真实世界数据用于药品经济价值评价的关键技术研究  

Research on techniques of economic evaluations of drugs by using the real-world data

作　　者：吴晶[1,2] 孙鑫[3,4] 张嘉会 谢诗桐 贺小宁 吴斌[5] 管晓东 任燕[3,4] 郭武栋[8] 孙利华[9] 赵琨 胡善联 WU Jing;SUN Xin;ZHANG Jiahui;XIE Shitong;HE Xiaoning;WU Bin;GUAN Xiaodong;REN Yan;GUO Wudong;SUN Lihua;ZHAO Kun;HU Shanlian(School of Pharmaceutical Science and Technology,Tianjin University,Tianjin 300072,P.R.China;Center for Social Science Survey and Data,Tianjin University,Tianjin 300072,P.R.China;Chinese Evidence-based Medicine Center,West China Hospital,Sichuan University,Chengdu 610041,P.R.China;NMPA Key Laboratory for Real World Data Research and Evaluation in Hainan,Chengdu 610041,P.R.China;Clinical Research Unit,Ren Ji Hospital,South Campus,School of Medicine,Shanghai Jiaotong University,Shanghai 200001,P.R.China;Department of Pharmacy Administration and Clinical Pharmacy,School of Pharmaceutical Sciences,Peking University,Beijing 100191,P.R.China;International Research Center for Medicinal Administration,Peking University,Beijing 100191,P.R.China;China National Health Development Research Center,National Health Commission,Beijing 100044,P.R.China;School of Business Administration,Shenyang Pharmaceutical University,Shenyang 110016,P.R.China;Vanke School of Public Health,Tsinghua University,Beijing 100084,P.R.China;China National Health Development Research Center National Health Commission,Beijing 100044,P.R.China;School of Public Health,Fudan University,Shanghai 200032,P.R.China)

机构地区：[1]天津大学药物科学与技术学院,天津300072 [2]天津大学社会科学调查与数据中心,天津300072 [3]四川大学华西医院中国循证医学中心,成都610041 [4]国家药品监督管理局海南真实世界数据研究与评价重点实验室,成都610041 [5]上海交通大学医学院附属仁济医院临床研究中心,上海200001 [6]北京大学药学院药事管理与临床药学系,北京100191 [7]北京大学医药管理国际研究中心,北京100191 [8]国家卫生健康委卫生发展研究中心,北京100044 [9]沈阳药科大学工商管理学院,沈阳110016 [10]清华大学万科公共卫生与健康学院,北京100084 [11]国家卫生健康委药物与卫生技术综合评估中心,北京100044 [12]复旦大学公共卫生学院,上海200032

出　　处：《中国循证医学杂志》2024年第5期529-535,共7页Chinese Journal of Evidence-based Medicine

基　　金：四川省中央引导地方科技发展专项(编号:2022ZYD0127)。

摘　　要：运用经济学工具评估药品的成本和健康获益,筛选出更具性价比的药品和技术,是我国为提高医疗资源配置效率的重大举措。由于严格试验条件和实际临床诊疗环境的固有差异,以基于临床试验数据的经济性评价结果指导相关医疗决策会导致一定的价值偏离风险。近年来,真实世界医疗大数据的发展为更好地反映药品或卫生技术在现实应用环境下的经济性提供了良好的机会,并逐渐成为新的研究热点。同时,实际临床环境的复杂性也对研究者和决策者构建、理解和应用真实世界证据提出了更高的要求。为进一步提高基于真实世界数据支持经济性评价的规范性,推动真实世界证据医疗卫生决策中的科学应用,本研究针对真实世界数据在经济性评价中的适用范围、研究规范以及质量评价原则等关键技术问题,结合国内外相关指导原则及最新研究进展,并综合国内多学科领域专家意见,共同制定本技术规范,旨在为广大研究者和决策者提供参考和指导,并为相关部门制定管理政策提供依据。The application of economic tools to evaluate the cost and health benefits and screen out more costeffective drugs and technologies is an important measure to improve efficiency of medical resource allocation in China.Given the inherent differences between strict clinical trials and clinical routine practice,using trial-based economic evaluations to guide relevant medical decisions may lead to a certain risk of value deviation.Recent development of realworld data provides opportunities to assess the cost-effectiveness of drugs under the practical utilization,and has gradually become a new research hotspot.However,the complexity of the actual clinical environment also puts higher demands on researchers and decision makers to construct,understand and apply real-world evidence.In order to further prompt the normalization of economic evaluation based on real-world data and promote the scientific application of real-world evidence in medical and health decision-making,this project aims at the crucial issues including scope,research design and quality evaluation,to clarify the key considerations on the using of real-world evidence in medical decision-making.Combined with the international guidelines,the latest advancement of relevant research areas and the advice and opinions from multidisciplinary experts,we aim to provide technical references and guidance for researchers and decision makers,and to strengthen the evidence base of management policies.

关 键 词：经济价值 经济性评价 成本-效果分析 真实世界数据 

分 类 号：R95[医药卫生—药学]