Scientific advice for orphan drugs: Critical points and perspectives

Article type: Research Article

Authors: Garcia, Jordi Llinares

Affiliations: European Medicines Agency, London, UK. E-mail: jordi.llinares@emea.europa.eu

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.

Keywords: Orphan designation, Committee for Orphan Medicinal Products, Protocol Assistance, significant benefit

DOI: 10.3233/PPL-2010-0264

Journal: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 45-48, 2010