Division of Dermatology, Department of Medicine, University of British Columbia, 835 West 10th Ave, Vancouver, BC, Canada.
Health Policy. 2010 May;95(2-3):216-28. doi: 10.1016/j.healthpol.2009.12.001. Epub 2009 Dec 29.
This study investigates issues associated with the United States Orphan Drug Act.
A comprehensive orphan drug database was compiled from FDA data and corporate annual reports of major pharmaceutical companies. Analysis allowed the generation of a descriptive orphan drug portrait as well as documentation of orphan drugs along their lifecycle.
Currently, 2002 products have obtained orphan drug designation with 352 drugs obtaining FDA approval. Approximately 33% of orphan drugs are oncology products. On average, products obtain 1.7 orphan designations with approximately 70% obtaining a single designation. At least 9% of orphan drugs have reached blockbuster status with two-thirds having two or more designations. An additional 25 orphan drugs had sales exceeding US$ 100 million in 2008 alone. Since 1983, at least 14 previously discontinued products have been recycled as orphan drugs.
The United States Orphan Drug Act has created issues which, in some cases, have led to commercial and ethical abuses. Orphan Drug Act reform is necessary but current incentives, including 7 year market exclusivity, should be maintained in order to favour patients as well as economic prosperity. Suggested reforms include price regulation, subsidy paybacks for profitable drugs and the establishment of an International Orphan Drug Office.
本研究调查了与美国孤儿药法案相关的问题。
从 FDA 数据和主要制药公司的公司年度报告中编制了一个全面的孤儿药数据库。分析允许生成孤儿药的描述性画像，并记录孤儿药在其生命周期中的情况。
目前，已有 2002 种产品获得了孤儿药指定，其中 352 种药物获得了 FDA 批准。大约 33%的孤儿药是肿瘤产品。平均而言，产品获得 1.7 项孤儿药指定，其中约 70%获得单一指定。至少有 9%的孤儿药达到了重磅炸弹药物的地位，其中三分之二有两个或更多的指定。2008 年，仅 25 种孤儿药的销售额就超过了 10 亿美元。自 1983 年以来，至少有 14 种以前停产的产品已被重新用作孤儿药。
美国孤儿药法案已经产生了一些问题，在某些情况下，这些问题已经导致了商业和伦理上的滥用。孤儿药法案的改革是必要的，但目前的激励措施，包括 7 年的市场独占权，应该保留，以有利于患者和经济繁荣。建议的改革包括价格监管、对盈利药物的补贴回报以及建立一个国际孤儿药办公室。
