Rare diseases, orphan drugs and their regulation: questions and misconceptions
Rare diseases, orphan drugs and their regulation: questions and misconceptions
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DOI：
10.1038/nrd3275
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发表时间：
2010-12-01
影响因子：
120.1
通讯作者：
Tambuyzer, Erik
中科院分区：
文献类型：
--
作者：
Tambuyzer, Erik
由患者组织推动的将罕见疾病作为健康优先事项的持续宣传努力，为行业开发治疗这些疾病的药物（称为孤儿药）提供了监管和经济激励。这些激励措施于1983年首先在美国引入，后来在日本，欧洲和其他地方引入，使一系列罕见疾病患者的治疗得到了实质性的改善。然而，孤儿药开发的出现也引发了一些问题，从稀有性的定义到孤儿药的定价及其对医疗保健系统的影响。本文就孤儿药开发及其监管方面的一些常见问题和误解提供了行业观点，旨在促进该领域的未来发展。
Sustained advocacy efforts driven by patients' organizations to make rare diseases a health priority have led to regulatory and economic incentives for industry to develop drugs for these diseases, known as orphan drugs. These incentives, enacted in regulations first introduced in the United States in 1983 and later in Japan, Europe and elsewhere, have resulted in substantial improvements in the treatment for patients with a range of rare diseases. However, the advent of orphan drug development has also triggered several questions, from the definition of rarity to the pricing of orphan drugs and their impact on health-care systems. This article provides an industry perspective on some of the common questions and misconceptions related to orphan drug development and its regulation, with the aim of facilitating future progress in the field.