New Regulation sets up a committee for orphan drugs

The European Parliament and the Council have now adopted a Regulation on orphan medicinal products, which sets up a new committee to be responsible for its implementation. Orphan drugs are those needed to treat diseases occurring so rarely they are not economically viable for...

The European Parliament and the Council have now adopted a Regulation on orphan medicinal products, which sets up a new committee to be responsible for its implementation.

Orphan drugs are those needed to treat diseases occurring so rarely they are not economically viable for the pharmaceutical industry to develop and bring to market. However, EU policy stipulates that patients suffering from rare conditions should have the right to the same treatment as other patients. For this to happen the pharmaceutical industry's research and development of orphan medicinal products must be stimulated, it says.

'Orphan medicinal products eligible for incentives should be easily and unequivocally identified; it seems most appropriate to achieve this result through the establishment of an open and transparent Community procedure for the designation of potential medicinal products as orphan medicinal products', the EU institutions state.

The EU has funded research on orphan medicinal products in the past under the Fourth RTD Framework Programme's Biomed II programme and funding continues under the Fifth RTD Framework Programme's Quality of Life and Management of Living Resources programme.

This latest Regulation lays down a Community procedure for the designation of such drugs as orphan medicinal products; to provide incentives for their research and development; and to bring them to market.

The legislation defines the criteria for a drug to be labelled 'orphan' and sets up a committee for orphan medicinal products. The committee will be responsible for examining applications for the designation of the label of 'orphan medicinal product' and will advise the Commission on the establishment and development of a policy on orphan medicinal products for the European Union. It will also be called on to assist the Commission on liaising internationally on matters relating to orphan medicinal products and the patient group.

It will be staffed for three years, by a member nominated by each Member State, three members nominated by the Commission to represent patient's organisations, and three members nominated by the Commission on the basis of recommendations from the European Association for the Evaluation of Medicinal Products.

The Regulation also sets down legislation relating to the procedure for designation and removal of an orphan medicinal product from the register, protocol assistance, Community marketing authorisation, market exclusivity and other incentives.

The Commission is now requested to publish a general report on its experiences of the implementation of this Regulation before 22 January 2006.