Asbury C H
Robert Wood Johnson Foundation, Princeton, NJ 08543-2316.
JAMA. 1991 Feb 20;265(7):893-7.
The 1983 Orphan Drug Act sought to increase market incentives and decrease regulatory barriers for products used to treat rare ("orphan") diseases. Major provisions included market exclusivity, tax credits, and regulatory process clarifications. This analysis compares pre- and post-Act industry and government data to examine changes associated with the law. While industry sponsored 34 marketed and 24 experimental orphan drugs in the 17 years prior to the Act, it has sponsored 39 of 42 marketed orphan products in the 7 years since the Act. An additional 301 experimental products have orphan designation. While 25 of 40 marketed orphan products reportedly had annual sales of less than $1 million, product sales for three conditions are more than $100 million annually. This prompted changes in the law, passed by Congress in 1990, but vetoed. Overall, the law has been associated with an increase in orphan product development. The law's costs and benefits to companies, patients, and the public should be examined if future changes are proposed.
1983年的《孤儿药法案》旨在提高市场激励，并减少用于治疗罕见（“孤儿”）疾病产品的监管障碍。主要条款包括市场独占权、税收抵免和监管流程的明确规定。本分析比较了该法案实施前后行业和政府的数据，以研究与该法律相关的变化。在该法案出台前的17年里，行业资助了34种已上市和24种实验性孤儿药，而自该法案通过后的7年里，行业已资助了42种已上市孤儿产品中的39种。另外还有301种实验性产品获得了孤儿药认定。虽然据报道40种已上市孤儿产品中有25种的年销售额低于100万美元，但有三种疾病的产品年销售额超过1亿美元。这促使国会在1990年通过了一项法律修改，但被否决。总体而言，该法律与孤儿产品开发的增加有关。如果未来提议进行修改，应该对该法律给公司、患者和公众带来的成本和收益进行审查。
