Finding an effective treatment for a patient suffering from one out of the more than 6,000 distinct rare diseases in the EU can be very challenging. An orphan medicine 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.1
The Committee for Orphan Medicinal Products (COMP) is in charge for assessing applications for designation as an orphan medicinal product. If a medicine reaches the marketing authorization stage, it is re-evaluated by the COMP whether the criteria are still met and the orphan medicine status can be maintained.
Medicinal products designated and authorized as orphan medicinal products receive market protection for 10 years.
1. Rare Diseases – European Commission https://health.ec.europa.eu/european-reference-networks/rare-diseases_en