Orphan medicines designation

Orphan Designation

The Committee for Orphan Medicinal Products (COMP) was created as part of Regulation (EC) No 141/2000 (the Orphan Drug Regulation) in order to review applications for an orphan designation.

Medicinal products intended for rare diseases can receive an orphan designation based on the following criteria:

  • The product is intended for an indication with a prevalence not exceeding 5 in 10 000 persons in the EU
  • The disease is life-threatening, seriously debilitating or a serious and chronic condition
  • No satisfactory method of diagnosis, prevention or treatment of the condition is already authorised in the EU. If any method exists, then the medicinal product has to demonstrate that it provides a significant benefit compared to the product for which orphan designation is being requested



The COMP reviews applications and adopts a positive opinion recommending orphan designation when the required criteria are met. The final decision is made by the European Commission.

Orphan designation at any stage of development

Orphan designation is possible at any stage of development prior to application for marketing authorisation. Demonstration of medical plausibility may be pre-clinical (not yet tested on human subjects) or may have reached the clinical trial phase. Following orphan designation, efficacy, safety and quality criteria need to be satisfied before the granting of a marketing authorisation.

See the latest orphan designations and marketing authorisations :link to COMP report and table to be updated each month.

A complete list of designated and authorised orphan medicinal products in Europe is available at ec.europa.eu

Over 1500 positive opinions from the COMP

The COMP has adopted over 1500 positive opinions in favour of orphan medicinal product designation since its inception, demonstrating the effectiveness of Regulation (EC) No 141/2000 and the legal framework encouraging the development of treatments for rare diseases.
The COMP also advises the European Commission on orphan medicinal product policies in the EU and assists in developing related guidelines and liaising internationally on matters related to orphan medicines.

EURORDIS has held two of the three seats assigned to patient organisation representatives on the COMP since its creation in 2000. See the full EURORDIS representation at the COMP.

Page created: 19/08/2009
Page last updated: 22/11/2016
The voice of rare disease patients in EuropeEURORDIS The international voice of people living with rare diseases, Rare Diseases International is a EURORDIS initiativeRare Disease International Bringing together patients, families and experts to share experiences in a moderated multilanguage forum, RareConnect is a EURORDIS initiative RareConnect The Rare Barometer Programme is a EURORDIS initiative that carries out surveys to transform rare disease patients' experiences into figures and facts that can be shared with decision-makers.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Join the largest gathering of rare disease stakeholders across Europe, at the biennial European Conference on Rare Diseases and Orphan Products. ECRD is a EURORDIS initiativeEuropean Conference on Rare Diseases