Compassionate use of medicinal products in Europe: current status and perspectives.

The medical treatment of patients with chronic, life-threatening or seriously disabling diseases can be very disappointing both for the suffering patient and their physicians in cases where patients cannot be treated satisfactorily with currently authorized medicines. It may be possible to successfully treat such patients with new pharmaceuticals that have not yet been authorized by suitable clinical trials. But clinical trials are time-consuming and costly, and not every patient meets the enrolment criteria for specific clinical trials. An aggravating factor is that the marketing authorization of promising new pharmaceuticals can take several years, which is valuable time lost from the patient's perspective. One way to tackle this problem is to allow seriously-ill patients to obtain the medicines through a " compassionate-use " programme. The term " compassionate use " is defined in Article 83 no. 2 of the Regulation (EC) no. 726/2004 of the European Parliament and of the Council 1 as " … making a medicinal product belonging to the categories referred to in Article 3 (1) and (2) available for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily by an authorized medicinal product. The medicinal product concerned must either be the subject of an application for a marketing authorization in accordance with Article 6 of this Regulation or must be undergoing clinical trials. " This regulatory framework was created by the European Legislature in 2004. Until then, France and Italy were the only two European countries with compassionate-use programmes incorporated into national law. However, the European framework does not include any binding process regulations for countries on how to introduce and implement their own compassionate-use programmes. At present, there is only a nonbinding guideline by the Committee for Medicinal Products for Human Use (CHMP), 2 the scientific committee of the European Medicines Agency (EMA). Member States should notify the EMA when they make use of the compassionate-use provision outlined in paragraph 1 of Article 83 of the Regulation (EC) no. 726/2004. The CHMP, after consulting the manufacturer or the applicant, may adopt opinions on the conditions for use and distribution and the patients targeted for compassionate use in a given therapeutic indication. The opinions will be updated on a regular basis. In January 2010, the CHMP published its first opinions on two compassionate-use programmes in the European Union. …