EU Policies & Directives

Orphan Drugs

An orphan medicinal product is one that is intended for the diagnosis, prevention or treatment of a condition that affects not more than 5 in 10 000 persons, according to the Regulation (EC) No 141/2000 on Orphan Medicinal Products which promotes research and development of medicines to treat rare diseases.

The Committee for Orphan Medicinal Products at the European Medicines Agency (EMA) is responsible for receiving/approving/rejecting orphan drugs’ designation applications.

Markets’ access to Orphan Drugs varies between European Member States as the heterogeneous approaches amongst countries makes patient access to orphan drugs complex.

The Regulation on Orphan Medicinal Products celebrated 10 years of enforcement in 2010. Read the relevant publication from the European Organization for Rare Diseases (EURORDIS) here

“The EU Regulation on Orphan Drugs has played its full role, beyond our expectations of 12 years ago. The Regulation is a powerful instrument to incentivise and channel investments in therapeutic areas where perspectives on return on investment were not sufficient under normal market conditions. Though this is not sufficient to address the huge medical needs rare disease patients are facing, we now know that the drivers for rare disease therapies are the co-existence of patient groups for the disease, of a patient registry and of a European network of researchers or clinicians: it is timely to better articulate policies in drug development with EU research and public health policy,” adds Yann Le Cam, EURORDIS’ Chief Executive and former COMP Vice Chair.

Useful links:

Regulation (EC) No 141/2000
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