国谈中罕见病用药概览与分析

Overview and analysis of rare disease drugs in the national drug list

近年来我国罕见病用药审评审批提速,国家医保药品目录准入谈判工作全面启动。该文通过对比分析历年国谈目录可知,自2017年至2022年,通过国家谈判纳入医保的罕见病用药从零逐年递增至29个,药品自上市到纳入医保时间明显缩短,罕见病用药可及性显著提升。国谈目录中罕见病治疗药物覆盖病种16个,其中进口企业产品26个(89.6%),国内企业产品3个(10.4%),提示我国罕见病用药的上市数量和医保覆盖数量虽然有所增加,但覆盖病种仍较少,国内药企研发创新能力较为薄弱,建议医保、医疗以及医药继续发挥协同联动作用,以期为我国罕见病用药保障提供可持续动力。

In recent years,the review and approval of rare disease drugs in China has been accelerated,and the negotiation of access to the national medical insurance drug catalogue has been fully launched.Through comparative analysis of the national conversation catalogue over the years,it can be seen that from 2017 to 2022,the number of rare disease drugs included in medical insurance through national negotiations has increased from zero to 29 year by year,the time from listing to inclusion in medical insurance has been significantly shortened,and the accessibility has been significantly improved.The list of rare diseases drugs covered 16 rare disease,including 26(89.6%)imported enterprise products and 3(10.4%)domestic enterprise products.It reveals that although the number of rare disease drugs listed and medical insurance coverage in China have increased,the coverage of rare diseases is still small,and the research and innovation ability of domestic pharmaceutical enterprises is relatively weak.It is suggested that medical insurance,medical treatment and medicine should continue to play a synergistic role to provide sustainable impetus for the guarantee of rare disease drug use in China.