Activities of Anders VISTISEN related to 2023/0131(COD)
Shadow reports (1)
REPORT 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 (53)
Amendment 221 #
Proposal for a regulation
Recital 9
Recital 9
(9) As to the scope of this Regulation, the authorisation of antimicrobials is, in principle, in ththe sole interest of safeguarding patients' health at Union level and managing public health concerns related to antimicrobial resistance, and therefore it should be made possible to authorise them at Union level.
Amendment 247 #
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 an advisory mechanism would address, as early as possible, questions related to borderline cases with other areas such as in particular biological products, food supplements and nutraceuticals, dietary products, biotechnology derived products, 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.
Amendment 273 #
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, healthcare professionals, industry, associations representing payers, academia or other stakeholders from all Member States, as relevant.
Amendment 276 #
Proposal for a regulation
Recital 43
Recital 43
(43) In the interest of public health, marketing authorisation decisions under the centralised procedure should be taken on the basis of the objective scientific criteria of quality, safety and efficacy of the medicinal product concerned, to the exclusion of economic and, demand, trade, geopolitical, expediency or commercial interest or any other considerations. However, Member States should be able, exceptionally, to prohibit the use in their territory of medicinal products for human use, biological products, food supplements and nutraceuticals, dietary products, biotechnology derived products.
Amendment 294 #
Proposal for a regulation
Recital 53
Recital 53
(53) Environmental risks may arise from medicinal products containing or consisting of genetically modified organisms. It is thus necessary to subject such medicinal products to an environmental risk-assessment procedure similar to the procedure under Directive 2001/18/EC of the European Parliament and of the Council51 , to be conducted in parallel with the evaluation, under a single Union procedure, of the quality, safety and efficacy of the medicinal product concerned. Tand the risks to the environment. Where the environmental risk-s assessment should be conducted in accordance with the requirements set out in this Regulation and in [revised Directive 2001/83/EC] which are based on the principles set out in Directive 2001/18/EC but taking into account the specificities of medicinal products. _________________ 51re absent or negligible, the normal requirements for the environmental risk assessment shall apply. To ensure the Union legislation on genetically modified organisms does not impede the development and approval of novel medicines containing and consisting of genetically modified organisms, it is also appropriate to foresee the adaptation of this Regulation and [revised Directive 2001/183/EC of the European Parliament and of the Council of 12 March 2001 on the deliberate release into the environment of genetically modified organisms and repealing Council Directive 90/220/EEC (OJ L 106, 17.4.2001, p. 1)] to lay down specific rules for these categories of medicines on the basis on an evaluation.
Amendment 313 #
Proposal for a regulation
Recital 68
Recital 68
(68) Before a medicinal product for human use is authorised for placing on the market of one or more Member States, it generally has to undergo extensive studies to ensure that it is safe, of high quality and effective for use in the target population. However, in the case of certain categories of medicinal products for human use, in order to meet unmet medical needs of patients and in the interest of public health, it may be necessary to grant a conditional marketing authorisation on the basis of less complete data than is normally the case. Such marketing authorisation should be granted subject to specific obligations. The categories of medicinal products for human use concerned should be the medicinal products, including orphan medicinal products, that aim at the treatment, prevention or medical diagnosis of seriously debilitating or life-threatening diseases, or that are intended to be used in emergency situations in response to public health threats. The decision to grant conditional authorization will be subjected to the principles of risk-mitigation, risk assessment, informed consent, close monitoring, transparency, following strict procedures of monitoring and reporting.
Amendment 322 #
Proposal for a regulation
Recital 76
Recital 76
Amendment 395 #
Proposal for a regulation
Recital 91
Recital 91
Amendment 397 #
Proposal for a regulation
Recital 92
Recital 92
Amendment 403 #
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 toonly authorised medicinal product in the Union shall 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 prescriptreatment. 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 according to the prescriptions of a pharmacopoeia and intended to be supplied directly to patients served by the pharmacy, mayto be authorised in all Member States to be considered as a satisfactory treatment if they are well known and safe and this is a general practice for the relevant patient population in the Unionmethod.
Amendment 409 #
Proposal for a regulation
Recital 94
Recital 94
(94) The competence to designate a medicinal product as an orphan medicinal product, in the form of a decision, is accorded to the Agency. The designation shall be valid from the day of the adoption of the decision by the Agency. This is expected to facilitate and expedite the designation procedure, while ensuring high level of scientific expertise. The orphan designation may be withdrawn at the request of the orphan medicine sponsor.
Amendment 410 #
Proposal for a regulation
Recital 95
Recital 95
Amendment 510 #
Proposal for a regulation
Recital 149
Recital 149
(149) It is therefore appropriate to envisage a centralised assessment of the ERA involving experts from the national competent authorities before and establish a science- and risk-based approach.
Amendment 525 #
(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 627 #
Proposal for a regulation
Article 7 – paragraph 5 – subparagraph 2 a (new)
Article 7 – paragraph 5 – subparagraph 2 a (new)
By derogation from this Article, where the marketing authorisation application concerns a medicinal product for human use containing or consisting of genetically modified organisms which comply with the criteria referred to in paragraph 3 of Article 5a of Regulation (EU) No 536/2014, Article 22 of [revised Directive 2001/83/EC] shall apply.
Amendment 628 #
Proposal for a regulation
Article 7 – paragraph 5 – subparagraph 2 b (new)
Article 7 – paragraph 5 – subparagraph 2 b (new)
No later than [note to OP = five years after the date of entry into application of this Regulation], the Commission shall conduct an impact assessment of the Union legislation on genetically modified organisms* on medicinal products containing or consisting of genetically modified organisms and produce an evaluation report, taking into account the experience gained from the application of this Regulation, [revised Directive 2001/83/EC], Regulation (EU) No 536/2014 and Regulation (EU) 2020/1043. On the basis of the impact assessment and evaluation, the Commission shall come forward with a legislative proposal to amend this Regulation and [revised Directive 2001/83/EC] to lay down specific rules on environmental risk assessments for medicines containing or consisting of genetically modified organisms outside the scope of the general Union legislation on genetically modified organisations.
Amendment 639 #
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 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 withdrawnrejected.
Amendment 708 #
Proposal for a regulation
Article 19 – paragraph 1 – subparagraph 2
Article 19 – paragraph 1 – subparagraph 2
In emergency situations, a conditional marketing authorisation or a new conditional therapeutic indication referred to in the first subparagraph may be granted also where comprehensive non-clinical or pharmaceutical data have not been supplied. However, this provision shall not apply to biological medicinal products.
Amendment 712 #
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 annuallyevery 3 months by the Agency for the first three years after granting the authorisation and every two years thereaftermonth thereafter. A conditional marketing authorisation cannot exceed 2 years.
Amendment 728 #
Proposal for a regulation
Article 19 – paragraph 6
Article 19 – paragraph 6
Amendment 763 #
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 765 #
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 769 #
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 778 #
Proposal for a regulation
Chapter II – Section 3
Chapter II – Section 3
Amendment 1055 #
Proposal for a regulation
Article 63 – paragraph 2
Article 63 – paragraph 2
Amendment 1057 #
Proposal for a regulation
Article 63 – paragraph 3
Article 63 – paragraph 3
3. The Commission shallmay 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 1080 #
Proposal for a regulation
Article 66 – paragraph 1
Article 66 – paragraph 1
1. An orphan designation shall be valid for seven yearsrom the day of the adoption of the decision granting the orphan designation by the Agency. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.
Amendment 1082 #
Proposal for a regulation
Article 66 – paragraph 2
Article 66 – paragraph 2
Amendment 1094 #
Proposal for a regulation
Article 66 – paragraph 5
Article 66 – paragraph 5
5. At any time, an orphan designation may be withdrawn at the request of the orphan medicine sponsor. The orphan medicine sponsor may provide a reasoned justification for the withdrawal request which shall be made publicly available.
Amendment 1104 #
Proposal for a regulation
Article 68 – paragraph 2
Article 68 – paragraph 2
2. Medicinal products designated as orphan medicinal products under the provisions of this Regulation shall be eligible for incentives made available by the Union and by the Member States to support research into, and the development and availability of, orphan medicinal products and in particular aid for research for small- and medium-sized undertakings and not-for-profit organisations provided for in framework programmes for research and technological development. For the purpose of paragraph 2, the definitions set out in Article 58a paragraph 1 of [revised Directive 2001/83/EC] shall apply.
Amendment 1127 #
Proposal for a regulation
Article 70 – title
Article 70 – title
Amendment 1130 #
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 following requirements are met:
Amendment 1132 #
Proposal for a regulation
Article 70 – paragraph 1 – point a
Article 70 – paragraph 1 – point a
(a) tThere is no medicinal product authorised in the Union for such condition orwhere, despite medicinal products being authorised for suchexists no satisfactory method of diagnosis, prevention or treatment of the condition in the Unquestion, the applicant demonstrates that the orphan medicinal product, in addition to having a significant benefit, will bring exceptional therapeutic advancement;at has been authorised in the Union, or
Amendment 1137 #
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 1147 #
Proposal for a regulation
Article 70 – paragraph 2
Article 70 – paragraph 2
2. A medicinal product for which an application hasBreakthrough designated orphan medicinal products shall be en submitted in accordance withtitled to enhanced scientific and regulatory support pursuant to Article 1360 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical needthis Regulation. 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).
Amendment 1150 #
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 other relevant stakeholders referred to in Article 162.
Amendment 1169 #
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 1184 #
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;
Amendment 1203 #
Proposal for a regulation
Article 71 – paragraph 2 – point c
Article 71 – paragraph 2 – point c
(c) fiseven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].
Amendment 1211 #
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 a market exclusivity period was still in force when the application for marketing authorisation was granted in the Unionsubmitted.
Amendment 1215 #
Proposal for a regulation
Article 71 – paragraph 5
Article 71 – paragraph 5
Amendment 1225 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 1
Article 72 – paragraph 1 – subparagraph 1
Amendment 1234 #
Proposal for a regulation
Article 72 – paragraph 1 – subparagraph 2
Article 72 – paragraph 1 – subparagraph 2
Amendment 1247 #
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 1268 #
Proposal for a regulation
Article 72 – paragraph 3
Article 72 – paragraph 3
3. The orphan medicinal products which benefit from the prolongation of market exclusivity referred to in the Upon request from the applicant, the period of market exclusivity 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 pursuant to [Revised Regulation] and that application includes the results of all studies conducted in compliance with an agreed paediatric investigation plan. The first subparagraph 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]. also apply where completion of the agreed paediatric investigation plan fails to lead to the authorisation of a paediatric indication, but the results of the studies conducted are reflected in the summary of product characteristics and, if appropriate, in the package leaflet of the medicinal product concerned. The 24-month extension of the period of market exclusivity shall be reflected in the marketing authorisation.
Amendment 1274 #
Proposal for a regulation
Article 72 – paragraph 4
Article 72 – paragraph 4
4. Article 71(3) equally applies ton orphan medicinal product which benefits from the prolongations of market exclusivity as referred to in paragraphs 1 and 2 3, shall not benefit from the rewards referred to in Article 86 [of revised Directive 2001/83/EC].
Amendment 1275 #
Proposal for a regulation
Article 72 – paragraph 4 a (new)
Article 72 – paragraph 4 a (new)
4 a. The total period of orphan marketing exclusivity for an orphan marketing authorisation shall not exceed twenty years from the date of the initial marketing authorisation.
Amendment 1402 #
Proposal for a regulation
Article 113 – paragraph 1 – point a
Article 113 – paragraph 1 – point a
(a) it is not possible to satisfactorily develop the medicinal product or category of products in compliance with the requirements applicable to medicinalsuch products due to scientific or regulatory challenges arising from characteristics or methods related to the product;
Amendment 1406 #
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 applicable Union legislation under the conditions set out in Article 114. By [OP please insert the date =18 months after the date of entering into force of this Regulation], the Commission shall draw up a list of applicable Union legislation for the application of this Article.
Amendment 1414 #
Proposal for a regulation
Article 113 – paragraph 3
Article 113 – paragraph 3
3. The Agency shall monitor the field of emerging medicinal products and mayshall 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. The Agency may set up consultation mechanism with other Union and non-Union regulatory agencies to facilitate its monitoring. The Agency shall draw up and regularly update a list of emerging medicinal or health products for which a regulatory sandbox may be established.
Amendment 1423 #
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 tomay be classified as a medicinal product or other categories of products falling under the scope of this Regulationlegislation referred to in paragraph 1, 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 1438 #
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], and other applicable Union legislation that cannot be complied with and shall include appropriate measures to mitigate potential risks to health and to the environment.
Amendment 1455 #
Proposal for a regulation
Article 114 – paragraph 3
Article 114 – paragraph 3
3. In duly justified cases, the marketing authorisation of a medicinal product developed under the regulatory sandbox may include derogations from the requirements set out in this Regulation and [revised Directive 2001/83/EC], and Regulation (EC) 1394/2007. Those derogations may entail adapted, enhanced, waived or deferred requirements. Each derogation shall be limited to what is apt and strictly necessary to attain the objectives pursued, duly justified and specified in the conditions to the marketing authorisation.