Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-threatening or very serious conditions that affect no more than 5 in 10,000 people in the European Union.
To date, the European Commission has already authorised 168 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has designated 1645 products as orphan medicinal products (this number refers to active orphan designations, not withdrawn or expired). The sponsors developing these products benefit from incentives such as protocol assistance. This assistance should facilitate the development and authorisation of innovative medicines for the benefit of the patients.