加快再生医学疗法研发和上市的注册机制研究

Overview of Expedited Regulatory Programs for Regenerative Medicine Therapies in US FDA

美国FDA针对所有治疗严重疾病的化学药品和生物制品制定了4种加快注册政策:快速通道资格、突破性疗法资格、优先审评资格、加速批准。除了适用于以上加速政策外,为促进再生医学疗法的研发和加快审评,以解决严重疾病患者未满足的医疗需求,FDA专门为治疗严重疾病的再生医学疗法制定了一种加快政策再生医学先进疗法认定。本文将主要介绍美国针对再生医学疗法的5种加快注册的激励政策,尤其是再生医学先进疗法认定。并且对我国新修订《药品注册管理办法》修订前后的加快药品注册程序进行比较。

The US FDA has launched four expedited regulatory programs for serious conditions for chemical drugs and biologics,including fast track designation,breakthrough therapy designation,priority review designation,and accelerated approval.In addition to these expedited programs,to facilitate development and regulatory review of regenerative medicine therapies intended to address unmet medical needs in patients with serious conditions,FDA introduced“regenerative medicine advanced therapy”(RMAT)designation for regenerative medicine therapies.This article mainly introduces five expedited programs for regenerative medicine therapies,particularly the RMAT designation,and compares these programs with those in China in place before and after issuance of the newly-revised Provisions for Drug Registration.