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Rare Diseases

Medicinal products for rare diseases are called "orphan drugs". They are used for rare diseases that are life-threatening or severe and for which there are no or only unsatisfactory treatment options so far. Diseases are considered rare if fewer than 5 in 10,000 persons in a country of the EU are affected. In contrast to other medicinal products they can already be administered in the course of the licensing procedure ("compassionate use").