EN Official Journal of the European Union CE 88/224

(2004/C 88 E/0228)

WRITTEN QUESTION E-0794/04

by Paul Rübig (PPE-DE) to the Commission

(11 March 2004)

Subject: Regulation (EC) No 141/2000 on orphan medicinal products

In connection with Regulation (EC) No 141/2000 (1) on orphan medicinal products, we request clarification on the following questions:

1. Can a medicinal product continue to be designated as an orphan drug if, after it has been designated as such but before it has received centralised authorisation, other medicinal products containing partly the same and partly different active ingredients are granted national authorisation in several Member States to treat the same condition?

2. Can a medicinal product continue to enjoy orphan drug status if the Committee for Proprietary Medicinal Products (CPMP) has granted approval for its use as a second-line treatment only and the first-choice drug is not authorised in all Member States?

3. Does the registration by one sponsor of an active ingredient for use as a second-line treatment prevent another sponsor from obtaining the designation of the same active ingredient as an orphan drug for use as a first-line treatment?

4. Can a sponsor apply for an active ingredient to be designated as an orphan drug if national authorisation has already been granted to an identical medicinal product manufactured by another pharmaceutical company?

5. Can an active ingredient be designated as an orphan drug for the treatment of a certain condition even if doctors are already using it without authorisation to treat that condition and thereby generate large profits for the sponsor? Can a medicinal product be designated as an orphan drug even if large profits are being generated by the use of this medicinal product in treating a different condition, profits which also contribute to funding the development of drugs to treat rare conditions? Is the requirement laid down in Article 3(1)(a) of Regulation (EC) No 141/2000 met in these cases?

Answer given by Mr Liikanen on behalf of the Commission

(20 April 2004)

The issues raised have been addressed in the Commission Communication of July 2003 (2). Following the first three years of application of the Regulation and in response to a number of requests for interpretation and clarification, the Commission wished to set out its position on certain matters relating to the implementation of the designation and market exclusivity provisions. These interpretations were intended to provide guidance to the European Medicines Evaluation Agency, the Member States, the pharmaceutical industry and other interested parties. Also, several clarifications were provided in order to avoid a departure from the spirit of the Regulation. The Communication, therefore, considers points in relation to Articles 3 (criteria for designation), 5 (procedure for designation and removal from the register), and 7 (Community marketing authorisation) of Regulation (EC) No 141/2000 of the Parliament and of the Council of 16 December 1999 on orphan medicinal products.

1. A medicinal product can continue to be designated as an orphan drug if, ‘after it has been designated as such but before it has received centralised authorisation, other medicinal products containing partly the same and partly different active ingredients are granted national authorisation in several Member States to treat the same condition’. However, Article 5(12) states that: ‘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.’ With reference to Article 5(12)(b), section B.2.3 of the Commission Communication of July 2003 mentioned above, states: ‘The Commission interprets this as meaning that the criteria for orphan designation shall be reviewed before a marketing authorisation is granted’.

2. The second question implies that orphan designation was based on significant benefit. Assessment of significant benefit is for the Committee on Orphan Medicinal Products (COMP). Section A.4 paragraph 5 of the same Commission Communication states: ‘With respect to potential availability of the product to the Community population, a medicinal product that is authorised and available in all Member States may constitute a significant benefit compared with a similar product that is authorised in a limited number of Member States only’.

3. The same active substance can be designated more than once for the same or overlapping therapeutic indications.

4. A sponsor may apply for orphan designation in the situation described in this question. However, the COMP would need to assess whether the medicinal product will be of significant benefit to those affected by that condition. Section A.4 paragraph 5 of the Commission Communication is also relevant here.

5. The objective of Regulation (EC) No 141/2000 is stated in its Article 1: ‘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’. Furthermore, the first recital of the Regulation states: ‘some conditions occur so infrequently that the costs of developing and bringing to the market a medicinal product to diagnose, prevent or treat the condition would not be recovered by the expected sales of the medicinal product; the pharmaceutical industry would be unwilling to develop the medicinal product under normal market conditions; these medicinal products are called orphan.’ The criteria of Article 3(1)(a), to be judged by the COMP, could be met even if a sponsor had made profits in the situations described in this question i.e. profits had been made from unauthorised use of the medicinal product in the condition for which designation is sought or from use of the medicinal product for another condition. Article 1 of the Regulation is clear: incentives are provided for research, development, authorisation and placing on the market of medicinal products.

(1) OJ L 18, 22.1.2000, p. 1.

(2) OJ C 178, 29.7.2003.