Disease Prevention

Orphan drugs diseases list

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 a defined number of 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.

Follwing orphan designation, efficacy, safety and quality criteria need to be satisfied before the granting of a marketing authorisation.

Over 1000 positive opinions from the COMPs

The COMP has adopted over 1000 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 orphan medcinal product-related matters.

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