Orphan medicinal products

The European Commission has published an inventory of Union and Member State incentives to support research into, and the development and availability of, orphan medicinal products.
To date, the Commission has authorised 117 orphan medicines for the benefit of patients suffering from rare diseases, 82% of which are new active substances. Around 25 of the orphan marketing authorisations are from SMEs. The number of products authorised has grown over the years but remains limited bearing in mind the existence of 5,000 to 8,000 distinct rare diseases, only 1 % of which are currently covered by authorised medicinal products in the EU. The incentives of the orphan drug legislation are therefore essential to facilitate pharmaceutical development. The most frequently authorised medicinal products are treatments for pulmonary arterial hypertension, acute myeloid leukaemia, cystic fibrosis, multiple myeloma and acute or chronic lymphoblastic leukaemia.

This paper also includes information about the European expert group on rare diseases, the EU funded research on rare diseases and examples of national measures to support R&D and the availability of orphan medicinal products (e.g. reduction of taxes, pilot project on joint procurement, direct reimbursement after marketing authorisation).

To download the inventory on the ec.europa.eu website