美国罕用药物法案及对中国的启示

A review of the Orphan Drug Act in the United States and its implications for China

美国罕用药物法案是世界上第一个鼓励研发罕用药物的立法。本文分析了该法案自1983年颁布以来的三次重要修订及其建立的一整套针对罕用药物研发、生产和销售的激励体系,探讨了法案在推动罕用药研制所取得的成功和对生物制药产业发展产生的影响,剖析了长期垄断和药物重新定位所带来的高价罕用药隐患。最后,通过借鉴美国的经验结合中国的实际情况提出建立一个有效的罕用药物立法,以改善中国罕见疾病患者健康状况和促进中国生物医药产业发展。

The United States is the first country that has introduced legislation designed to stimulate orphan drug research and development .In this paper , we analyze three major amendments of the Orphan Drug Act ( ODA) since 1983 and its incentive system .We then discuss the success of the ODA in encouraging orphan drug research and development and boosting the biopharmaceutical industry .Following that , we investigate the high price of orphan drugs caused by market exclusivity and drug repositioning .Finally, we draw experience from the ODA to make some suggestions on how orphan drug legislation can be devised in China with the aim of improving the health of rare -dis-ease patients and facilitating the development of Chinese biopharmaceutical industry .