Affiliations: European Medicines Agency, London, UK. E-mail:
Abstract: The orphan designation procedure relates to medicinal products for
human use aimed at the treatment, prevention or diagnosis of diseases. It is a
free of charge procedure and can be requested at any stage of the development
by a sponsor who can be either a company or an individual, as long as is
established in EU, Iceland, Liechtenstein, and Norway. Once the Committee for
Orphan Medicinal Products (COMP) adopts opinion on designation, the European
Commission grants a decision that gives access to the incentives.