Enzmann H, Lütz J
Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, BRD.
Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2008 May;51(5):500-8. doi: 10.1007/s00103-008-0523-8.
The designation as an orphan medicinal product is required in order to benefit from European Union (EU) incentives for the development of drugs for rare diseases. The European Commission decides on the designation based on the opinion of the Committee for Orphan Medicinal Products within the European Medicines Agency. Designation as an orphan drug is clearly different from marketing authorization. Criteria for the designation as an orphan drug are the low prevalence of the disease (less than 5 in 10 000 inhabitants of the EU), severity of the disease and the expected significant benefit for the patients. Incentives include fee reductions and free scientific advice during development and up to ten years market exclusivity after marketing authorization. This article describes the procedure of orphan drug designation and highlights problems regarding coverage of treatment costs and increasingly individualized medicine.
为了受益于欧盟针对罕见病药物研发的激励措施，需要指定为孤儿药。欧盟委员会根据欧洲药品管理局孤儿药品委员会的意见做出指定决定。指定为孤儿药与上市许可明显不同。指定为孤儿药的标准是疾病的低患病率（欧盟每10000名居民中少于5人患病）、疾病的严重性以及对患者预期的显著益处。激励措施包括研发期间的费用减免和免费科学建议，以及上市许可后长达十年的市场独占权。本文描述了孤儿药指定程序，并强调了治疗费用覆盖范围和日益个体化的药物方面的问题。
