The EU challenges on the designation of orphan medicinal products

Article type: Research Article

Authors: Josep Torrent-Farnell, ^; | Rosa Morros,

Affiliations: Committee for Orphan Medicinal Products, European Agency for the Evaluation of Medicinal Products, London, UK | Clinical Pharmacology and Therapeutics Department, Sant Pau Hospital, Autonomous University of Barcelona, Spain

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Abstract: The European Parliament and the Council have recently adopted a legal framework which aims to foster research and promote the development of new therapies for the treatment of rare diseases. It has been well recognised that the US Orphan Drug Act has been a major breakthrough in promoting product development for patients suffering from rare disorders and now after 17 years of experience it is regarded as an essential tool to promote and preserve public health. Several sources indicate that almost 8000 different severe and incapacitating conditions may exist affecting as many as 25--30 million European citizens. Of these disorders about 4000 have a genetic origin affecting primarily neonates and children. The European Agency for the Evaluation of Medicinal Products (EMEA) is responsible for putting in place a community procedure for the evaluation of the applications orphan medial product designation through the creation of a new scientific body, the Committee for Orphan Medicinal Products (COMP). This paper reviews the role and the objectives of the COMP and the EMEA in the development of medicinal products for rare disorders and the key elements for submitting an application for the designation which if successful leads to several incentives.

Journal: Pharmaceuticals, Policy and Law, vol. 3, no. 0, pp. 19-30, 2001