用于药品临床价值和经济价值评价的真实世界数据关键技术考量  

Key considerations for using real-world data to evaluate the clinical and economic value of drugs

作　　者：谭婧[1,2] 熊益权 黄诗尧 任燕 高培[3] 陈英耀 毛琛[5] 彭晓霞[6] 赵绍阳 伍红艳[8] 张田甜[9] 官海静 孙鑫[1,2] TAN Jing;XIONG Yiquan;HUANG Shiyao;REN Yan;GAO Pei;CHEN Yingyao;MAO Chen;PENG Xiaoxia;ZHAO Shaoyang;WU Hongyan;ZHANG Tiantian;GUAN Haijing;SUN Xin(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;Department of Epidemiology and Biostatistics,School of Public Health,Peking University,Beijing 100191,P.R.China;School of Public Health,Fudan University,Shanghai 200433,P.R.China;Department of Epidemiology,School of Public Health,Southern Medical University,Guangzhou 510515,P.R.China;National Center for Children’s Health,China,Clinical Epidemiology and Evidence-based Medicine Center,Beijing Children’s Hospital,Capital Medical University,Beijing 100045,P.R.China;School of economics,Sichuan University,Chengdu 610041,P.R.China;School of Medicine and Health Management,Guizhou Medical University,Guiyang 550000,P.R.China;College of Pharmacy,Jinan University,Guangzhou 510515,P.R.China;Department of Pharmacy,Beijing Tiantan Hospital,Capital Medical University,Beijing 100045,P.R.China)

机构地区：[1]四川大学华西医院中国循证医学中心,成都610041 [2]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 [3]北京大学公共卫生学院流行病与卫生统计学系,北京100191 [4]复旦大学公共卫生学院,上海200433 [5]南方医科大学公共卫生学院流行病学系,广州510515 [6]国家儿童医学中心,首都医科大学附属北京儿童医院临床流行病学与循证医学中心,北京100045 [7]四川大学经济学院,成都610041 [8]贵州医科大学医药卫生管理学院,贵阳550000 [9]暨南大学药学院,广州510515 [10]首都医科大学附属北京天坛医院药学部,北京100045

出　　处：《中国循证医学杂志》2024年第5期516-522,共7页Chinese Journal of Evidence-based Medicine

基　　金：基于多模态融合的医学人工智能集成预测和辅诊建模方法及应用研究(编号:2021B0101420005);国家自然科学基金面上项目(编号:71974138、72174132);四川省中央引导地方科技发展专项(编号:2022ZYD0127)。

摘　　要：随着全球创新药品研发的加速,如何从众多药品中遴选出有效、安全、经济的产品给医保准入和目录动态调整等管理决策带来了新的挑战。真实世界数据为药品临床价值和经济价值评价提供了新的思路,但对于可用的真实世界数据范围、数据标准和证据范畴仍存在一定的疑惑。本文基于国内外真实世界数据支持药品临床价值和经济价值评价的现状,联合全国真实世界数据、药品临床价值和经济价值评价等领域专家,经多轮讨论形成了真实世界数据用于药品临床和经济价值评价的关键技术考量。本文首先明确了可用于药品临床和经济价值评价的真实世界数据范围;其次,针对真实世界数据的数据归属、数据治理和质量标准提出了具体的要求和指导性意见;最后,总结了真实世界数据用于药品临床和经济价值评价等方面的证据范畴。With the acceleration of global innovative drug development,selecting safe,effective,and cost-effective products from numerous drugs has posed new challenges for the decision-making process of medical insurance drug access and dynamic updating of insurance directory.Real-world data(RWD)provides a new perspective for evaluation of clinical and economic value of drugs,but there are still uncertainties regarding the scope,quality standards,and evidence categories of RWD that can be used.Based on the current status of domestic and international RWD supporting the assessment of the clinical and economic value of drugs,this paper,in collaboration with national RWD and healthcare experts,has developed the key considerations for using real-world data to evaluate the clinical and economic value of drugs.This paper first clarifies the scope of RWD that can be used to evaluate the clinical and economic value of drugs evaluate;secondly,provides specific requirements and guidance on data attribution,data governance,and quality standards for RWD;finally,summarizes the evidence categories of RWD supporting evaluate the clinical and economic value of drugs evaluate.

关 键 词：真实世界数据 真实世界证据 药品临床价值 药品经济价值 

分 类 号：R95[医药卫生—药学]