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Orphan medicinal products

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

ACT

European Parliament and Council Regulation (EC) No 141/2000 of 16 December 1999 on orphan medicinal products [See amending act(s)].

SUMMARY

As a result of the high cost of research and development, the pharmaceuticals industry is reluctant to develop medicinal products intended for the treatment of rare conditions, as well as those called 'orphan medicinal products', for which the market is smaller. 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, for example by granting exclusive marketing rights for a ten-year period.

Orphan medicinal product: criteria for designation

A medicinal product must be designated an orphan medicinal product:

  • if it is intended for the diagnosis, prevention or treatment of a condition affecting no more than five per ten thousand persons in the Community;
    if it is intended for treating a serious or debilitating disease and it is unlikely that without incentives marketing it would generate sufficient return to justify the necessary investment.

Committee for Orphan Medicinal Products

A 'Committee for Orphan Medicinal Products', set up within the European Medicines Agency, is responsible for assessing the applications for a medicinal product to be designated an orphan medicinal product and to deliver an opinion to the Commission which makes the decision concerning the designation of a medicinal product as an orphan medicinal product. The Regulation 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'.

Marketing authorisation procedure

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.

Exclusive marketing rights

Where marketing authorisation is granted in respect of an orphan medicinal product, the latter will enjoy, with certain exceptions and limitations, 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.

Other incentives

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. 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.

Committee

The Commission shall be assisted by the Permanent Committee for Medicinal Products for Human Use established by Directive 2001/83/EC.

REFERENCES

ActEntry into force - Date of expiryDeadline for transposition in the Member StatesOfficial Journal

Regulation EC No 141/2000

22.1.2000

-

OJ L 18 of 22.1.2000

Amending act(s)Entry into force - Date of expiryDeadline for transposition in the Member StatesOfficial Journal

Regulation EC No 596/2009

7.8.2009

-

OJ L 188 of 18.7.2009

The successive amendments and corrections to Regulation (EC) No 141/2000 have been incorporated in the original text. This consolidated version is of documentary value only.

RELATED ACTS

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.

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].

Last updated: 15.11.2010
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