基本医疗保障制度下完善我国罕见病高价值治疗药物医保准入的思路探讨  

Discussion on improving the medical insurance access of high-value orphan drugs in China under the basic medical insurance system

作　　者：朱琳 胡嘉浩 刘宇晗 李芬[1,2] 金春林 ZHU Lin;HU Jiahao;LIU Yuhan;LI Fen;JIN Chunlin(Shanghai Health Development Research Center(Shanghai Medical Information Center),Shanghai 200040,China;Financial Management Center of Shanghai Municipal Health Commission,Shanghai 200040,China)

机构地区：[1]上海市卫生和健康发展研究中心(上海市医学科学技术情报研究所),上海200040 [2]上海市卫生健康委员会财务管理中心,上海200040

出　　处：《世界临床药物》2023年第9期994-1000,共7页World Clinical Drug

基　　金：上海市药品监测与临床综合评价专项(23Y07001)。

摘　　要：罕见病高价值治疗药物能填补临床空白,较标准治疗方案显著改善患者症状和生活质量,但因在患者数量规模、真实世界疗效、安全性表现及基金预算影响等方面存在不确定性,使医保部门决策风险的不确定性极大,叠加高价因素导致其医保准入难度不断增加。由于罕见病的特殊性,按照常规药物的遴选评价原则及采取“严准入、宽续约”思路进行目录管理存在一定局限性。从国际经验来看,罕见病治疗药物的医保准入需要差异化的评估体系。文章在分析罕见病高价值治疗药物医保准入的困境及成因基础上,借鉴国际经验并结合国内医保准入实践,提出完善我国罕见病高价值治疗药物医保准入政策的若干思路,以期为有关部门调整罕见病医疗保障思路和创新遴选评价机制提供参考,进一步提升罕见病高价值治疗药物的患者可及性。High-value orphan drugs for rare diseases can fll the clinical therapeutic gaps and significantly improve symptoms and patients'quality of life compared with standard treatment regimens.However,due to uncertainties in the number of patients,real-world efficacy and safety performance of drug,and fund budget impact and other factors,the decision-making risk of the medical insurance department is extremely uncertain.The high price also leads to the increasing difficulty of medical insurance access.Due to the particularity of rare diseases,there are certain limitations in drug list adjustments using the same selection and evaluation principle of conventional drugs and following the track of"strict access,loose extension"for high-value orphan drugs.According to the international experience,the medical insurance access of rare disease drugs needs a differentiated evaluation system.This article explored the dilemma of medical insurance access for high-value orphan drugs and analyzes the causes.Drawing on international experiences and combining with domestic medical insurance access practice,we put forward some ideas to improve the medical insurance access for high-value orphan drugs in China,in order to provide references for relevant department to make adjustments on the medical security for rare diseases and innovate the selection and evaluation mechanism,and further improve the accessibility of high-value orphan drugs for patients with rare diseases.

关 键 词：医疗保障制度 罕见病 医保准入 药物遴选 价值评估 

分 类 号：R95[医药卫生—药学]