 126 – paragraph 2 a (new)
Article 126 – paragraph 2 a (new)
2 a. Where relevant, the Commission may within the scope of its competences take appropriate measures to address findings of the Agency according to the assessment in Article 122 – paragraph 6 a (new).
Amendment 510 #
Proposal for a regulation
Article 146 – paragraph 8 – subparagraph 1
Article 146 – paragraph 8 – subparagraph 1
The scientific committees and any working parties and scientific advisory groups established in accordance with this Article shall in general matters establish contacts, on an advisory basis, with parties concerned with the use of medicinal products for human use, in particular patient and consumer organisations, including paediatric patients, and healthcare professionals’ associations. For that purpose working groups of patient and consumer organisations and healthcare professionals’ associations shall be established by the Agency. They shall ensure a fair representation of healthcare professionals, patients and consumers covering a wide range of experience and disease areas, including orphan, paediatric and geriatric diseases and advanced therapy medicinal products, and a broad geographical range.
Amendment 511 #
Proposal for a regulation
Article 146 – paragraph 8 – subparagraph 1 a (new)
Article 146 – paragraph 8 – subparagraph 1 a (new)
The Agency shall establish a procedure ensuring the remuneration through the Agency's budget, in accordance with the financial rules applicable to the Agency, of patients’ organisations representatives appointed as members or alternate members on scientific committees.
Amendment 520 #
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, international organisations, industries or other stakeholders, as relevant.
Amendment 526 #
Proposal for a regulation
Article 2 – paragraph 2 – point 7
Article 2 – paragraph 2 – point 7
(7) ‘significant benefit’ means a clinically relevant advantage or a major contribution to patient care of an orphan medicinal product if such an advantage or contribution benefits a substantial part of the target population;
Amendment 527 #
Proposal for a regulation
Annex I – point 3
Annex I – point 3
3. Medicinal products for human use containing an active substance which on 20 May 2004 was not authorised in the Union, excluding allergen products or herbal medicinal products, which shall in any case not be authorised by the Union.
Amendment 543 #
Proposal for a regulation
Article 2 – paragraph 2 – point 10
Article 2 – paragraph 2 – point 10
(10) ‘regulatory sandbox’ means a regulatory framework during which it is possible to develop, validate and test in a controlled environment innovative or adapted regulatory solutions that facilitate the development and authorisation of innovative products which are likely to fall in the scope of this Regulation, pursuant to a specific plan and for a limited time under regulatory supervision.
Amendment 661 #
Proposal for a regulation
Article 15 – paragraph 1 – point d
Article 15 – paragraph 1 – point d
Amendment 672 #
Proposal for a regulation
Article 15 – paragraph 1 a (new)
Article 15 – paragraph 1 a (new)
1 a. The marketing authorisation may furthermore be refused if, after verification of the particulars and documentation submitted in accordance with Article 6, the view is taken that 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.
Amendment 764 #
Proposal for a regulation
Article 26 – paragraph 4 – subparagraph 1
Article 26 – paragraph 4 – subparagraph 1
When compassionate use is envisaged by a Member State, or by the applicant or sponsor, the Committee for Medicinal Products for Human Use, after consulting the manufacturer or the applicant, may adopt opinions on the conditions for use, the conditions for distribution and the patients targeted. The opinions shall be updated where necessary.
Amendment 771 #
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 on its website in an accessible, searchable data format.
Amendment 841 #
Proposal for a regulation
Article 40 – paragraph 2
Article 40 – paragraph 2
2. The voucher referred to in paragraph 1 shall give the right to its holder to an additional 12 monthsperiod of data protection for one authorised medicinal product as set out according to paragraph 3 of this Article.
Amendment 845 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – introductory part
Article 40 – paragraph 3 – subparagraph 1 – introductory part
An antimicrobial shall be considered ‘priority antimicrobial’ if preclinical and clinical data underpin a significant clinical benefit with respect to antimicrobial resistance and it has at least one of the following characteristics:.
Amendment 852 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – point a
Article 40 – paragraph 3 – subparagraph 1 – point a
Amendment 854 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – point b
Article 40 – paragraph 3 – subparagraph 1 – point b
Amendment 863 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 1 – point c
Article 40 – paragraph 3 – subparagraph 1 – point c
Amendment 866 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 2
Article 40 – paragraph 3 – subparagraph 2
In the scientific assessment of the criteriapriority antimicrobials as referred to in the first subparagraph, and in the cathe Agency shall develop a set of antibiotics, the Agency shallcriteria takeing into account the ‘WHO priority pathogens list for R&D of new antibiotics’, or an equivalent list established at Union level; health system benefits, including with regard to safety and ease of administration; and pharmacological benefits, including novelty of the product.
Amendment 870 #
Proposal for a regulation
Article 40 – paragraph 3 – subparagraph 2 a (new)
Article 40 – paragraph 3 – subparagraph 2 a (new)
The Agency shall develop a weighting of each criteria and a corresponding scoring system allowing priority antimicrobials to be designated one of three categories of vouchers according to its expected impact on combatting antimicrobial resistance. The agency shall assign each voucher category a corresponding financial value which will be payed to the applicant following the auction process set out in Article 41.
Amendment 876 #
Proposal for a regulation
Article 40 – paragraph 4 – subparagraph 1 – point b
Article 40 – paragraph 4 – subparagraph 1 – point b
(b) provide information on all direct EU financial support received for research related to the development of the priority antimicrobial.
Amendment 907 #
Proposal for a regulation
Article 41 – paragraph 1 – subparagraph 1
Article 41 – paragraph 1 – subparagraph 1
A voucher may be used to extend the data protection fgranted by the Commission shall be subject to a public auction by the Agency. The financial value to be payed to the applicant by the winner of the auction is predetermined by the voucher category a period of 12 months of the priority antimicrobial or anothers set out in Article 40. Thus, those wishing to participate in the auction shall bid for the shortest data protection extension for which they are willing to pay the financial amount of the voucher category to the applicant. The voucher may be used to extend the data protection of a medicinal product authorised in accordance with this Regulation ofor the same or different marketing authorisation holderperiod of the winning bid.
Amendment 911 #
Proposal for a regulation
Article 41 – paragraph 1 – subparagraph 2
Article 41 – paragraph 1 – subparagraph 2
A voucher shall only be used once and in relation to a single centrally authorised medicinal product and only if that product is within its first fourleast two years of regulatory data protection is remaining for that product.
Amendment 919 #
Proposal for a regulation
Article 41 – paragraph 3
Article 41 – paragraph 3
3. AWhen an auction is completed, and the voucher mayhas been transferred to another marketing authorisation holder andthe auction winner, the voucher shall not be transferred further.
Amendment 922 #
Proposal for a regulation
Article 41 – paragraph 4
Article 41 – paragraph 4
4. AThe Agency shall market ing authorisation holder to whom a voucher is transferred shall notify the Agency of the transfer within 30 days, stating the value of the transaction between the two parties. The Agency shall make this informationformation regarding the voucher category value and the lenght of data exclusivity extension of the winning bid publicly available.
Amendment 947 #
Proposal for a regulation
Article 43 – paragraph 1
Article 43 – paragraph 1
This Chapter shall apply untilbe subject to a review by the Commission by [Note to OP: insert the date of 15 years after the date of entry into force of this Regulation] or until the date when the Commission has granted a total of 10 vouchers in accordance with this Chapter, whichever date is the earliest.
Amendment 1006 #
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 and new indications of medicinal products, that, based on preliminary evidence submitted by the developer fulfil one or more of the following conditions:
Amendment 1025 #
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)or advanced therapy medicinal products;
Amendment 1052 #
Proposal for a regulation
Article 63 – paragraph 2
Article 63 – paragraph 2
Amendment 1059 #
Proposal for a regulation
Article 63 – paragraph 3
Article 63 – paragraph 3
3. The Commission shall adopt the necessary provisions for implementing this Article by means of implementing acts in accordance with the procedure laid down in Article 173(2) in order to further specify the requirements referred to in paragraph 1.
Amendment 1071 #
Proposal for a regulation
Article 64 – paragraph 4 – subparagraph 1
Article 64 – paragraph 4 – subparagraph 1
The Agency shall adopt a decision granting or refusing the orphan designation based on the criteria referred to in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) within 90 days of the receipt of a valid application. The application is considered valid if it includes all the particulars and documentation referred to in paragraph 2.
Amendment 1110 #
Proposal for a regulation
Article 68 – paragraph 2 – subparagraph 1 (new)
Article 68 – paragraph 2 – subparagraph 1 (new)
For the purpose of paragraph 2, the definitions set out in Article 58a paragraph 1 of [revised Directive 2001/83/EC] shall apply.
Amendment 1126 #
Proposal for a regulation
Article 70 – title
Article 70 – title
Breakthrough Designated Orphan medicinal products addressing a high unmet medical need
Amendment 1129 #
Proposal for a regulation
Article 70 – paragraph 1 – introductory part
Article 70 – paragraph 1 – introductory part
1. An orphan medicinal product shall be considered as addressing a high unmetdesignated as a breakthrough orphan medicinal needproduct where it fulfilscan be demonstrated at the moment of designation that the one of the following requirements are met:
Amendment 1131 #
Proposal for a regulation
Article 70 – paragraph 1 – point a
Article 70 – paragraph 1 – point a
(a) there is no medicinal product authorised in the Union for such condition orwhere, despite medicinal products being authorised for such condition in the Union, the applicant demonstrates that the orphan medicinal product, in addition to having a significant benefit, will bring exceptional therapeutic advancementexists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Union, or;
Amendment 1138 #
Proposal for a regulation
Article 70 – paragraph 1 – point b
Article 70 – paragraph 1 – point b
(b) twhere, despite medicinal products being authorised for such condition in the Union, the applicant demonstrates that the orphan medicinal product uses a new and unique mechanism of action and the use of the orphan medicinal product results in a meaningful prevention of or reduction in disease morbidity or mortality or a major contribution to patient care for the relevant patient population.
Amendment 1145 #
Proposal for a regulation
Article 70 – paragraph 2
Article 70 – paragraph 2
Amendment 1149 #
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, as well as the stakeholders such as representatives of patients' organisations in the relevant disease areas, healthcare professionals, orphan medicinal product sponsors, representatives of pharmaceutical industry and other relevant stakeholders referred to in Article 162.
Amendment 1151 #
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, all relevant stakeholders and the authorities or bodies referred to in Article 162.
Amendment 1168 #
Proposal for a regulation
Article 71 – paragraph 2 – point a
Article 71 – paragraph 2 – point a
(a) nineten years for orphan medicinal products other than those referred to in points (b) and (c);
Amendment 1188 #
Proposal for a regulation
Article 71 – paragraph 2 – point b
Article 71 – paragraph 2 – point b
(b) twelven years for breakthrough orphan medicinal products addressing a high unmet medical need as referred to in Article 70;
Amendment 1204 #
Proposal for a regulation
Article 71 – paragraph 2 – point c
Article 71 – paragraph 2 – point c
(c) fivesix years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].
Amendment 1206 #
Proposal for a regulation
Article 71 – paragraph 2 – point c a (new)
Article 71 – paragraph 2 – point c a (new)
(c a) twelve years for orphan medicinal products addressing requirements set in Article 70, paragraph 1 point (a) and (b) in the paediatric population.
Amendment 1212 #
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, 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, provided that a market exclusivity period was still in force when the application for marketing authorisation was granted in the Unionsubmitted.
Amendment 1227 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 1
Article 72 – paragraph 1 – subparagraph 1
Amendment 1230 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 1
Article 72 – paragraph 1 – subparagraph 1
Amendment 1236 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 2
Article 72 – paragraph 1 – subparagraph 2
Amendment 1249 #
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 124 months for orphan medicinal products referred to in Article 71(2), points (a) and (b), 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 1257 #
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.
Amendment 1269 #
Proposal for a regulation
Article 72 – paragraph 3
Article 72 – paragraph 3
3. The orphan medicinal products which benefit from the prolongatUpon request from the applicant, the periond of market exclusivity referred to in the paragraph 2 shall not benefit from the additional period of data protection referred to in Article 81(2), point (d), of [revised Directive 2001/83/EC]for orphan medicinal products referred to in Article 71(2), points (a) and (b) shall be prolonged by an additional 24 months where an application for orphan marketing authorisation is submitted in respect of a designated orphan medicinal product and that application includes the results of all studies conducted in compliance with an agreed paediatric investigation plan.
Amendment 1277 #
Proposal for a regulation
Article 72 – paragraph 4 a (new)
Article 72 – paragraph 4 a (new)
4 a. An orphan medicinal product which benefits from the prolongation of market exclusivity as referred to in paragraph 3, shall not benefit from the rewards referred to in Article 86 [of revised Directive 2001/83/EC].
Amendment 1278 #
Proposal for a regulation
Article 72 – paragraph 4 b (new)
Article 72 – paragraph 4 b (new)
4 b. The holder of an orphan marketing authorisation shall be entitled to a total maximum period of 14 years of orphan marketing exclusivity from the time the orphan medicinal product in question first obtains an authorization as defined in Article 69.
Amendment 1391 #
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, and implement digital technologies in healthcare proceedings, capable of achieving 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 1405 #
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 or other relevant Union legislation under the conditions set out in Article 114. By [OP please insert the date = 12 months after the date of entering into force of this Regulation], the Commission shall make an assessment of other relevant Union legislation, and where appropriate draw up a list for which this Article shall apply.
Amendment 1419 #
Proposal for a regulation
Article 113 – paragraph 3 a (new)
Article 113 – paragraph 3 a (new)
3 a. The regulatory sandbox may also cover the exploration and application of medical devices, in-vitro diagnostics, in silico methods, AI and machine learning technologies in the developement, manifacturing, quality control, and administration of medicinal products, as their use can significanlty add to the efficiency, safety, and effectiveness of the life cycle of medicinal products.
Amendment 1426 #
Proposal for a regulation
Article 113 – paragraph 4 – subparagraph 1
Article 113 – paragraph 4 – subparagraph 1
Where the Agency considers it appropriate to set up a regulatory sandbox for medicinal products which are likely to fall under the scope of this Regulation or other relevant Union legislation, it shall provide a recommendation to the Commission. The Agency shall list eligible products or category of products in that recommendation and shall include the sandbox plan referred to in paragraph 1.
Amendment 1428 #
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, with academia, HTA bodies, patients and their caregivers, healthcare professionals, sponsors, or developers. 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 and other relevant Union legislation 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 1440 #
Proposal for a regulation
Article 113 – paragraph 7 – point c
Article 113 – paragraph 7 – point c
(c) include as part of the sandbox plan the requirements of this Regulation and, of [revised Directive 2001/83/EC] or of other relevant Union legislation that cannot be complied with and shall include appropriate measures to mitigate potential risks to health and to the environment.
Amendment 1481 #
Proposal for a regulation
Article 116 – paragraph 1 – point d
Article 116 – paragraph 1 – point d
(d) a temporary disruption in supply of a medicinal product in a given Member State, of an expected duration of in excess of two weeks or, based on the demand forecast of the marketing authorisation holder no less than sixthree months before the start of such temporary disruption of supply or, if this is not possible and where duly justified, as soon as they become aware of such temporary disruption, to allow the Member State to monitor any potential or actual shortage in accordance with Article 118(1).
Amendment 1491 #
Proposal for a regulation
Article 117 – paragraph 1
Article 117 – paragraph 1
1. The marketing authorisation holder as defined in Article 116(1) shall have in place and keep up to date a shortage prevention plan, for any medicinal product placed on the marketUnion list of critical medicinal products referred to in Article 131. The Commission is empowered to adopt delegated acts in accordance with Article 175 to supplement this Regulation by determining medicinal products for which a shortage prevention plan shall be maintained and kept up to date, including due to the lack of availability of alternatives. To put in place the shortage prevention plan, the marketing authorisation holder shall include the minimum set of information set out in Part V of Annex IV and take into account the guidance drawn up by the Agency according to paragraph 2.
Amendment 1506 #
Proposal for a regulation
Article 117 – paragraph 2
Article 117 – paragraph 2
2. The Agency, in collaboration with the working party referred to in Article 121(1), point (c), patients´ organisations and healthcare professionals and other interested parties shall draw up guidance to marketing authorisation holders as defined in Article 116(1) to put in place the shortage prevention plan.
Amendment 1531 #
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 accessible by the relevant competent authorities.
Amendment 1558 #
Proposal for a regulation
Article 121 – paragraph 2 – point f
Article 121 – paragraph 2 – point f
(f) as soon as possible inform the Agency of any actions foreseen or taken by that Member State to mitigate the shortage at national level.
Amendment 1561 #
Proposal for a regulation
Article 121 – paragraph 5 – point d
Article 121 – paragraph 5 – point d
(d) as soon as possible inform the Agency of any actions foreseen or taken by that Member State in accordance with points (b) and (c) and report 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 1592 #
Proposal for a regulation
Article 122 – paragraph 6 a (new)
Article 122 – paragraph 6 a (new)
Amendment 1630 #
Proposal for a regulation
Article 126 – paragraph 2 a (new)
Article 126 – paragraph 2 a (new)
2 a. Where relevant, the Commission may within the scope of its competences take appropriate measures to address findings of the Agency according to the assessment in Article 122 – paragraph 6 a (new).