The website of the European Medicines Agency (EMA)

The European Medicines Agency plays a role in defining who can benefit from compassionate use

 

The European Medicines Agency (EMA) can be requested to give an opinion only for products developed by biotechnology processes, for advanced therapy medicinal products, for medicinal products containing a new active substance and indicated for: 1) HIV, 2) cancer, 3) neurodegenerative disorders, 4) diabetes, 5) auto-immune diseases & 6) viral diseases and for orphan medicines.

Any EU Member State can request the opinion of the committee of experts at the EMA, the Committee for Human Medicinal Products (CHMP). Based on the efficacy and safety information available, the CHMP can decide whether or not a compassionate use for a new product undergoing clinical trials is relevant or not, and for which patients.

The CHMP opinion is not binding; there is no obligation for Member States to follow this opinion. The CHMP does not address the organisational part of the Compassionate Use Programme; this is regulated by each Member State.

However, when a Member State authorises a Compassionate Use Programme, it should notify the EMA.

The EMA website contains information on CUPs, including a Q&A document, here:
 

http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000293.jsp&mid=WC0b01ac058007e691

 

Page created: 30/10/2013
Page last updated: 07/11/2014
 
 
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