Compassionate use

Compassionate use is a treatment option that allows the use of an unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials.

Agency Role

The European Medicines Agency (EMA) provides recommendations through the Committee for Medicinal Products for Human Use (CHMP), but these do not create a legal framework. Compassionate use programmes are coordinated and implemented by Member States, which set their own rules and procedures.

These programmes are only put in place if the medicine is expected to help patients with life-threatening, long-lasting or seriously debilitating illnesses, which cannot be treated satisfactorily with any currently authorised medicine.

The medicine must be undergoing clinical trials or have entered the marketing-authorisation application process and while early studies will generally have been completed, its safety profile and dosage guidelines may not be fully established.

How to request an opinion

National competent authorities can ask EMA for an opinion on how to administer, distribute and use certain medicines for compassionate use. The CHMP also identifies which patients would benefit, and Member States should take note of these recommendations when making decisions.

Manufacturers and marketing-authorisation applicants should not contact EMA to request an opinion, but they may wish to inform the Agency of applications underway at national level. National competent authorities will inform the Agency if they are making a product available to a group of patients for compassionate use.

Established by Article 83 of Regulation (EC) No 726/2004, this tool is designed to:

• facilitate and improve access to compassionate use programmes by patients in the EU;
• favour a common approach regarding the conditions of use, the conditions for distribution and the patients targeted for the compassionate use of unauthorised new medicines;
• increase transparency between Member States in terms of treatment availability.

Comparison to individual basis treatment

Compassionate use should not be confused with ‘named-patient basis’ treatments, which see doctors obtain medicines directly from manufacturers before authorisation. This is done on an individual basis under the direct responsibility of the doctor, and the Agency does not need to be informed.

In general, medicines that are not yet authorised are first made available through clinical trials and patients should always be considered for inclusion in trials before being offered compassionate use programmes.

Compassionate use opinions

The list of compassionate use opinions includes the CHMP’s recommendations on how a medicine should be used, and the type of patient who should be eligible.