Mikami Koichi
Soc Hist Med. 2019 Aug;32(3):609-630. doi: 10.1093/shm/hkx098. Epub 2017 Nov 27.
This paper examines the history of orphan drug policy, from the emergence of 'orphans' in the American pharmaceutical market in the 1960s, through the debates and agitations that resulted in the passage of the US Orphan Drug Act of 1983, to attempts in the 1990s to prevent abuse of that Act and restore its original intentions. Although an increased number of drugs for rare diseases have since been developed and marketed, the extremely high price of some such drugs is considered a major public health issue internationally. The present paper traces the origins of this issue to the market-based approach to resolving the problem of orphan drugs embodied in the 1983 Act. The paper also makes visible an alternative trajectory that existed for a while in the United Kingdom but was eventually abandoned in order to help the biotechnology industry grow in the context of an increasingly integrated European drug market.
本文考察了孤儿药政策的历史，从20世纪60年代美国制药市场上“孤儿”的出现，到引发1983年美国《孤儿药法案》通过的辩论和鼓动，再到20世纪90年代防止该法案被滥用并恢复其初衷的尝试。尽管自那时以来，治疗罕见病的药物数量有所增加并投放市场，但一些此类药物极高的价格在国际上被视为一个重大的公共卫生问题。本文将这个问题的根源追溯到1983年法案中体现的以市场为基础解决孤儿药问题的方法。本文还揭示了一条曾在英国存在过一段时间但最终被放弃的替代轨迹，这样做是为了在日益一体化的欧洲药品市场背景下帮助生物技术产业发展。
