Skip to content
Generic filters
Exact matches only
Search in title
Search in content
Search in excerpt

Comprehensive new EURORDIS website section explains Compassionate Use

April 2014

Two hands entwinedEURORDIS is pleased to unveil a comprehensive new website section devoted to Compassionate Use. With information and resources useful to patients, healthcare professionals and industry, this new section explains what Compassionate Use is, who is eligible, how Compassionate Use Programmes work, where to find help, and much more.

Compassionate Use refers to the administration of a medicinal product that has not yet obtained regulatory approval to a patient who is usually critically ill and has no alternative treatment available. In these exceptional cases, the patient is not part of the clinical study for the medicine and cannot wait until the end of the clinical trial period and the evaluation of the marketing authorisation application.

Available in seven languages, the new Compassionate Use website section is part of EURORDIS’ Campaign for Access to Medicines for People with Rare Diseases spearheaded by François Houÿez, the Treatment Information and Access Director & Health Policy Advisor for EURORDIS. The new section has information on how patients and health professionals can request a Compassionate Use treatment, a list of tips for members of the pharmaceutical industry on making products available, and complementary informational resources, including the European Medicines Agency, the National Institutes of Health (USA) and the EudraCT Register database of authorised clinical trials in Europe. There is also a glossary of relevant terms.

The new section provides key information on Compassionate Use Programmes in different EU Member States. In France, for example, some 70% of the authorised orphan medicinal products were available via an ATU (Authorisation for Temporary Use) before marketing authorisation, on average 34 months before the marketing authorisation). Links to the websites of national health authorities are provided.

It is important to remember that Compassionate Use Programmes administer medicines that have not completed testing for safety and efficacy. For severely ill patients who are running out of time, the risk may be one they are willing to take. Compassionate Use can save lives.

Louise Taylor, Communications and Development Writer, EURORDIS