Good to know

This section explains the role of Compassionate Use Programmes: who are they for, and the conditions for accessing  a medicine under compassionate use.

 

What is a Compassionate Use Programme?

A medicinal product given on a compassionate basis is first and foremost a treatment. It is a medicinal product not yet fully evaluated, prescribed to treat people with no other therapeutic options.

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What a Compassionate Use Programme isn’t

A Compassionate Use Programme is not an experiment or a clinical trial. It is intended  to treat the person

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Can anyone access medicines on a compassionate basis?

Before a medicine is authorised and placed on the market, it must be duly evaluated to learn more about its efficacy and safety. The first priority is to conduct clinical trials that will respond to these questions. Only patients who cannot be part of a clinical trial are eligible for a compassionate use.

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Are there unlimited supplies of a medicine that can be used for compassionate use?

A company developing a new product first produces necessary quantities of the product for its clinical trials (a few hundred to a few thousands of patients, pilot production). When the product is authorised, the company needs to manufacture enough product to satisfy the demand in a larger population (commercial batches). Between these two stages, the size of a Compassionate Use Programme may vary.

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Main characteristics of CUPs in different EU Member States

See the main characteristics of Compassionate Use Programmes in 7 different EU Member states

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