Compassionate use

All medicinal products must be authorised before they can be marketed in the European Union. However, a treatment option for patients in the European Union suffering from a disease for which no satisfactory authorised alternative therapy exists or who cannot enter a clinical trial, may be the use of an unauthorised medicinal product in a compassionate use programme. Compassionate use programmes are intended to facilitate the availability to patients of new treatment options under development. National compassionate use programmes, making medicinal products available either on a named patient basis or to cohorts of patients, are governed by individual Member States legislation.

While compassionate use implementation remains the competence of a Member State, Article 83 of Regulation (EC) No 726/2004 on compassionate use is complementary to national legislations and provides an option for Member States that wish to receive a opinion from the Committee on Human Medicinal Products (CHMP) regarding the conditions for use, for distribution and targeted patients for compassionate use of a specific medicinal product which falls within the scope of Article 83(1) and 83(2).

The objectives of article 83 are to:

• Facilitate and improve the access of patients in the European Union to compassionate use programmes
• Favour a common approach regarding the conditions of use, the conditions for distribution and the patients targeted for the compassionate use of unauthorised new medicinal products,
• Increase transparency between member states in terms of treatment availability.

Related documents:

• Guideline on compassionate use of medicinal products, pursuant to article 83 of regulation (EC) no 726/2004
• Question and Answer document on Compassionate use for centralised medicinal products

Contact details:

compassionateuse@ema.europa.eu

Compassionate use (CU) opinions from the CHMP