A4-0078/99

Proposal for a European Parliament and Council Regulation on orphan medicinal products (COM(98)0450 - C4-0470/98 - 98/0240(COD))

The proposal was approved with the following amendments:

(Amendments 1 and 2)

Recital 2a (new)

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(2a) Whereas, as regards investment in research into, and the development of, medicinal products to counter certain infectious diseases which occur mainly in tropical regions but rarely within the territory of the European Union, additional incentive measures are probably needed, as shown by the experience of the United States; whereas the Commission should examine, in close cooperation with industry and the scientific world in the Member States, appropriate international bodies and non-governmental organisations and those of the developing countries concerned, the situation of orphan medicinal products and put forward suitable proposals to the European Parliament and the Council in the form of a draft regulation leading to the introduction of a policy to support research into, and the development of, therapeutic or prophylactic medicinal products which would enable the main infectious diseases currently prevailing in developing countries to be kept under control;

(Amendement 3)

Recital 5

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(5) Whereas objective criteria for designation should be established; whereas these criteria should be based on the prevalence of the condition for which diagnosis, prevention or treatment is sought; whereas a prevalence of no more than five affected persons per ten thousand is generally regarded as the appropriate threshold; whereas medicinal products intended for a life-threatening or seriously debilitating communicable disease should be eligible even when the prevalence is higher than five per ten thousand;

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(5)

Whereas objective criteria for designation should be established; whereas these criteria should be based on the prevalence of the condition for which diagnosis, prevention or treatment is sought; whereas a prevalence of no more than five affected persons per ten thousand is generally regarded as the appropriate threshold; whereas medicinal products intended for a life-threatening or serious and chronic disease should be eligible even when the prevalence is higher than five per ten thousand;

(Amendment 4)

Recital 8

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(8) Whereas experience in the United States and Japan shows that the strongest incentive for industry to invest in the development and marketing of orphan medicinal products is the prospect of obtaining market exclusivity for a certain number of years, during which part of the investment might be recovered; whereas data protection under Article 4(8)(a)(iii) of Council Directive 65/65/EEC is not sufficient incentive for that purpose; whereas market exclusivity should however be limited to the therapeutic indication for which orphan medicinal product designation has been obtained; whereas, in the interest of patients, the market exclusivity granted to an orphan medicinal product should not prevent the marketing of a similar medicinal product which is safer, more effective or otherwise clinically superior;

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(8)

Whereas experience in the United States and Japan shows that the strongest incentive for industry to invest in the development and marketing of orphan medicinal products is the prospect of obtaining market exclusivity for a certain number of years, during which part of the investment might be recovered; whereas data protection under Article 4(8)(a)(iii) of Council Directive 65/65/EEC is not sufficient incentive for that purpose; whereas market exclusivity should however be limited to the therapeutic indication for which orphan medicinal product designation has been obtained, without in any way jeopardising existing intellectual property rights; whereas, in the interest of patients, the market exclusivity granted to an orphan medicinal product should not prevent the marketing of a similar medicinal product which is safer, more effective or otherwise clinically superior;

(Amendment 5)

Recital 10

>Original text>

(10) Whereas the specific programme Biomed 2, of the fourth framework programme for research and technological development (1994 to 1998), is supporting research on the treatment of rare diseases, including methodologies for rapid schemes for the development of orphan medicinal products and inventories of available orphan medicinal products in Europe; whereas these grants are to promote the establishment of cross-national cooperation in order to implement basic and clinical research on rare diseases; whereas research on rare diseases will continue to be a priority for the Commission, as it has been introduced in the Commission's proposal for the fifth framework programme (1998 to 2002) for research and technological development; whereas this Regulation establishes a legal framework which will allow the swift and effective implementation of the outcome of this research;

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(10)

Whereas the specific programme Biomed 2, of the fourth framework programme for research and technological development (1994 to 1998), is supporting research on the treatment of rare diseases, including methodologies for rapid schemes for the development of orphan medicinal products and inventories of available orphan medicinal products in Europe; whereas these grants are to promote the establishment of cross-national cooperation in order to implement basic and clinical research on rare diseases; whereas research on rare diseases will continue to be a priority for the Commission and the European Parliament, as it has been introduced in the Commission's proposals for the fifth framework programme (1998 to 2002) for research and technological development; whereas this Regulation establishes a legal framework which will allow the swift and effective implementation of the outcome of this research;

(Amendment 7)

Article 3(2)

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2. Notwithstanding paragraph 1, a medicinal product may also be designated as orphan medicinal product if its sponsor can establish that the medicinal product is intended for a life-threatening or seriously debilitating communicable disease in the Community and that it is unlikely that, without incentives, the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment.

>Text following EP vote>

2.

Notwithstanding paragraph 1, a medicinal product may also be designated as orphan medicinal product if its sponsor can establish that the medicinal product is intended for a life-threatening disease, a debilitating disease or a serious and chronic disease in the Community and that it is unlikely that, without incentives, the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment.

(Amendment 8)

Article 4(1)

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1. A Committee for Orphan Medicinal Products, hereinafter referred to as 'the committee', is hereby set up.

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1.

A Committee for Orphan Medicinal Products, hereinafter referred to as 'the committee', is hereby set up within the European Agency for the Evaluation of Medicinal Products.

(Amendment 9)

Article 4(3)

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3. The Committee shall consist of one member nominated by each Member State, three members nominated by the Commission to represent patient organisations and three members nominated by the Commission on the basis of a recommendation from the Agency. The members of the Committee shall be appointed for a term of three years which shall be renewable. They shall be chosen by reason of their role and experience in treatment of or research into rare diseases.

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3.

The Committee shall consist of one member nominated by each Member State, three members nominated by the European Parliament to represent patient organisations and three members nominated by the Commission on the basis of a recommendation from the Agency. The members of the Committee shall be appointed for a term of three years which shall be renewable. They shall be chosen by reason of their role and experience in treatment of or research into rare diseases. Whenever necessary they may seek the assistance of an expert.

(Amendment 10)

Article 4(6a) (new)

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6a. Members of the Committee shall be required, even after their duties have ceased, not to disclose information of the kind covered by the obligation of professional secrecy.

(Amendment 11)

Article 5(1)

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1. In order to obtain the designation of a medicinal product as orphan medicinal product, the sponsor shall submit an application to the Agency.

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1.

In order to obtain the designation of a medicinal product as orphan medicinal product, the sponsor shall submit an application to the Agency, at any stage of the development of the pharmaceutical product before an application for registration is submitted.

(Amendment 12)

Article 5(9a) (new)

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9a. Each year the sponsor shall provide the Agency with a report on the state of development of the designated medicinal product.

(Amendment 13)

Article 5(9b) (new)

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9b. In order to secure the transfer to another sponsor of the designation of an orphan medicinal product, the holder of that designation shall submit a specific application to the Agency. In consultation with the Member States, the Agency and the interested parties, the Commission shall adopt a detailed guide concerning the form in which transfer applications must be submitted and also the contents of such applications.

(Amendment 14)

Article 6(2)(a)

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(a) assistance in the development of a protocol and for the follow up of clinical investigations;

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(a)

assistance in the development of a protocol for pre-clinical and clinical trials during the development phase and for the follow up of clinical investigations; in exceptional cases the Agency may authorise the medicinal product being made available, under specific conditions, before marketing authorisation has been granted. It can propose an explanatory note to that effect;

(Amendment 15)

Article 7(2)

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2. A special contribution from the Community distinct from that provided for in Article 57 of Regulation (EEC) No 2309/93, will be allocated every year to the Agency. This contribution will be used exclusively by the Agency to waive, in part or in total, the fees payable under Community rules adopted pursuant to Regulation (EEC) No 2309/93. A detailed report of the use made of this special contribution shall be presented by the Executive Director of the Agency at the end of each year. Any surplus occurring in a given year shall be carried forward and deducted from the special contribution for the following year.

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2.

A special contribution from the Community, on a scale sufficient to cover all the applications submitted and thus able to produce the maximum incentive, and distinct from that provided for in Article 57 of Regulation (EEC) No 2309/93, will be allocated every year to the Agency. This contribution will be used exclusively by the Agency to waive, in part or in total, the fees payable under Community rules adopted pursuant to Regulation (EEC) No 2309/93. A detailed report of the use made of this special contribution shall be presented by the Executive Director of the Agency at the end of each year. Any surplus occurring in a given year shall be carried forward and deducted from the special contribution for the following year.

(Amendment 16)

Article 8(1)

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1. Where a marketing authorisation is granted pursuant to Regulation (EEC) 2309/93 in respect of an orphan medicinal product, the Community and the Member States shall not, for a period of 10 years, accept another application for a marketing authorisation, nor grant a marketing authorisation or extend an existing marketing authorisation, for the same therapeutic indication, in respect of a similar medicinal product.

>Text following EP vote>

1.

Where a marketing authorisation is granted pursuant to Regulation (EEC) 2309/93 in respect of an orphan medicinal product and without prejudice to the provisions of the law on intellectual property or any other provision of Community law, the Community and the Member States shall not, for a period of 10 years, accept another application for a marketing authorisation, nor grant a marketing authorisation or extend an existing marketing authorisation, for the same therapeutic indication, in respect of a similar medicinal product.

(Amendment 18)

Article 8(2a) (new)

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2a. On a proposal from the Commission and in cooperation with the European Agency for the Evaluation of Medicinal Products and the interested parties, an Orphan Medicinal Product Innovation Promotion Fund shall be set up, to be administered by the Agency. The fund shall be set up by means of the proceeds from the sale of orphan medicinal products following the expiry of the ten-year period of market exclusivity referred to in paragraph 1. The running of the fund shall be supervised by the Commission. The revenue obtained shall be used in a similar way to the budget headings relating to EU investment programmes.

(Amendment 19)

Article 8(5)

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5. For the purpose of this Article, a 'similar medicinal product' means one which consists of:

- the same chemical active substance or active moiety of the substance, including isomers and mixture of isomers, complexes, esters, other non-covalent derivatives, provided that the pharmacological and toxicological activities of the latter are qualitatively and quantitatively identical to those of the original product,

>Text following EP vote>

5. The Commission shall adopt definitions of 'similar medicinal product' and of 'clinical superiority' in the form of an implementing Regulation, in accordance with the procedure laid down in Article 72 of Regulation (EEC) No 2309/93. The Commission shall also, in consultation with the Member States, the Agency and interested parties, draw up detailed guidance for the application of this Article and the implementing Regulation.

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- a substance with the same biological activity (including those that differ from the original substance in molecular structure, source material and/or manufacturing process) provided that the pharmacological activity of said substance is qualitatively and quantitatively identical to that of the original product,

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- a substance with the same radiopharmaceutical activity (including those with a different radionuclide, ligand, site of labelling or molecule-radionuclide coupling mechanism) provided that its diagnostic or therapeutic indications are identical to those of the original product.

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(Amendment 20)

Article 8(6)

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6. The Commission shall, in consultation with the Member States, the Agency and interested parties, draw up detailed guidance for the application of this Article.

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Deleted.

(Amendment 21)

Article 9(1)

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1. Medicinal products designated as orphan medicinal products under the provisions of this Regulation shall be eligible for incentives made available by the Community and by the Member States to support the research, development and availability of orphan medicinal products.

>Text following EP vote>

1.

Medicinal products designated as orphan medicinal products under the provisions of this Regulation shall be eligible for incentives made available by the Community and by the Member States to support the research, development and availability of orphan medicinal products and in particular aid for research for SMUs provided under the Fifth Framework Programme for Research and Technological Development.

(Amendment 22)

Article 9(4)

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4. Within one year from the adoption of this Regulation, the Commission shall publish a detailed inventory of all incentives made available by the Community and the Member States to support the research, development and availability of orphan medicinal products. This inventory will be updated on a regular basis.

>Text following EP vote>

4.

Within one year from the adoption of this Regulation, the Commission shall publish a detailed inventory of all incentives made available by the Community and the Member States to support the research, development and availability of orphan medicinal products and a series of operational proposals, following the example of other countries which already have legislation governing orphan medicinal products, so that such incentives may be applied uniformly and without unjustified delay throughout the EU.. This inventory will be updated on a regular basis.

(Amendment 40)

Article 11, 1st paragraph a (new)

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An application for designation as an orphan medicinal product submitted to the Agency after 1 April 1999 and before the entry into force of this Regulation shall be dealt with as an application submitted under this Regulation and the resulting decision shall take effect on the date of entry into force of this Regulation. Such an orphan medicinal product may benefit from market exclusivity, provided that the marketing authorisation referred to in Article 8(1) is granted after the entry into force of this Regulation.

Legislative resolution embodying Parliament's opinion on the proposal for a European Parliament and Council Regulation on orphan medicinal products (COM(98)0450 - C4-0470/98 - 98/0240(COD))(Codecision procedure: first reading)

The European Parliament,

- having regard to the Commission proposal to Parliament and the Council, COM(98)0450 - 98/0240 (COD) ((OJ C 276, 4.9.1998, p. 7.)),

- having regard to Articles 189b(2) and 100a of the EC Treaty pursuant to which the Commission submitted the proposal to Parliament (C4-0470/98),

- having regard to Rule 58 of its Rules of Procedure,

- having regard to the report of the Committee on the Environment, Public Health and Consumer Protection and the opinions of the Committee on Budgets, the Committee on Research, Technological Development and Energy and the Committee on Development and Cooperation (A4-0078/99),

1. Approves the Commission proposal, subject to Parliament's amendments;

2. Calls on the Commission to alter its proposal accordingly, pursuant to Article 189a(2) of the EC Treaty;

3. Calls on the Council to incorporate Parliament's amendments in the common position that it adopts in accordance with Article 189b(2) of the EC Treaty;

4. Points out that the Commission is required to submit to Parliament any modification it may intend to make to its proposal as amended by Parliament;

5. Instructs its President to forward this opinion to the Council and Commission.