论美国《孤儿药法案》及其启示

The U.S.Orphan Drug Act and its enlightenment

1983年美国颁布实施的《孤儿药法案》是世界上第一部专门针对孤儿药研发问题的立法。该法案在患者组织的强力助推之下出台,设计了以孤儿药身份认定为基础的激励机制,基本达成国会立法目的,并被学界视为美国卫生法律与政策领域最为成功的立法实践之一。我国关于孤儿药研发立法相对滞后,有必要梳理和借鉴相关经验。

The Orphan Drug Act in 1983 marked the first piece of legislation in the world addressing issues of orphan drug RD.Substantially affected and driven by patient advocacy groups during its formulation,the Act offered various incentives predicated on orphan drug designation.While by and large achieving its legislative purpose,the Act was recognized by academics as one of the most successful legislations of the U.S.The uncomfortable lagging behind in orphan drug legislation in China has made it necessary to understand and learn the experience herein.