Synonym(s)
orphan drug; orphan drugs
DefinitionThis section has been translated automatically.
Drugs for the treatment of rare diseases (orphan diseases). More than 5000 such diseases have been identified to date. The EC Regulation on orphan medicinal products came into force on 22.01.2000. This regulation contains the following regulations, among others:
- Granting of orphan medicinal product status
- Rare disease status can be based on both epidemiological (< 5 patients under 10,000 in the EU) and economic criteria (no chance of recovering development costs).
- An application for orphan status can be submitted at any stage of the development of such a drug prior to application for marketing authorisation.
General informationThis section has been translated automatically.
- Furthermore, the granting of "orphan" status is also associated with a complete or partial exemption from fees charged by the EMA, e.g. for advice on development and for the processing of applications for authorisation and amendments (since 2006 this has only applied to small and medium-sized companies).
- A specially appointed committee, the Committee for Orphan Medicinal Products (COMP), prepares an opinion on submitted applications for the granting of "orphan" status within 90 days, on which the Commission decides within 30 days of receipt at the latest.
- The subsequent marketing authorisation, including the assessment of quality, efficacy and safety, will be granted later, as for other medicinal products, in a centralised procedure by the Committee for Medicinal Products for Human Use (CHMP) at the EMA with subsequent confirmation by the European Commission.
- Since September 2005 there is now also a legal basis for "Compassionate use" programmes in Germany (AMG). This enables patients who cannot be included in clinical studies to have access to drugs that have not yet been approved. The prerequisite for this is the existence of tolerability and efficacy studies.
Prof. Dr. med. Peter Altmeyer