Orphan Medicinal Products

Medicines for rare diseases

Rare diseases are life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. The European Medicines Agency (EMA) is responsible for reviewing applications from sponsors for the designation of medicines for rare diseases ('orphan medicines'). The application is evaluated at EU level by the Committee for Orphan Medicinal Products (COMP). The Medicines Authority is involved at a European Level through the nomination of professionals on the CHMP and the COMP. More information on medicines for rare diseases can be found on the EMA website at this link.

Patients who are encountering problems with the availability in Malta of medicines for a rare disease may contact the Medicines Authority for support.