Medicines for rare diseases

Legal background to the procedure for orphan designation in the EU

The European Medicines Agency plays a central role in the development and authorisation of medicines for rare diseases. These medicines are termed ‘orphan medicines’ in the medical world.

About 30 million people living in the European Union (EU) suffer from a rare disease. Rare diseases are defined as life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in EU. This is equivalent to around 250,000 people or less for each disease.

 

Orphan designation:

The Agency is responsible for reviewing applications from sponsors for the designation of medicines for rare diseases.

 

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