Compassionate Use in Europe

The European Medicines Agency (EMA) defines "compassionate use" as “the use of an unauthorised medicine outside a clinical study in individual patients under strictly controlled conditions” or as “a treatment option that allows the use of an unauthorised medicine”.

Briefly, the European legal basis relating to compassionate use is provided for by:

• Article 5 of the Directive 2001/83/EC, which states that: “A Member State may, in accordance with legislation in force and to fulfil special needs, exclude from the provisions of this Directive [requirement for a marketing authorisation] medicinal products supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised healthcare professional and for use by an individual patient under his/her direct personal responsibility.”
• Article 83 of Regulation (EC) no. 726/2004, which provides, among other things, that: “By way of exemption from Article 6 of Directive 2001/83/EC, Member States may make a medicinal product for human use belonging to the categories referred to in Article 3(1) and 3(2) of Regulation (EC) No 726/2004 available for compassionate use. ”

The implementation of compassionate use remains a responsibility of each Member State, which also need to consider the EMA Guideline on Compassionate Use of Medicinal Products. We are facing a substantial heterogeneity within the EU with regard to requirements and characteristics of compassionate use programmes. 

As an international Life Sciences and Healthcare practice we have put together a comparative guide which outlines the position on compassionate use, including detailed sections on Belgium, France, Germany, Italy, Spain and the United Kingdom.

If you would like to discuss this further, please get in touch with your usual Osborne Clarke contact or one of our experts listed below.