Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years
Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years
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DOI：
10.1038/nrd3160
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发表时间：
2010-07-01
影响因子：
120.1
通讯作者：
Cote, Timothy R.
中科院分区：
文献类型：
--
作者：
Braun, M. Miles;Farag-El-Massah, Sheiren;Cote, Timothy R.
1983年美国的《孤儿药物法案》刺激了罕见疾病新疗法的发展。为了提供孤儿指定产品及其适应症的第一个全面概述，本文定量分析了1983年至2008年8月美国食品和药物管理局(FDA)孤儿指定和批准的特征和分布。在1892个孤儿指定产品中，326个获得了上市批准，代表了247种不同的药物和200多种不同的疾病。大约一半的批准发生在指定被授予后的4年内。孤儿指定和批准的最常见患者人数不到10,000人，癌症是最常见的疾病领域。讨论了这些发现对未来罕见疾病治疗方法的开发和营销的影响。
The 1983 US Orphan Drug Act has stimulated the development of new therapies for rare diseases. To provide the first comprehensive overview of orphan-designated products and their indications, this article quantitatively analyses the characteristics and distribution of orphan designations and approvals by the US Food and Drug Administration from 1983 to August 2008. Of the 1,892 orphan-designated products, 326 received marketing approval, representing 247 different drugs and more than 200 different diseases. About half of the approvals had occurred by 4 years after designation was granted. The most common patient population size for orphan designations and approvals was fewer than 10,000 patients, and cancer was the most common disease area. The implications of such findings for future development and marketing of therapies for rare diseases are discussed.