摘要
罕见病用药是当前全球临床需求面临的重要问题之一,推动罕见病用药研发是监管方和工业界共同面对的问题。当前我国药品监管机构已出台了诸多政策,在实践中已取得了切实的成果,但我国罕见病药物研发整体起步较晚,在供给侧改革中尚需完善相关制度。本文对当下国内罕见病用药现状以及目前研发注册方面的激励政策进行了总结,结合部分法规中的薄弱环节提出相关建议,为未来我国罕见病用药政策制定提供参考。
Rare disease drugs is one of the most important problems in current global clinical demand.It is a common challenge faced by regulators and industry to promote the research and development(R&D)of rare disease drugs.At present,China's drug regulatory agencies have issued a lot of policies,and have achieved tangible outcomes in practice.However,the R&D of rare disease drugs in China started late as a whole,and the relevant systems still need to be completed in the supply-side reform.This paper summarizes the current situation of drug use for rare diseases and the incentive policy of research and development registration,and relevant suggestions were put forward aiming at the weak points in some regulations,so as to provide reference for the policy making of drug use for rare diseases in the future.
作者
王轩
董凌云
钱莉苹
张颖
朱文涛
WANG Xuan;DONG Lingyun;QIAN Liping;ZHANG Ying;ZHU Wentao(Yifan Pharmaceutical Research Institute(Beijing)Co.,Ltd.,Beijing 100176,China;Yeedozencom(Beijing)Co.,Ltd.,Beijing 100053,China;Hefei Yifan Biopharmaceutical co.,Ltd.,Hefei 230093,China;Beijing University of Chinese Medicine,Beijing 100029,China)
出处
《药学与临床研究》
2023年第3期279-285,共7页
Pharmaceutical and Clinical Research
关键词
罕见病用药
法规建设
政策建议
Rare disease drugs
Regulations construction
Policy advise
