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 people, 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 (COMP) at the European Medicines Agency (EMA) is responsible for receiving, approving, and/or 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 EU introduced a new legislation in 2000 with the aim of providing incentives for the development of medicines for rare diseases (so-called orphan medicinal products). To date, the European Commission has already authorised more than 200 orphan medicines for the benefit of patients suffering from rare diseases.
The sponsors responsible for these medicines benefit from incentives such as fee waivers for the regulatory procedures and a 10 year market exclusivity.
Equally important, the European Commission has designated around 2000 products as orphan medicinal products (this number refers to active orphan designations, not withdrawn or expired).
Useful links:
EU Regulation on Orphan Medicinal Products
EU Latest Orphan Medicinal Products