Orphan designation

This section provides guidance and procedural information on applying for orphan designation for medicines for rare diseases in the European Union (EU), including the incentives available for sponsors developing orphan medicines.

Applications for orphan designation of orphan medicines are reviewed by the European Medicines Agency's Committee for Orphan Medicinal Products (COMP).

To qualify for orphan designation, a medicine must meet one of these criteria:

• It is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people in the EU at the time of submission of the designation application;
• It is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition and without incentives it is unlikely that the revenue after marketing of the medicinal product would cover the investment in its development.

In both cases, there must also be either no satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorised, or, if such a method does exists, the medicine must be of significant benefit to those affected by the condition.

More information

• How to apply for orphan designation
• Questions and answers on applications
• Application guidance documents
• Submission deadlines
• Incentives
• Annual report on development
• Transfers
• Maintenance
• Guidance