Legal Regulation of Access to Unauthorised Medicines for Patients Suffering from Rare Diseases

Indrė Špokienė

Abstract


In the European Union (EU), medicines can be marketed only after they have been authorised. However, some medicines need to be accessible before they have been authorised. This especially applies to medicines, which are expected to benefit severely ill patients who cannot be treated with authorised medicines or who have a disease for which no medicine has been authorised yet. The treatment with unauthorised medicines may be the last chance for patients who suffer from rare diseases and cannot enrol in an ongoing clinical trial or be treated with a potentially life-saving medicine. The aim of this article is to analyse the aspects of the legal regulation of access to unauthorised medicines for such patients. The fact that 27–36 million patients with rare diseases live in the EU and there are approximately 200,000–240,000 of such patients in Lithuania underlines the relevance of the study.

Keywords


legal regulation of unauthorised medicines; investigational drugs; access to medicines; patients suffering from rare diseases; “compassionate use” programmes

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"Societal studies" ISSN online 2029-2244 / ISSN print 2029-2236