目的:探讨美国孤儿药法案的相关问题。方法:以美国FDA和制药企业的年度报告数据为基础,建立综合孤儿药数据库。结果:数据库显示,目前美国FDA一共准许了2002种产品的孤儿药地位,其中获得上市批准的孤儿药产品共计352种,其中大约有33%为抗肿瘤产品。目前,至少有9%的孤儿药已达到重磅药的地位,有43种孤儿药的年销售额(2008)超过了1亿美元,而1983年以来,至少有14种先前停产的产品作为孤儿药重新生产。结论:美国孤儿药法案在某种程度上导致了过高利润和道德问题。其改革虽然必要,但应从病患和市场繁荣角度出发,目前仍需激励,尤其是7年市场独占权应当保留。建议改革立足于价格管制,增加高利润药品税收和创立国际性的孤儿药组织。 Objectives: To explore the issues related to the orphan drug act in the United States. Methods: Based on the annual reports of FDA and pharmaceutical companies in the United States, a comprehensive orphan drug database was established. Results: The database shows that at present, the FDA of the United States of America approved a total of orphans for 2002 products, of which 352 were listed or approved as orphan products, of which about 33% were anti-tumor products. Currently, at least 9% of orphans have reached heavy pound status with 43 orphans having annual sales of more than $ 100 million (2008), and since 1983 at least 14 previously discontinued products have been orphaned Drug re-production. Conclusion: The U.S. Orphan Drug Act has, to some extent, led to excessive profits and moral problems. Although its reform is necessary, it still needs to be stimulated from the viewpoint of patient and market prosperity. In particular, the market exclusive right for seven years should be retained. It is proposed that the reform be based on price controls, increase taxes on high-margin drugs and create an international orphan drug organization.