我国罕见病药品保障的研究综述

A Review of Researches on the Guarantee of Rare Disease Drugs in OurCountry

目的梳理当前国内与罕见病药品保障相关的研究,总结研究视角和方向。方法检索2010—2021年有关国内对提升罕见病药品保障水平的研究文献,分析目前的研究到了何种程度,取得什么样的成果,存在什么问题。结果当前我国对于罕见病药品保障的研究聚焦在伦理性、药物经济学、卫生事业管理和市场准入4个方面,对提升罕见病保障水平的必要性、重要性和可行性展开理论和实证的探究。结论我国对于罕见病药品保障的理论研究较为全面,但对于筹资、创新支付等实践层面的探讨仍较为有限。

Objective To sort out the current domestic research related to the protection of rare disease drugs,and summarize the research perspective and direction.Methods Search the domestic research literature on raising the level of drug protection for rare diseases from 2010 to 2021,and analyze the extent of the current research,what results have been achieved,and what problems there are.Results The current research on the protection of rare disease drugs in our country focuses on the four aspects of ethics,pharmacoeconomics,health service management and market access,and has developed theoretical and empirical studies on the necessity,importance and feasibility of raising the level of rare disease protection.Conclusion Our country's theoretical research on the protection of rare disease drugs is relatively comprehensive,but the discussion on practical aspects such as financing and innovative payment is still relatively limited.