美国生物制品中孤儿药的研发现状 及对中国孤儿药研发策略的思考

过去30 年, 美国在全球第一部《孤儿药法案》推动和相关政策法规引导下，生物科技飞速发展，尤其在孤儿药研发领域，成果卓著。 对这30 年来美国FDA 批准的生物制品中孤儿药产品作一解析，回顾美国对生物制品中孤儿药的研发策略，介绍孤儿药开发的新型商业 模式，同时反思中国孤儿药研发策略。

Current Status of Biological Orphan Drugs R&D in the US and Reflection on the Development Strategy for Orphan Drugs in China

Over the past three decades, under the impetus of the world''s first "Orphan Drug Act" and the guidance of the related policies and regulations, the US enjoyed rapid development in biotechnology and outstanding achievements in orphan drug development. In this article, the biological orphan drugs approved by FDA in the past 30 years were analyzed, the US strategy for orphan drug development was reviewed, the new business model for orphan drug development was introduced and the Chinese strategy for orphan drug development was reflected.