Definition of orphan drug by the EMA
Автор: Endo-ERN
Загружено: 2025-07-10
Просмотров: 21
Описание:
Armando Magrelli, Istituto Superiore di Sanità (ISS) Roma, Italy
The so-called ‘orphan drugs’ are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions.
The process from the discovery of a new molecule to its marketing is long (10 years in average), expensive (several tens of millions of euros) and very uncertain (among ten molecules tested, only one may have a therapeutic effect). Developing a drug intended to treat a rare disease does not allow the recovery of the capital invested for its research.
About 30 million people living in the European Union (EU) suffer from a rare disease. The European Medicines Agency (EMA) plays a central role in facilitating the development and authorisation of medicines for rare diseases, which are termed ‘orphan medicines’ in the medical world.
The Orphan regulation was established to address this unmet need and to foster the research on the rare diseases.
The orphan status is assigned via COMP opinion to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine so that it can benefit from incentives such as protection from competition once on the market.
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