美国孤儿药开发的政策、策略与实践

Policies, Strategies and Practices of Orphan Drug Development in the United States

简述了罕见病及孤儿药的定义和研发意义,重点介绍了美国针对孤儿药开发的现有制度法规、激励政策及管理经验,并以实例分析从研发、注册和上市等角度探讨了美国孤儿药开发的策略,旨在为从事孤儿药开发工作的人士提供参考。

This article briefly introduced the definitions and research significance of rare diseases and orphan drugs. Current US regulations, incentive policies and management experience on orphan drugs were reviewed. Case analyses were provided to discuss strategies in orphan drug development in US from the aspects of RD, registration and commercialization, so as to provide references for orphan drug researchers.