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.

Running a Compassionate Use Programme (CUP) consists of making a medicinal product available for compassionate reasons to a group of patients (or sometimes individual patients on a case-by-case basis) with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily by an authorised medicinal product1.

  • The demand is made by the doctor to the pharmaceutical company that is developing the product, and to national authorities who need to accept the compassionate use.
  • The European legislation (for medicines authorised via the centralised procedure) states that the medicinal product concerned must either be the subject of an application for a marketing authorisation or must be undergoing clinical trials2: in other terms, it is not requested that the medicine in question be already authorised somewhere in the world. If the product has been or is currently being  tested in a clinical trial for the disease you have, then your doctor can request a compassionate use authorisation.
  • Most EU Member States have a special scheme to dispense a medicine on a compassionate basis. Many other European countries have similar processes. These processes are more or less complex and time consuming.


Extract of the EMA Q&A document on compassionate use

Extract of the EMA Q&A document on compassionate use

1REGULATION (EC) Nº 726/2004 Title V, article 83.2
 2Ibidem, article 83.2

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