Abstract
Orphan drugs are medicinal products used for the therapy of rare serious diseases (with prevalence < 50/100.000). Development, preclinical and clinical testing of drugs for rare diseases can be costly, lengthy, and problematic from many reasons.
In order to facilitate timely drug supply for patients with these diseases, orphan drug cathegory was created. Orphan drug designation usually applies for particular disease (or particular subtype or stage of the disease).
In European Union orphan designation is granted by Committee for Orphan Medicinal Products. Currently, more than 1.000 medicinal products have been designated as orphan medicinal products for various disease conditions.
The very aim of orphan drugs is to provide therapy for those patients who suffer from serious, rare conditions, for which there is currently no available satisfactory treatment.