Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products

Article 1

Purpose

The purpose of this Regulation is to lay down a Community procedure for the designation of medicinal products as orphan medicinal products and to provide incentives for the research, development and placing on the market of designated orphan medicinal products.

Article 2

Definitions

For the purposes of this Regulation:

(a) ‘medicinal product’ means a medicinal product for human use, as defined in Article 2 of Directive 65/65/EEC;

(b) ‘orphan medicinal product’ means a medicinal product designated as such under the terms and conditions of this Regulation;

(c) ‘sponsor’ means any legal or natural person, established in the Community, seeking to obtain or having obtained the designation of a medicinal product as an orphan medicinal product;

(d) ‘Agency’ means the European Agency for the Evaluation of Medicinal Products.

Article 3

Criteria for designation

A medicinal product shall be designated as an orphan medicinal product if its sponsor can establish:

(a) that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10 thousand persons in the Community when the application is made, or that it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in the Community and that without incentives it is unlikely that the marketing of the medicinal product in the Community would generate sufficient return to justify the necessary investment; and

(b) that there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorised in the Community or, if such method exists, that the medicinal product will be of significant benefit to those affected by that condition.

Article 4

Committee for Orphan Medicinal Products

The task of the Committee shall be:

(a) to examine any application for the designation of a medicinal product as an orphan medicinal product which is submitted to it in accordance with this Regulation;

(b) to advise the Commission on the establishment and development of a policy on orphan medicinal products for the European Union;

(c) to assist the Commission in liaising internationally on matters relating to orphan medicinal products, and in liaising with patient support groups;

(d) to assist the Commission in drawing up detailed guidelines.

Article 5

Procedure for designation and removal from the register

The application shall be accompanied by the following particulars and documents:

(a) name or corporate name and permanent address of the sponsor;

(b) active ingredients of the medicinal product;

(c) proposed therapeutic indication;

(d) justification that the criteria laid down in Article 3(1) are met and a description of the stage of development, including the indications expected.

A designated orphan medicinal product shall be removed from the Community Register of Orphan Medicinal Products:

(a) at the request of the sponsor;

(b) if it is established before the market authorisation is granted that the criteria laid down in Article 3 are no longer met in respect of the medicinal product concerned;

(c) at the end of the period of market exclusivity as laid down in Article 8.

Article 6

Protocol assistance

Article 7

Community marketing authorisation

Article 8

Market exclusivity

By way of derogation from paragraph 1, and without prejudice to intellectual property law or any other provision of Community law, a marketing authorisation may be granted, for the same therapeutic indication, to a similar medicinal product if:

(a) the holder of the marketing authorisation for the original orphan medicinal product has given his consent to the second applicant, or

(b) the holder of the marketing authorisation for the original orphan medicinal product is unable to supply sufficient quantities of the medicinal product, or

(c) the second applicant can establish in the application that the second medicinal product, although similar to the orphan medicinal product already authorised, is safer, more effective or otherwise clinically superior.

Article 9

Other incentives

Article 10

General report

Before 22 January 2006, the Commission shall publish a general report on the experience acquired as a result of the application of this Regulation, together with an account of the public health benefits which have been obtained.

Article 10a

The period laid down in Article 5(6) of Decision 1999/468/EC shall be set at three months.

Article 10b

Exercise of the delegation

Article 11

Entry into force

This Regulation shall enter into force on the day of its publication in the Official Journal of the European Communities.

It shall apply as from the date of adoption of the implementing Regulations provided for in Article 3(2) and Article 8(4).

This Regulation shall be binding in its entirety and directly applicable in all Member States.