Sittings · Document

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

Laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006

Committee on the Environment, Public Health and Food Safety

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AM_Com_LegReport

Amendment 1062

Fulvio Martusciello

Proposal for a regulation

Article 64 – paragraph 1

Text proposed by the Commission

Amendment

1. The orphan medicine sponsor shall submit an application for the designation of the orphan medicinal product to the Agency at any stage of the development of the medicinal product before the application for marketing authorisation referred to in Articles 5 and 6 is submitted.

1. The orphan medicinal product sponsor shall submit an application for the designation of the orphan medicinal product to the Agency at any stage of the development of the medicinal product before the application for marketing authorisation referred to in Articles 5 and 6 is submitted.

Or. en

Amendment 1063

Fulvio Martusciello

Proposal for a regulation

Article 64 – paragraph 2 – subparagraph 1 – introductory part

Text proposed by the Commission

Amendment

The application of the orphan medicine sponsor shall be accompanied by the following particulars and documentation:

The application for the designation of the orphan medicinal product shall be accompanied by the following particulars and documentation:

Or. en

Amendment 1064

Tomislav Sokol

Proposal for a regulation

Article 64 – paragraph 2 – subparagraph 1 – introductory part

Text proposed by the Commission

Amendment

The application of the orphan medicine sponsor shall be accompanied by the following particulars and documentation:

The application for the designation of the orphan medicinal product shall be accompanied by the following particulars and documentation:

Or. en

Amendment 1065

Tilly Metz

Proposal for a regulation

Article 64 – paragraph 2 – subparagraph 1 – point d

Text proposed by the Commission

Amendment

(d) justification that the criteria laid down in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) are fulfilled and a description of the stage of development, including the expected therapeutic indication.

(d) justification that the criteria laid down in Article 63(1) are fulfilled and a description of the stage of development, including the expected therapeutic indication.

Or. en

Amendment 1066

Fulvio Martusciello

Proposal for a regulation

Article 64 – paragraph 3

Text proposed by the Commission

Amendment

3. The Agency shall, in consultation with the Member States, the Commission and interested parties, draw up detailed guidelines on the required procedure, format and content of applications for designation and for the transfer of the orphan designation pursuant to Article 65.

3. The Agency shall verify the validity of the application and share its draft scientific conclusions with the applicant. The applicant shall be invited to provide their observations on the draft conclusions.The Agency shall, in consultation with the Member States, the Commission and interested parties, draw up detailed guidelines on the required procedure, format and content of applications for designation and for the transfer of the orphan designation pursuant to Article 65.

Or. en

Amendment 1067

Tomislav Sokol

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 1

Text proposed by the Commission

Amendment

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.

Within the timelines for adoption of a decision foreseen in paragraph 3, the Agency shall transmit its scientific conclusions to the applicant.

Within 30 days of receipt of the scientific conclusions, the sponsor may submit to the Agency a written request, citing detailed grounds, for a re-examination.

Within 30 days following receipt of a request for re-examination, the Agency shall confirm or revise its previous scientific conclusions.Where the Agency considers it necessary, it may consult the Committee for Medicinal Products for Human Use or the appropriate working parties when re-examining the above mentioned scientific conclusions.

If, within the 30-day period referred to in subparagraph 3, the applicant does not request re-examination, the scientific conclusions shall become definitive.

The Agency shall adopt a decision within a period not exceeding 10 days following the date on which the scientific conclusions have become definitive.

The application is considered valid if it includes all the particulars and documentation referred to in paragraph 2.

Or. en

Amendment 1068

Fulvio Martusciello

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 1

Text proposed by the Commission

Amendment

The Agency shall adopt a decision granting or refusing the orphan designation based on the criteria referred to in Article 63(1) within 90 days of the receipt of a valid application.

Within the timelines for adoption of a decision foreseen in subparagraph 1, the Agency shall transmit its scientific conclusions to the applicant.

Within 30 days of receipt of the scientific conclusions, the sponsor may submit to the Agency a written request, citing detailed grounds, for a re-examination.

If, within the 30-day period referred to in subparagraph 3, the applicant does not request re-examination, the scientific conclusions shall become definitive.

The Agency shall adopt a decision within a period not exceeding 10 days following the date on which the scientific conclusions have become definitive

The application is considered valid if it includes all the particulars and documentation referred to in paragraph 2.

Or. en

Amendment 1069

Margarita de la Pisa Carrión

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 1

Text proposed by the Commission

Amendment

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

Or. en

Amendment 1070

Tilly Metz

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 1

Text proposed by the Commission

Amendment

The Agency shall adopt a decision granting or refusing the orphan designation based on the criteria referred to in Article 63(1) 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.

Or. en

Amendment 1071

Pernille Weiss

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 1

Text proposed by the Commission

Amendment

Or. en

Justification

See amendment to Article 63 – paragraph 2.

Amendment 1072

Margarita de la Pisa Carrión

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 1

Text proposed by the Commission

Amendment

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 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.

Or. en

Amendment 1073

Fulvio Martusciello

Proposal for a regulation

Article 64 – paragraph 4 – subparagraph 3

Text proposed by the Commission

Amendment

The decision together with the Annexes referred to in this paragraph shall be notified to the applicant.

The Agency shall adopt a decision within a period not exceeding 10 days following the date on which the scientific conclusions have become definitive.

Or. en

Amendment 1074

Tilly Metz

Proposal for a regulation

Article 64 – paragraph 5

Text proposed by the Commission

Amendment

5. Decisions of the Agency on granting or refusing the orphan designation shall be made public after deletion of any information of a commercially confidential nature.

5. Decisions of the Agency and a summary of consultation referred to in paragraph 4 on granting or refusing the orphan designation shall be made public after deletion of any information of a commercially confidential nature.

Or. en

Amendment 1075

Tiemo Wölken

Proposal for a regulation

Article 65 – paragraph 2 – point c a (new)

Text proposed by the Commission

Amendment

(c a) detailed reason(s) for the transferring of the orphan designation

Or. en

Amendment 1076

Fulvio Martusciello

Proposal for a regulation

Article 66 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

deleted

Or. en

Amendment 1077

Erik Poulsen

Proposal for a regulation

Article 66 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

deleted

Or. en

Amendment 1078

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 66 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

deleted

Or. en

Amendment 1079

Stelios Kympouropoulos

Proposal for a regulation

Article 66 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

deleted

Or. en

Amendment 1080

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 66 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

1. An orphan designation shall be valid from 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.

Or. en

Justification

Limiting the validity of the OD will disproportionally impact smaller companies, who often request the OD early-on in the development to secure additional funding. There is also little data to support that shortening the duration of the OD will accelerate development.According to data, the average time from orphan designation to marketing authorisation for mature companies was 4.7 years and 7.3 years for small biotech companies. In cases where the OD is transferred, the time to marketing authorisation was longer on average.

Amendment 1081

Tomislav Sokol

Proposal for a regulation

Article 66 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan designation shall be valid for seven years. During this period, the orphan medicine sponsor shall be eligible for incentives referred to in Article 68.

Or. en

Amendment 1082

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 66 – paragraph 2

Text proposed by the Commission

Amendment

2. By way of derogation from paragraph 1, on the basis of a justified request of the orphan medicine sponsor, the Agency may extend the validity, where the orphan medicine sponsor can provide evidence that the relevant studies supporting the use of the designated orphan medicinal product in the applied conditions are ongoing and promising with regard to the filing of a future application. Such an extension shall be limited in time, taking into account the expected remaining time needed to file an application for marketing authorisation.

deleted

Or. en

Amendment 1083

Erik Poulsen

Proposal for a regulation

Article 66 – paragraph 2

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1084

Stelios Kympouropoulos

Proposal for a regulation

Article 66 – paragraph 2

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1085

Tomislav Sokol

Proposal for a regulation

Article 66 – paragraph 2

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1086

Fulvio Martusciello

Proposal for a regulation

Article 66 – paragraph 2

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1087

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 66 – paragraph 2

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1088

Stelios Kympouropoulos

Proposal for a regulation

Article 66 – paragraph 3

Text proposed by the Commission

Amendment

3. By way of derogation from paragraph 1, where an orphan designation is valid at the time when a marketing authorisation for an orphan medicinal product has been submitted in accordance with Article 5, the orphan designation shall remain valid until a decision is adopted by the Commission in accordance with Article 13(2).

deleted

Or. en

Amendment 1089

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 66 – paragraph 3

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1090

Erik Poulsen

Proposal for a regulation

Article 66 – paragraph 3

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1091

Fulvio Martusciello

Proposal for a regulation

Article 66 – paragraph 3

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1092

Fulvio Martusciello

Proposal for a regulation

Article 66 – paragraph 4

Text proposed by the Commission

Amendment

4. An orphan designation ceases to be valid once an orphan medicine sponsor has obtained a marketing authorisation for the relevant medicinal product in accordance with Article 13(2).

4. An orphan designation ceases to be valid once an orphan medicine sponsor has obtained a marketing authorisation for the relevant medicinal product in accordance with Article 13(2). An orphan designation shall however remain valid in case the indication of the initial marketing authorisation addresses only a subset of the population affected by the designated orphan condition or where the orphan medicinal product sponsor can provide evidence that studies supporting the use of the designated orphan medicinal product are planned or ongoing with respect to additional indications within the scope of the designated condition / orphan designation.

Or. en

Amendment 1093

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 66 – paragraph 4

Text proposed by the Commission

Amendment

4. An orphan designation ceases to be valid once an orphan medicine sponsor has obtained a marketing authorisation for the relevant medicinal product in accordance with Article 13(2).

4. An orphan designation ceases to be valid once an orphan medicine sponsor has obtained a marketing authorisation for the relevant medicinal product in accordance with Article 13(2). However, when the original marketing authorization only addresses part of the population affected by the designated orphan condition, the orphan designation shall remain valid.

Or. en

Amendment 1094

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 66 – paragraph 5

Text proposed by the Commission

Amendment

5. At any time, an orphan designation may be withdrawn at the request of the orphan medicine sponsor.

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.

Or. en

Amendment 1095

Tomislav Sokol

Proposal for a regulation

Article 66 – paragraph 5

Text proposed by the Commission

Amendment

5. At any time, an orphan designation may be withdrawn at the request of the orphan medicine sponsor.

Or. en

Amendment 1096

Tilly Metz

Proposal for a regulation

Article 67 – paragraph 3 – point c a (new)

Text proposed by the Commission

Amendment

(c a) the stage of development;

Or. en

Amendment 1097

Andrey Slabakov

Proposal for a regulation

Article 67 – paragraph 3 – point f a (new)

Text proposed by the Commission

Amendment

(f a) any request and related decision under article 66 (2).

Or. en

Amendment 1098

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

Proposal for a regulation

Article 68 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. The orphan medicine sponsor may, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

1. The orphan medicine sponsor shall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

Or. en

Justification

Protocol assistance has proven to increase the rate of success

Amendment 1099

Susana Solís Pérez

Proposal for a regulation

Article 68 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. The orphan medicine sponsor may, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

1. The orphan medicine sponsor shall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

Or. en

Amendment 1100

Stelios Kympouropoulos

Proposal for a regulation

Article 68 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. The orphan medicine sponsor may, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

1. The orphan medicine sponsor shall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

Or. en

Amendment 1101

Kateřina Konečná

Proposal for a regulation

Article 68 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. The orphan medicine sponsor may, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

1. The orphan medicine sponsor shall, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

Or. en

Amendment 1102

Maria Angela Danzì

Proposal for a regulation

Article 68 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. The orphan medicine sponsor may, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

1. The orphan medicine sponsor will, prior to the submission of an application for marketing authorisation, request advice from the Agency on the following:

Or. it

Amendment 1103

Tilly Metz

Proposal for a regulation

Article 68 – paragraph 1 – point a

Text proposed by the Commission

Amendment

(a) the conduct of the various tests and trials necessary to demonstrate the quality, safety and efficacy of the medicinal product, as referred to Article 138(1), second subparagraph, point (p);

(a) the conduct of the various tests and trials necessary to demonstrate the quality, safety, efficacy and environmental impact of the medicinal product, as referred to Article 138(1), second subparagraph, point (p);

Or. en

Amendment 1104

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 68 – paragraph 2

Text proposed by the Commission

Amendment

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 provided for in framework programmes for research and technological development.

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.

Or. en

Amendment 1105

Alessandra Moretti

Proposal for a regulation

Article 68 – paragraph 2

Text proposed by the Commission

Amendment

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 entities, such as for example the European Medicine Facility as established in article 40a, provided for in framework programmes for research and technological development.

Or. en

Amendment 1106

Tilly Metz

Proposal for a regulation

Article 68 – paragraph 2

Text proposed by the Commission

Amendment

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 entities not engaged in economic activity provided for in framework programmes for research and technological development.

Or. en

Amendment 1107

Maria Angela Danzì

Proposal for a regulation

Article 68 – paragraph 2

Text proposed by the Commission

Amendment

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 entities provided for in framework programmes for research and technological development.

Or. it

Justification

Not-for-profit entities can play an important role in developing innovative treatments (e.g. advanced therapy medicinal products, which are orphan medicinal products) and improving their access to cancer treatment patents at a fair price.

Amendment 1108

Cindy Franssen, Tom Vandenkendelaere

Proposal for a regulation

Article 68 – paragraph 2

Text proposed by the Commission

Amendment

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 entities provided for in framework programmes for research and technological development.

Or. en

Amendment 1109

Sirpa Pietikäinen

Proposal for a regulation

Article 68 – paragraph 2

Text proposed by the Commission

Amendment

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 entities provided for in framework programmes for research and technological development.

Or. en

Amendment 1110

Pernille Weiss, Stelios Kympouropoulos

Proposal for a regulation

Article 68 – paragraph 2 – subparagraph 1 (new)

Text proposed by the Commission

Amendment

For the purpose of paragraph 2, the definitions set out in Article 58a paragraph 1 of [revised Directive 2001/83/EC] shall apply.

Or. en

Justification

See amendment in Article 58a of the draft report on the revised Directive 2001/83/EC.

Amendment 1111

Tomislav Sokol

Proposal for a regulation

Article 68 – paragraph 2 – subparagraph 1 (new)

Text proposed by the Commission

Amendment

For the purpose of paragraph 2, the definitions set out in Article 58a paragraph 1 of [revised Directive 2001/83/EC] shall apply.

Or. en

Amendment 1112

Tilly Metz

Proposal for a regulation

Article 69 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

In addition, the applicant shall demonstrate that the medicinal product has been granted an orphan designation and that the criteria set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) are fulfilled for the therapeutic indication sought.

In addition, the applicant shall demonstrate that the medicinal product has been granted an orphan designation and that the criteria set out in Article 63(1) are fulfilled for the therapeutic indication sought.

Or. en

Amendment 1113

Tomislav Sokol

Proposal for a regulation

Article 69 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Where appropriate, the applicant shall provide relevant evidence to demonstrate that the medicinal product addresses a high unmet medical need as specified in Article 70(1).

Where appropriate, the applicant shall provide relevant evidence to demonstrate that the medicinal product constitutes a breakthrough orphan medicinal product in accordance with in Article 70(1).

Or. en

Amendment 1114

Tilly Metz

Proposal for a regulation

Article 69 – paragraph 3 – subparagraph 1

Text proposed by the Commission

Amendment

The Committee for Medicinal Products for Human Use shall assess whether the medicinal product fulfils the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2). In the situation referred in paragraph 2, subparagraph 2, that Committee shall also assess whether the medicinal product addresses a high unmet medical need as specified in Article 70(1).

The Committee for Medicinal Products for Human Use shall assess whether the medicinal product fulfils the requirements set out in Article 63(1). In the situation referred in paragraph 2, subparagraph 2, that Committee shall also assess whether the medicinal product addresses a high unmet medical need as specified in Article 70(1).

Or. en

Amendment 1115

Tomislav Sokol

Proposal for a regulation

Article 69 – paragraph 3 – subparagraph 1

Text proposed by the Commission

Amendment

The Committee for Medicinal Products for Human Use shall assess whether the medicinal product fulfils the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2). In the situation referred in paragraph 2, subparagraph 2, that Committee shall also assess whether the medicinal product constitutes a breakthrough orphan medicinal product in accordance with in Article 70(1).

Or. en

Amendment 1116

Tilly Metz

Proposal for a regulation

Article 69 – paragraph 4

Text proposed by the Commission

Amendment

4. The orphan marketing authorisation shall cover only those therapeutic indications, which fulfil the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2) at the time when the orphan marketing authorisation is granted.

4. The orphan marketing authorisation shall cover only those therapeutic indications, which fulfil the requirements set out in Article 63(1) at the time when the orphan marketing authorisation is granted.

Or. en

Amendment 1117

Tilly Metz

Proposal for a regulation

Article 69 – paragraph 6

Text proposed by the Commission

Amendment

6. An applicant may submit an application for a separate marketing authorisation for other indications which do not fulfil the requirements set out in Article 63(1) or in the relevant delegated acts adopted in accordance with Article 63(2).

6. An applicant may submit an application for a separate marketing authorisation for other indications which do not fulfil the requirements set out in Article 63(1).

Or. en

Amendment 1118

Tilly Metz

Proposal for a regulation

Article 69 – paragraph 6 a (new)

Text proposed by the Commission

Amendment

6 a. As laid down in Article 17, authorised products shall be subject to re-evaluation after 5 years. In cases of orphan medicinal products, the Committee for Medicinal Products for Human use shall in addition to the product’s safety, efficacy and environmental impact also review the criteria in set in Article 63 and assess the profitability of the product, taking into account all authorised indications, as laid down in Article 72.

Or. en

Amendment 1119

Maria Angela Danzì

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(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 advancement;

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. it

Amendment 1120

Maria Veronica Rossi, Gianna Gancia

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. it

Amendment 1121

Jessica Polfjärd

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. en

Justification

The criteria set out in this article are already in a large extend addressed in the requirements of unmet medical needs (with the exemption of advancement in therapeutic which are exceptional addressed in a different article). Criteria for high met medical need for orphan medicines as a distinct category risk undermining unmet medical needs by suggesting some needs are less important. In this regard, a definition of unmet medical needs would need a more holistic approach and better understanding to not undermine the purpose of this regulation with regard to these needs, medicines and treatments.

Amendment 1122

Margarita de la Pisa Carrión

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. en

Amendment 1123

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. en

Amendment 1124

Sunčana Glavak

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. en

Amendment 1125

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

Proposal for a regulation

Article 70

Text proposed by the Commission

Amendment

Article 70

deleted

Orphan medicinal products addressing a high unmet medical need

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

Or. en

Justification

95% of people living with a rare disease do not have an approved therapy available to them (European Commission). In this context, creating an artificial hierarchy of patients and their needs, or considering that some are more "meaningful", is innaprorpiate and does not reflect the reality of rare diseases. See amendment on article 71 for alternative proposal.

Amendment 1126

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 70 – title

Text proposed by the Commission

Amendment

Orphan medicinal products addressing a high unmet medical need

Breakthrough Designated Orphan medicinal products

Or. en

Amendment 1127

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 70 – title

Text proposed by the Commission

Amendment

Orphan medicinal products addressing a high unmet medical need

Breakthrough Designated Orphan Medicinal Products

Or. en

Amendment 1128

Fulvio Martusciello

Proposal for a regulation

Article 70 – paragraph 1

Text proposed by the Commission

Amendment

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

deleted

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

Or. en

Amendment 1129

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 70 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

1. An orphan medicinal product shall be designated as a breakthrough orphan medicinal product where it can be demonstrated at the moment of designation that the one of the following requirements are met:

Or. en

Amendment 1130

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 70 – paragraph 1 – introductory part

Text proposed by the Commission

Amendment

1. An orphan medicinal product shall be considered as addressing a high unmet medical need where it fulfils the following requirements:

1. An orphan medicinal product shall be designated as a breakthrough orphan medicinal product where it can be demonstrated at the moment of designation that the following requirements are met:

Or. en

Amendment 1131

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 70 – paragraph 1 – point a

Text proposed by the Commission

Amendment

(a) there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Union, or;

Or. en

Amendment 1132

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 70 – paragraph 1 – point a

Text proposed by the Commission

Amendment

(a) There exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Union, or

Or. en

Amendment 1133

Andrey Slabakov

Proposal for a regulation

Article 70 – paragraph 1 – point a

Text proposed by the Commission

Amendment

(a) there is no medicinal product authorised in the Union for such condition, or;

Or. en

Amendment 1134

Tilly Metz

Proposal for a regulation

Article 70 – paragraph 1 – point a

Text proposed by the Commission

Amendment

(a) there is no medicinal product authorised in the Union for such condition or where, 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 advancement; and

Or. en

Amendment 1135

Andrey Slabakov

Proposal for a regulation

Article 70 – paragraph 1 – point a a (new)

Text proposed by the Commission

Amendment

(a a) 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 clinical benefit based on improved efficacy, safety and contribution to patient care, will bring a therapeutic advancement to a part of the relevant patient population;

Or. en

Amendment 1136

Andrey Slabakov

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

deleted

Or. en

Amendment 1137

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) Where, 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 medicinal product results in meaningful prevention of or reduction in disease morbidity or mortality or a major contribution to patient care for the relevant population.

Or. en

Amendment 1138

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) where, 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 medicinal product results in meaningful prevention of or reduction in disease morbidity or mortality or a major contribution to patient care for the relevant population.

Or. en

Amendment 1139

Cindy Franssen, Tom Vandenkendelaere

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality or in a meaningful positive impact on quality of life for the relevant patient population.

Or. en

Amendment 1140

Ondřej Knotek, Frédérique Ries, Billy Kelleher

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity, mortality, severity or long term side effects for the relevant patient population.

Or. en

Amendment 1141

Marian-Jean Marinescu

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) the use of the medicinal product results in a meaningful reduction in disease morbidity, acute or long-term toxicity or mortality for the relevant patient population.

Or. en

Justification

A medicinal product should also be considered as addressing a UMN or high UMN when it reduces acute or long-term toxicity for children with cancer

Amendment 1142

Tilly Metz

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) the use of the orphan medicinal product results in a substantial reduction in disease morbidity or mortality for the relevant patient population.

Or. en

Amendment 1143

Kateřina Konečná

Proposal for a regulation

Article 70 – paragraph 1 – point b

Text proposed by the Commission

Amendment

(b) the use of the orphan medicinal product results in a meaningful reduction in disease morbidity or mortality for the relevant patient population.

(b) the use of the orphan medicinal product results in a reduction in disease morbidity or mortality for the relevant patient population.

Or. en

Amendment 1144

Marian-Jean Marinescu

Proposal for a regulation

Article 70 – paragraph 1 – point b a (new)

Text proposed by the Commission

Amendment

(b a) the use of the medicinal product results in a substantial improvement of patients' quality of life.

Or. en

Justification

A medicinal product should also be considered as addressing a UMN or high UMN when it improves quality of life for patients.

Amendment 1145

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 70 – paragraph 2

Text proposed by the Commission

Amendment

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

deleted

Or. en

Amendment 1146

Fulvio Martusciello

Proposal for a regulation

Article 70 – paragraph 2

Text proposed by the Commission

Amendment

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

deleted

Or. en

Amendment 1147

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 70 – paragraph 2

Text proposed by the Commission

Amendment

2. A medicinal product for which an application has been submitted in accordance with Article 13 of [revised Directive 2001/83/EC] shall not be considered as addressing a high unmet medical need.

2. Breakthrough designated orphan medicinal products shall be entitled to enhanced scientific and regulatory support pursuant to Article 60 of this 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).

Or. en

Amendment 1148

Fulvio Martusciello

Proposal for a regulation

Article 70 – paragraph 3

Text proposed by the Commission

Amendment

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

deleted

Or. en

Amendment 1149

Tomislav Sokol, Stelios Kympouropoulos, Pernille Weiss

Proposal for a regulation

Article 70 – paragraph 3

Text proposed by the Commission

Amendment

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

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.

Or. en

Amendment 1150

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 70 – paragraph 3

Text proposed by the Commission

Amendment

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

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.

Or. en

Amendment 1151

Pernille Weiss, Stelios Kympouropoulos

Proposal for a regulation

Article 70 – paragraph 3

Text proposed by the Commission

Amendment

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

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.

Or. en

Amendment 1152

Ondřej Knotek, Catherine Amalric, Billy Kelleher

Proposal for a regulation

Article 70 – paragraph 3

Text proposed by the Commission

Amendment

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission, the authorities, bodies and stakeholders referred to in Article 162.

Or. en

Amendment 1153

Andrey Slabakov

Proposal for a regulation

Article 70 – paragraph 3

Text proposed by the Commission

Amendment

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission and the authorities or bodies referred to in Article 162.

3. Where the Agency adopts scientific guidelines for the application of this Article, it shall consult the Commission, the authorities and bodies referred to in Article 162.

Or. en

Amendment 1154

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 1

Text proposed by the Commission

Amendment

1. Where an orphan marketing authorisation is granted and without prejudice to intellectual property law, the Union and the Member States shall not grant a marketing authorisation or extend an existing marketing authorisation, for the same therapeutic indication, in respect of a similar medicinal product for the duration of market exclusivity set out in paragraph 2.

1. Where an orphan marketing authorisation is granted and without prejudice to intellectual property law, a similar medicinal product authorized for the same therapeutic indication shall not be placed on the market for the duration of market exclusivity set out in paragraph 2.

Or. en

Amendment 1155

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

deleted

Or. en

Amendment 1156

Maria Angela Danzì

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) twelve years for orphan medicinal products for whose indication the Union has not approved a satisfactory treatment method;

Or. it

Amendment 1157

Frédérique Ries, Catherine Amalric, Max Orville, Billy Kelleher

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) eight years for orphan medicinal products other than those referred to in points (b), (ba) and (c);

Or. en

Amendment 1158

Ondřej Knotek, Billy Kelleher

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) nine years for orphan medicinal products other than those referred to in points (b), (c) and (ca);

Or. en

Amendment 1159

Catherine Amalric, Max Orville, Billy Kelleher, Ondřej Knotek

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) nine years for orphan medicinal products other than those referred to in points (b), (c) and (ca);

Or. en

Amendment 1160

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) twelve years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1161

Margarita de la Pisa Carrión

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) twelve years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1162

Dolors Montserrat

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) twelve years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1163

Jessica Polfjärd

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) twelve years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Justification

It is important to further facilitate for further investment in areas where the scientific challenges are acute. Improved and strengthened incentives will strengthen the competitiveness in the EU and ensure that the EU is a pioneer to development and research and develop new, cutting-edge medicines in different areas. Hence, to condition an enhanced baseline protection to objective criteria is needed, since it would provide operators with sufficient certainty to undertake investments and in particularly during challenging and high-risk circumstances.

Amendment 1164

Fulvio Martusciello

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) twelve years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1165

Kateřina Konečná

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) eight years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1166

Tilly Metz

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) seven years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1167

Stelios Kympouropoulos

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) ten years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1168

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) ten years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Amendment 1169

Anders Vistisen

on behalf of the ID Group

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) ten years for orphan medicinal products other than those referred to in points (b) and (c);

Or. en

Justification

The amendment proposes to build on the current OMP Regulation – with a 10-year OME baseline – and adapt the COM proposal on modulation to better reflect the realities of the industry landscape, in particular investments decisions which are made years before marketing authorisation. The COM and Rapporteur proposal to reduce the baseline OME to 9 and 8 years respectively, together with the introduction of criteria-based extensions will both reduce appetite to support early development and clarity of investment horizon.

Amendment 1170

Maria Veronica Rossi, Gianna Gancia

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) ten years for orphan medicinal products other than those referred to in point (b);

Or. it

Amendment 1171

Andreas Glück

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) ten years for orphan medicinal products other than those referred to in point (c);

Or. en

Justification

Status Quo shall be kept.

Amendment 1172

Sunčana Glavak

Proposal for a regulation

Article 71 – paragraph 2 – point a

Text proposed by the Commission

Amendment

(a) nine years for orphan medicinal products other than those referred to in points (b) and (c);

(a) Ten years for orphan medicinal products other than those referred to in point (c);

Or. en

Amendment 1173

Maria Veronica Rossi, Gianna Gancia

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

deleted

Or. it

Amendment 1174

Andreas Glück

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

deleted

Or. en

Amendment 1175

Sunčana Glavak

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

deleted

Or. en

Amendment 1176

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) thirteen years for orphan medicinal products which satisfy any of the following criteria:

i. there is no medicinal product authorised in the Union for such condition or where

ii. even though the Union has authorized medicinal products for such condition, the applicant demonstrates that the orphan medicinal product, in addition to having a significant benefit, will result in exceptional therapeutic advancement or iii. the condition affects not more than 0,5 in 10,000 persons in the Union when the application for an orphan designation is submitted.

Or. en

Amendment 1177

Dolors Montserrat

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) thirteen years for orphan medicinal products which fulfil one of the following requirements:

i. there is no medicinal product authorised in the Union for such condition or where

ii. 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 advancement or

iii. the condition affects not more than 0,5 in 10,000 persons in the Union when the application for an orphan designation is submitted.

Or. en

Amendment 1178

Maria Angela Danzì

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) ten years for orphan medicinal products if:

1. the Union has authorised fewer than three (3) orphan medicinal products for the indication in question, or;

2. notwithstanding any authorisations for medicinal products for the condition in question, no orphan medicinal product has received EU authorisation for part of the target population or subsets thereof as regards the therapeutic indication of the new medicinal product, or;

3. the Union has authorised an orphan medicinal product for use according to its indication, but that orphan medicinal product either has a new mechanism of application or is a new technology;

Or. it

Amendment 1179

Fulvio Martusciello

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) thirteen years for orphan medicinal products which fulfil one of the following requirements:

i. there is no medicinal product authorised in the Union for such condition or where

iii. the condition affects not more than 0,5 in 10,000 persons in the Union when the application for an orphan designation is submitted.

Or. en

Amendment 1180

Kateřina Konečná

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

The market exclusivity for the orphan indication shall not preclude that generics and biosimilars can enter the market for other uses of the medicinal product that are not protected by data and market protection, patents, or supplementary protection certificates.

Or. en

Justification

Rare diseases, especially those with very low prevalence, often pose unique scientific and clinical challenges. To encourage companies to invest in research for these conditions, additional incentives, such as extended market exclusivity and research grants, are vital. A graduation system to provide different levels of incentives depending on factors to best address the unmet needs of people living with a rare disease should be introduced. For example, targeting very to ultra-rare conditions, or those with no therapeutic option yet, may constitute a basis for a reward.

Amendment 1181

Margarita de la Pisa Carrión

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) therteen years for orphan medicinal products that are the first for that indication, that represent an important advance or that involve treatment for diseases of very low prevalence

Or. en

Amendment 1182

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

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) twelve years for orphan medicinal products where no satisfactory treatment has been approved in the Union for the indication in question.

Or. en

Amendment 1183

Stelios Kympouropoulos

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

Or. en

Amendment 1184

Anders Vistisen

on behalf of the ID Group

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

Or. en

Amendment 1185

Andrey Slabakov, Elisabetta De Blasis

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

Or. en

Amendment 1186

Tilly Metz

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) eight years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

Or. en

Amendment 1187

Jessica Polfjärd

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) thirteen years for orphan medicinal products and that one of the following requireiments can be fullfilled:

Or. en

Justification

Amendment 1188

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 71 – paragraph 2 – point b

Text proposed by the Commission

Amendment

(b) ten years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70;

(b) twelve years for breakthrough orphan medicinal products as referred to in Article 70;

Or. en

Amendment 1189

Jessica Polfjärd

Proposal for a regulation

Article 71 – paragraph 2 – point b – point i (new)

Text proposed by the Commission

Amendment

i) no medicinal product in the Union are authorised for such condition;

Or. en

Justification

Amendment 1190

Jessica Polfjärd

Proposal for a regulation

Article 71 – paragraph 2 – point b – point ii (new)

Text proposed by the Commission

Amendment

ii) regardless if medicinal products in the Union are being authorised for such condition, the applicant can demonstrate that the orphan medicinal product (apart of having a demonstrated benifit) will contribute to exceptional advancement for therapeutic;

Or. en

Justification

Amendment 1191

Jessica Polfjärd

Proposal for a regulation

Article 71 – paragraph 2 – point b – point iii (new)

Text proposed by the Commission

Amendment

iii) the condition affects no more than 0,5 in 10,000 persons in the European Union when the application designated for an orphan has been submitted.

Or. en

Justification

Amendment 1192

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

Proposal for a regulation

Article 71 – paragraph 2 – point b a (new)

Text proposed by the Commission

Amendment

(b a) Ten years for orphan medicinal products where one of the following criteria applies:

i. fewer than three orphan medicinal products have been approved in the Union for the indication in question;

ii. despite medicinal products being authorised for the indication in question, none has been approved in the Union for the relevant subpopulation that is covered by the therapeutic indication of the new medicinal product;

iii. an orphan medicinal product has been approved in the Union for the indication, but the new orphan medical product will represent a new mechanism of action or technology, and will result in significant reduction in disease morbidity or mortality for the relevant patient population, or a major contribution to the quality of life of the relevant population.

Or. en

Amendment 1193

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 2 – point b a (new)

Text proposed by the Commission

Amendment

(b a) ten years for an orphan medicinal product where:

1. fewer than three orphan medicinal products have been approved in the Union for the indication in question, or;

2. despite medical products being authorised for such a condition, no orphan medicinal product has been approved in the Union for a part of the target population or subpopulation that is covered by the therapeutic indication for the new medicinal product, or;

3. an orphan medicinal product has been approved in the Union for the indication, but the orphan medical product will represent a new mechanism of action or technology;

Or. en

Amendment 1194

Catherine Amalric, Max Orville, Billy Kelleher, Ondřej Knotek

Proposal for a regulation

Article 71 – paragraph 2 – point b a (new)

Text proposed by the Commission

Amendment

(b a) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70 in the paediatric population;

Or. en

Amendment 1195

Stelios Kympouropoulos

Proposal for a regulation

Article 71 – paragraph 2 – point b a (new)

Text proposed by the Commission

Amendment

(b a) twelve years for orphan medicinal products addressing an unmet medical need as referred to in Article 70 in the paediatric population;

Or. en

Amendment 1196

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 2 – point b b (new)

Text proposed by the Commission

Amendment

(b b) eight years for an orphan medicinal product that does not fall under paragraphs (b), (ba) or (bb);

Or. en

Amendment 1197

Tilly Metz

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

deleted

Or. en

Justification

Products marketed under Article 13 are based on bibliographic data only, meaning the MA holder did not run any additional clinical trial and based their finding on existing available research. Given that the product would generate profit by being sold, we do not find it reasonable to provide market exclusivity in this case

Amendment 1198

Jessica Polfjärd

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) seven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Justification

Amendment 1199

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) seven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1200

Dolors Montserrat

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) seven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1201

Margarita de la Pisa Carrión

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) seven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1202

Fulvio Martusciello

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) seven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1203

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) seven years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1204

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

(c) six years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1205

Kateřina Konečná

Proposal for a regulation

Article 71 – paragraph 2 – point c

Text proposed by the Commission

Amendment

(c) five years for orphan medicinal products which have been authorised in accordance with Article 13 of [revised Directive 2001/83/EC].

Or. en

Amendment 1206

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 71 – paragraph 2 – point c a (new)

Text proposed by the Commission

Amendment

(c a) twelve years for orphan medicinal products addressing requirements set in Article 70, paragraph 1 point (a) and (b) in the paediatric population.

Or. en

Amendment 1207

Marian-Jean Marinescu

Proposal for a regulation

Article 71 – paragraph 2 – point c a (new)

Text proposed by the Commission

Amendment

(c a) twelve years for orphan medicinal products addressing a high unmet medical need as referred to in Article 70 in the paediatric population;

Or. en

Justification

Some paediatric cancers do not exist in adults. Therefore the original path of first in adult second in child through PIPs does not work. Therefore there is a need for first-in-child incentive.

Amendment 1208

Maria Angela Danzì

Proposal for a regulation

Article 71 – paragraph 2 – point c a (new)

Text proposed by the Commission

Amendment

(ca) eight years for orphan medicinal products that are not referred to in points (a), (b) or (c);

Or. it

Amendment 1209

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

Proposal for a regulation

Article 71 – paragraph 3

Text proposed by the Commission

Amendment

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 marketing authorisation was granted in the Union.

3. Where a marketing authorisation holder holds more than one orphan marketing authorisations for the same active substance, other than in cases foreseen in Article 72, paragraph 2, subparagraph 2, those authorisations shall not benefit from separate market exclusivity periods. The duration of the market exclusivity shall start from the date when the first orphan marketing authorisation was granted in the Union.

Or. en

Amendment 1210

Maria Veronica Rossi, Gianna Gancia

Proposal for a regulation

Article 71 – paragraph 3

Text proposed by the Commission

Amendment

3. Where a marketing authorisation holder holds more than one orphan marketing authorisations for the same active substance, those authorisations shall benefit from separate market exclusivity periods whose duration is outlined in paragraph 2. The duration of each market exclusivity period shall start from the date when the corresponding orphan marketing authorisation was granted in the Union.

Or. it

Amendment 1211

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 71 – paragraph 3

Text proposed by the Commission

Amendment

3. Where a marketing authorisation holder holds more than one orphan marketing authorisations for the same active substance, those authorisations shall benefit from separate market exclusivity periods, provided a market exclusivity period was still in force when the application for marketing authorisation was submitted.

Or. en

Amendment 1212

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 71 – paragraph 3

Text proposed by the Commission

Amendment

3. Where a marketing authorisation holder holds more than one orphan marketing authorisations for the same active substance, those authorisations shall benefit from separate market exclusivity periods, provided that a market exclusivity period was still in force when the application for marketing authorisation was submitted.

Or. en

Amendment 1213

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 3 a (new)

Text proposed by the Commission

Amendment

3 a. Where the orphan marketing authorisation holders benefit from market exclusivity for an orphan medicinal product under paragraph 1 of this Article, they must demonstrate that they meet the requirements set in paragraph 1 of Article 72a of this Regulation.

Or. en

Amendment 1214

Tilly Metz

Proposal for a regulation

Article 71 – paragraph 4 – point c a (new)

Text proposed by the Commission

Amendment

(c a) a compulsory license has been granted in accordance with [Regulation on compulsory licensing for crisis management and amending Regulation (EC) 816/2006].

Or. en

Amendment 1215

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 71 – paragraph 5

Text proposed by the Commission

Amendment

5. The submission, validation and assessment of the application for the marketing authorisation and granting the marketing authorisation for a generic or biosimilar product to the reference medicinal product for which market exclusivity has expired, shall not be prevented by the market exclusivity of a similar product to the reference medicinal product.

deleted

Or. en

Amendment 1216

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 5

Text proposed by the Commission

Amendment

5. The submission, validation and assessment of the application for the marketing authorisation and granting the marketing authorisation for a generic or biosimilar product to the reference medicinal product, shall not be prevented by the market exclusivity of a similar product to the reference medicinal product.

Or. en

Amendment 1217

Andrey Slabakov

Proposal for a regulation

Article 71 – paragraph 6

Text proposed by the Commission

Amendment

6. The market exclusivity of the orphan medicinal product shall not prevent the submission, validation and assessment of an application for a marketing authorisation for a similar medicinal product, including generics and biosimilars, where the remainder of the duration of the market exclusivity is less than two years.

6. The market exclusivity of the orphan medicinal product shall not prevent the submission, validation, assessment of an application for and granting of a marketing authorisation for a similar medicinal product, including generics and biosimilars, where the remainder of the duration of the initial market exclusivity is less than two years.

Or. en

Amendment 1218

Tiemo Wölken

Proposal for a regulation

Article 71 – paragraph 6

Text proposed by the Commission

Amendment

6. The market exclusivity of the orphan medicinal product shall not prevent the submission, validation, assessment or granting of an application for a marketing authorisation for a similar medicinal product, including generics and biosimilars, where the remainder of the duration of the market exclusivity is less than two years.

Or. en

Amendment 1219

Tilly Metz

Proposal for a regulation

Article 71 – paragraph 7

Text proposed by the Commission

Amendment

7. Where the Agency adopts scientific guidelines for the application of paragraphs 1 and 4, it shall consult the Commission.

7. The Agency shall adopt scientific guidelines for the application of paragraphs 1 and 4, it shall consult the Commission.

Or. en

Justification

This should be independent scientific guidelines, Commission is a political institution

Amendment 1220

Maria Veronica Rossi, Gianna Gancia

Proposal for a regulation

Article 72

Text proposed by the Commission

Amendment

Article 72

deleted

Prolongation of market exclusivity

The periods of market exclusivity referred to in Article 71, paragraph 2, points (a) and (b), shall be prolonged by 12 months, where the orphan marketing authorisation holder can demonstrate that the conditions referred to in Article 81(2), point (a), and Article 82(1) [of revised Directive 2001/83/EC] are fulfilled.

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

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) 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.

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

3. The orphan medicinal products which benefit from the prolongation 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].

4. Article 71(3) equally applies to the prolongations of market exclusivity referred to in paragraphs 1 and 2.

Or. it

Amendment 1221

Tilly Metz

Proposal for a regulation

Article 72 – title

Text proposed by the Commission

Amendment

Prolongation of market exclusivity

Prolongation and reduction of market exclusivity period

Or. en

Amendment 1222

Frédérique Ries, Catherine Amalric, Susana Solís Pérez, Max Orville, Andreas Glück, Billy Kelleher

Proposal for a regulation

Article 72 – paragraph 1

Text proposed by the Commission

Amendment

1. The periods of market exclusivity referred to in Article 71, paragraph 2, points (a) and (b), shall be prolonged by 12 months, where the orphan marketing authorisation holder can demonstrate that the conditions referred to in Article 81(2), point (a), and Article 82(1) [of revised Directive 2001/83/EC] are fulfilled.

deleted

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

Or. en

Amendment 1223

Tilly Metz

Proposal for a regulation

Article 72 – paragraph 1

Text proposed by the Commission

Amendment

deleted

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

Or. en

Amendment 1224

Andrey Slabakov

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1225

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1226

Margarita de la Pisa Carrión

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1227

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1228

Fulvio Martusciello

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1229

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1230

Pernille Weiss

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Justification

See amendment to Article 81(2), point (a), and Article 82(1) of the draft report on the revised Directive 2001/83/EC.

Amendment 1231

Andreas Glück

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Justification

Tying incentive systems to market access is not the right approach and leads to uncertainty among producers. Supply of medical medicinal product in all Member States can also be achieved differently, for example by an obligation to submit an application for pricing and reimbursement in all Member States.

Amendment 1232

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 1

Text proposed by the Commission

Amendment

deleted

Or. en

Amendment 1233

Andrey Slabakov

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Amendment 1234

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Amendment 1235

Fulvio Martusciello

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Amendment 1236

Pernille Weiss

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Justification

See amendment to Article 81(2) to (5) of the draft report on the revised Directive 2001/83/EC.

Amendment 1237

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Amendment 1238

Margarita de la Pisa Carrión

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Amendment 1239

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 1 – subparagraph 2

Text proposed by the Commission

Amendment

The procedures set out in Articles 82(2) to (5) [of revised Directive 2001/83/EC] shall accordingly apply to the prolongation of market exclusivity.

deleted

Or. en

Amendment 1240

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 1 a (new)

Text proposed by the Commission

Amendment

1 a. The period of market exclusivity shall be prolonged by an additional 24 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. If the newly approved therapeutic indication meets one of the requirements listed in Article 71(2) point (b), and where the first orphan marketing authorisation was not granted a period of market exclusivity as referred in Article 71(2) point (b), the period of market exclusivity shall be prolonged by 36 months in total.

Or. en

Amendment 1241

Margarita de la Pisa Carrión

Proposal for a regulation

Article 72 – paragraph 1 a (new)

Text proposed by the Commission

Amendment

1 a. The period of market exclusivity shall be prolonged by 24 months for orphan medicinal products referred to in Article 71, paragraph 2, point (a), if one of its therapeutic indications fulfils one of the following requirements:

i. there is no medicinal product authorised in the Union for such condition or;

iii. the condition affects not more than 0,5 in 10,000 persons in the Union when the application for an orphan designation is submitted. When applying to new therapeutic indications, this prolongation shall apply for indications approved at least two years before the end of the exclusivity period.

Or. en

Justification

The HUMN incentive should also apply to follow-on indications in order to account for the reality of science: indications aiming to address underserved areas can be developed after the initial MA. Such an extension is critical to effectively steer R&D in all areas of UMN. This is also to align with the definition of Unmet Medical Need under Article 83 of the Directive that explicitly refers to the indications of the products. For High UMN, the definition should include diseases with a very low prevalence (<0.5/10,000) as alternative criteria to first-in-condition medicines or those providing exceptional therapeutic advancement. There is indeed a need to further incentivize research and development in diseases that affect such small populations, for which there is typically less investment due to the risky, lengthy and costly nature of such research.

Amendment 1242

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 2

Text proposed by the Commission

Amendment

2. 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) 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.

deleted

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

Or. en

Amendment 1243

Andrey Slabakov

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

The period of market exclusivity shall be prolonged by an additional 36 months for orphan medicinal products referred to in Article 71(2), points (a), (b) and (c), if at least four years before the end of the exclusivity period, the orphan marketing authorisation holder obtains a marketing authorisation for a different orphan condition.

Or. en

Amendment 1244

Maria Angela Danzì

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

The period of market exclusivity shall be prolonged by an additional 36 months for orphan medicinal products referred to in Article 71(2), points (a),(b) and (c a), if at least two years before the end of the exclusivity period, the orphan marketing authorisation holder obtains a marketing authorisation for a new therapeutic indication for a different orphan condition.

Or. it

Amendment 1245

Fulvio Martusciello

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

The period of market exclusivity shall be prolonged by an additional 24 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. If the newly approved therapeutic indication meets one of the requirements listed in Article 71(2) point (b), and where the first orphan marketing authorisation was not granted a period of market exclusivity as referred in Article 71(2) point (b), the period of market exclusivity shall be prolonged by 36 months in total.

Or. en

Amendment 1246

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

The period of market exclusivity shall be prolonged by an additional 24 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. If the recently approved therapeutic indication satisfies one of the criteria mentioned in Article 71(2) point (b) and a period of market exclusivity was not granted for the first orphan marketing authorization as mentioned in Article 71(2) point (b), the period of market exclusivity shall be extended by a total of 36 months.

Or. en

Amendment 1247

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

Or. en

Justification

it is proposed to increase the incentive for new indication to 24 months and to implement a decrease in additional OME to 12 months after 2 new indications for different orphan conditions. The overall cap to OME introduced will also prevent misuse. Considering in practice very few marketing authorisations extend beyond 2 to 3 indications, the proposal would reward continuous investments in a specific therapy that could be extended to new patient population. The amendments below also introduces the choice to the marketing authorisation holders as regards the completion of a paediatric investigation plan (PIP). With the amendment, sponsors could decide between the extension of supplementary protection certificate (SPC) or OME. This would ensure that all sponsors complete PIP even when they would not be eligible for a SPC extension.

Amendment 1248

Margarita de la Pisa Carrión

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

Or. en

Amendment 1249

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

Or. en

Amendment 1250

Tilly Metz

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

The period of market exclusivity shall be prolonged by an additional 6 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.

Or. en

Justification

We can only accept these provisions if exclusivity periods established in Article 71 are shortened, in no way we would agree going beyond (current) accumulated 11 years of exclusivity

Amendment 1251

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

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 1

Text proposed by the Commission

Amendment

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.

Or. en

Amendment 1252

Maria Angela Danzì

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

deleted

Or. it

Amendment 1253

Fulvio Martusciello

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

deleted

Or. en

Amendment 1254

Kateřina Konečná

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

deleted

Or. en

Justification

Once a product has been approved for a specific indication, any new, additional indication or extension of the existing orphan indication, requires a separate assessment by the EMA and marketing authorisation decision by the Commission. As it is not a simple or quick process and in fact, only a few medicines have more than one orphan indication, capping the extension of market exclusivity to two years may deter sponsors from investigating in new areas of clinical development.

Amendment 1255

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

deleted

Or. en

Amendment 1256

Andrey Slabakov

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

deleted

Or. en

Amendment 1257

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2

Text proposed by the Commission

Amendment

Such a prolongation may be granted twice, if the new therapeutic indications are each time for different orphan conditions.

Such a prolongation may be granted twice, if the new therapeutic indications are for different orphan conditions.

Or. en

Amendment 1258

Andrey Slabakov

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2 a (new)

Text proposed by the Commission

Amendment

When the marketing authorisation holder obtains any further marketing authorisations for one or more therapeutic indications covering different orphan conditions for the same orphan medicinal product, these specific therapeutic indications shall be protected by a separate market exclusivity of three years, starting from the expiration of the market exclusivity for the entire product under Article 71, where relevant as extended under paragraphs 1 and 2 of this Article.

Or. en

Amendment 1259

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

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2 a (new)

Text proposed by the Commission

Amendment

Any subsequent marketing authorisation obtained by the marketing authorisation holder for one or more new therapeutic indications for different orphan conditions shall receive a separate market exclusivity of three years, starting from the expiration date of the market exclusivity for the entire product under Article 71, where relevant as extended under paragraphs 1 and 2 of this article.

Or. en

Justification

This amendment aims at reaping the maximum benefits from the research already done by encouraging the exploration of further indications while addressing the risk of evergreening (stacking of indication protections) which delays the entry of generics.

Amendment 1260

Maria Angela Danzì

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2 a (new)

Text proposed by the Commission

Amendment

When the orphan marketing authorisation holder obtains any further marketing authorisations for one or more therapeutic indications relative to different orphan conditions for the same orphan medicinal product, these specific therapeutic indications shall be protected by a separate market exclusivity of three years from the expiry of the market exclusivity for the entire product pursuant to Article 71, where appropriate in accordance with Article 71(1) and (2).

Or. it

Amendment 1261

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2 a (new)

Text proposed by the Commission

Amendment

After the orphan medical product in question first receives an authorization as stated in Article 69, the holder of an orphan marketing authorization shall be entitled to a maximum of [15] years of orphan marketing exclusivity.

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Amendment 1262

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 2 – subparagraph 2 b (new)

Text proposed by the Commission

Amendment

If requested by the applicant as an option instead of the stipulated reward in Article 86 of the revised Directive, the duration of market exclusivity for orphan medicinal products, as specified in Article 71(2), points (a) and (b), will be extended by an extra 24 months. This extension applies when an application for orphan marketing authorization is submitted for a designated orphan medicinal product under the Revised Regulation, and the application incorporates the findings of all studies conducted in adherence to an agreed-upon paediatric investigation plan. The first subparagraph shall also be applicable where fulfilment of the stipulated paediatric investigation plan does not result in the approval of a pediatric indication. However, the findings from the conducted studies should be incorporated into the summary of product characteristics and, if deemed suitable, into the package leaflet of the respective medicinal product. The extension of the market exclusivity period by 24 months will be acknowledged in the marketing authorization.

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Amendment 1263

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 2 a (new)

Text proposed by the Commission

Amendment

2 a. The holder of an orphan marketing authorisation shall be entitled to a total maximum period of [15] years of orphan marketing exclusivity from the time the orphan medicinal product in question first obtains an authorization as defined in Article 69.

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Amendment 1264

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

Proposal for a regulation

Article 72 – paragraph 2 a (new)

Text proposed by the Commission

Amendment

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.

Or. en

Justification

This amendment aims at rewarding orphan medicinal product sponsors choosing Europe first, hereby helping European patients to access innovative treatment early.

Amendment 1265

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 2 b (new)

Text proposed by the Commission

Amendment

2 b. As an alternative to the reward foreseen under Article 86 [of revised Directive] and 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 shall 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 will be reflected in the marketing authorisation.

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Amendment 1266

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 2 c (new)

Text proposed by the Commission

Amendment

2 c. An orphan medicinal product which benefits from the prolongation of market exclusivity as referred to in paragraph 4, shall not benefit from the rewards referred to in Article 86 [of revised Directive].

Or. en

Amendment 1267

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 2 d (new)

Text proposed by the Commission

Amendment

2 d. The limitation referred to in paragraph 3 shall not apply where the period of orphan marketing exclusivity is extended in accordance with paragraph 4 in relation to such extension.

Or. en

Amendment 1268

Anders Vistisen

on behalf of the ID Group

Aurélia Beigneux

Proposal for a regulation

Article 72 – paragraph 3

Text proposed by the Commission

Amendment

3. 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 shall 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.

Or. en

Amendment 1269

Tomislav Sokol, Pernille Weiss

Proposal for a regulation

Article 72 – paragraph 3

Text proposed by the Commission

Amendment

3. 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 and that application includes the results of all studies conducted in compliance with an agreed paediatric investigation plan.

Or. en

Amendment 1270

Dolors Montserrat

Proposal for a regulation

Article 72 – paragraph 3

Text proposed by the Commission

Amendment

Or. en

Amendment 1271

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 3 a (new)

Text proposed by the Commission

Amendment

3 a. The restriction mentioned in paragraph 3 shall not apply when the orphan marketing exclusivity period is prolonged as outlined in paragraph 4 concerning that extension.

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Amendment 1272

Massimiliano Salini, Francesca Peppucci

Proposal for a regulation

Article 72 – paragraph 3 b (new)

Text proposed by the Commission

Amendment

3 b. The extended market exclusivity mentioned in paragraph 2 does not apply to orphan medicinal products, and they will not receive the extra period of data protection outlined in Article 81(2), point (d) of the revised Directive 2001/83/EC.

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Amendment 1273

Tilly Metz

Proposal for a regulation

Article 72 – paragraph 4

Text proposed by the Commission

Amendment

4. Article 71(3) equally applies to the prolongations of market exclusivity referred to in paragraphs 1 and 2.

deleted

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Amendment 1274

Anders Vistisen, Aurélia Beigneux

Proposal for a regulation

Article 72 – paragraph 4

Text proposed by the Commission

Amendment

4. Article 71(3) equally applies to the prolongations of market exclusivity referred to in paragraphs 1 and 2.

4. 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].

Or. en

Amendment 1275

Anders Vistisen, Aurélia Beigneux

Proposal for a regulation

Article 72 – paragraph 4 a (new)

Text proposed by the Commission

Amendment

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.

Or. en

Amendment 1276

Tilly Metz

Proposal for a regulation

Article 72 – paragraph 4 a (new)

Text proposed by the Commission

Amendment

4 a. The market exclusivity period shall be reduced to five years if, at the end of the fifth year, it is established, in respect of the medicinal product concerned, that the criteria laid down in Article 63 are no longer met or where it is shown on the basis of available evidence that the product is sufficiently profitable not to justify maintenance of market exclusivity. The sponsor shall provide the Agency with the information necessary to re-assess the criteria laid down in Article 63 and the externally audited product-related profit margins for all authorised indications at least nine months before end of the fifth year.

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