Rare disease policies to improve care for patients in Europe

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

卷:1852

Article

Rodwell, Charlotte¹  Ayme, Segolene¹ 

[1] INSERM, US Orphanet 14, F-75014 Paris, France

关键词: Rare diseases;    Policy;    Healthcare planning;    Codification;    Registries;    Orphan drugs;   

DOI  :  10.1016/j.bbadis.2015.02.008

来源: Elsevier

【 摘 要 】

Rare diseases are those with a particularly low prevalence; in Europe, diseases are considered to be rare when they affect not more than 5 in 10 000 persons in the European Union. The specificities of rare diseases make the area a veritable public health challenge: the limited number of patients and scarcity of knowledge and expertise single rare diseases out as a distinctive domain of high European added-value. The Orphan Medicinal Product Regulation of 1999 was the first European legislative text concerning rare diseases, followed by many initiatives, including recommendations by the Council of Ministers of the European Union in 2009. These initiatives contributed to the development of rare diseases policies at European and national level aimed at improving care for patients with rare diseases. A review of the political framework at European level and in European countries is provided to demonstrate how legislation has created a dynamic that is progressively improving care for patients with rare diseases. This article is part of a Special Issue entitled: Current Research on the Neuronal Ceroid Lipofuscinoses (Batten Disease). (C) 2015 The Authors. Published by Elsevier B.V.

【 授权许可】

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