Orphan medicinal products

The purpose of this Regulation is to introduce incentives to develop and market medicinal products for the prevention, diagnosis and cure of rare conditions ('orphan medicinal products').

ACT

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

SUMMARY

The high cost of pharmaceutical research and development makes the pharmaceuticals industry reluctant to develop medicinal products intended for the treatment of rare conditions as well as those that are uneconomic ('orphan medicinal products'). The aim of this Regulation is to establish a Community procedure for designating orphan medicinal products and to introduce incentives for orphan medicinal products research, development and marketing, in particular by granting exclusive marketing rights for a ten-year period.

A medicinal product must be designated an orphan medicinal product:

if it is intended for the diagnosis, prevention or treatment of a condition affecting fewer than five per ten thousand persons in the Community;
or
if it is intended for treating a serious or debilitating disease and is unlikely to be marketed without incentives, and it will carry a notable benefit for people affected by such problems.

A 'Committee for Orphan Medicinal Products', set up within the European Medicines Agency , is responsible for delivering an opinion, within 90 days, on applications for a medicinal product to be designated an orphan medicinal product. The Commission takes a decision within 30 days following receipt of the opinion. The proposal gives the sponsor of the medicinal product the possibility of appealing against the Committee's opinion. Medicinal products designated as orphan medicinal products are entered on the 'Community Register of Orphan Medicinal Products'.

Orphan medicinal products are mandatorily subject to the 'centralised' marketing authorisation procedure provided for in Regulation (EC) No 726/2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use. The sponsor of an orphan medicinal product can be exempted from payment of the fees payable to the European Medicines Agency.

Where marketing authorisation is granted in respect of an orphan medicinal product, the latter will enjoy exclusive marketing rights for a ten-year period. However, at the request of a Member State, this period can be reduced to six years if that Member State can establish that the conditions justifying the designation as an orphan medicinal product are no longer met or that the price being charged for the medicinal product in question is excessive.

Moreover, exclusivity does not prevent the marketing of a second medicinal product if the holder of the marketing authorisation of the original orphan medicinal product has given his consent to the second applicant, or if he is unable to supply sufficient quantities of the medicinal product, or if another medicinal product proves safer, more effective or otherwise clinically superior to the first.

Orphan medicinal products can be eligible for further incentives made available by the Commission and the Member States to support the research, development and availability of orphan medicinal products, and more particularly, the measures aid research in favour of small and medium sized enterprises. Member States must inform the Commission of measures taken to this end. The Commission must publish a regular inventory detailing the incentives introduced by the Community and the Member States.

REFERENCES

Act	Entry into force - Date of expiry	Deadline for transposition in the Member States	Official Journal
Regulation EC No 141/2000	22.1.2000	-	OJ L 18 of 22.1.2000

RELATED ACTS

Commission Decision 2006/86/EC of 12 April 2006 appointing members of the Committee for Orphan Medicinal Products (Text with EEA relevance) [Official Journal L 104 of 13.04.2006].

Since the terms of office of the members of the Committee for Orphan Medicinal Products expired on 15 April 2006, two new members were appointed and the terms of office of other members were renewed by this Decision. The members were appointed for a term of three years, from 16 April 2006.

Regulation (EC) No 726/2004 of the European Parliament and of the Council of 31 March 2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency (Text with EEA relevance) [Official Journal L 136 of 30.04.2004].

This Regulation amends, improves and replaces Regulation (EEC) No 2309/93 laying down Community procedures for the authorization and supervision of medicinal products for human and veterinary use and establishing a European Agency for the Evaluation of Medicinal Products. Regulation (EEC) No 2309/93 is therefore repealed.

Commission Regulation (EC) No 847/2000 of 27 April 2000, laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts 'similar medicinal product' and 'clinical superiority' [Official Journal L 103 of 28.04.2000].

Commission communication regarding the nomination of members of the Committee for Orphan Medicinal Products [Official Journal C 110 of 15.04.2000] and [Official Journal C 86 of 16.03.2001].

For more information, please consult the webpage of DG Enterprise and Industry on pharmaceutical products.

Last updated: 3.12.2007