Finding effective treatment for a patient with one of the 6,000 rare diseases can be very difficult. An orphan medicines is a medicinal product developed for the treatment of a rare disease. The EU’s orphan designation program encourages the development of medicines to help these patients.
Over 1900 orphan medicines with designation
The Committee for Orphan Medicinal Products (COMP) is in charge of reviewing applications for orphan designation. If a medicine makes it to the marketing authorization stage, the COMP will assess it again to check whether the criteria are still met and the orphan designation can be maintained for the authorized medicine.
Over 140 orphan medicines authorized in the EU
Orphan-designated medicines that eventually make it to the market, and for which it can be demonstrated that they maintain the criteria for the orphan designation, are granted 10 years of market protection.