真实世界数据用于药品临床价值评价的关键技术考量  

Key technical considerations for the evaluation of clinical value of drugs using realworld data

作　　者：徐嘉悦 何俏 刘艳梅[1,2] 刘佳利 姚明宏 赵杨 黎国威[4] 黄丽红 舒啸尘 李玲 王雯[1,2] 孙鑫 XU Jiayue;HE Qiao;LIU Yanmei;LIU Jiali;YAO Minghong;ZHAO Yang;LI Guowei;HUANG Lihong;SHU Xiaochen;LI Ling;WANG Wen;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;School of Public Health Nanjing Medical University,Nanjing 211166,P.R.China;Guangdong Second Provincial General Hospital,Jinan University,Guangzhou 510317,P.R.China;Department of Biostatistics,Zhongshan Hospital,Fudan University,Shanghai 200032,P.R.China;Department of Epidemiology and Biostatistics,School of Public Health,Medical College Soochow University,Suzhou 215123,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]南京医科大学公共卫生学院,南京211166 [4]暨南大学附属广东省第二人民医院,广州510317 [5]复旦大学附属中山医院,上海200032 [6]苏州大学公共卫生学院流行病与卫生统计学系,江苏苏州215123

出　　处：《中国循证医学杂志》2024年第5期523-528,共6页Chinese Journal of Evidence-based Medicine

基　　金：广东省重点领域研发计划资助(编号:2021B0101420005);国家自然科学基金项目(编号:72304198);四川省中央引导地方科技发展专项(编号:2022ZYD0127);四川大学华西医院学科卓越发展1·3·5工程项目(编号:ZYGD23004)。

摘　　要：临床价值评价是药品临床综合评价的核心,是支持药品供应保障决策的重要环节。真实世界数据能反映实际诊疗过程,是评估药品实际临床需求、评价药品有效性和安全性证据的重要数据来源。本技术规范明确了真实世界数据用于药品临床价值评价的适用范围,以及开展价值评价研究的关键考量:包括适用于真实世界数据研究的药品临床价值评价维度,和如何有效利用真实世界数据进行评价,并针对研究设计和统计分析提出了基于真实世界数据的药品临床价值评价的研究实施要点,以帮助研究者正确认识和规范使用真实世界研究开展药品临床价值评价。Assessing the clinical value of pharmaceuticals is crucial for comprehensive evaluation in clinical practice and plays a vital role in supporting decision-making for drug supply assurance.Real-world data(RWD)offers valuable insights into the actual diagnosis and treatment processes,serving as a significant data source for evaluating the clinical demand,effectiveness,and safety of drugs.This technical guidance aims to elucidate the scope of application of RWD for the clinical value assessment of pharmaceuticals,as well as the key considerations for conducting value assessment research.These considerations include identifying the dimensions of clinical value that necessitate RWD and effectively utilizing RWD for evaluation purposes.Additionally,this guidance provides essential points for implementing pharmaceutical clinical value assessment based on real-world data,with a specific focus on study design and statistical analysis.By doing so,this guidance assists researchers in accurately comprehending and standardizing the utilization of real-world research in conducting pharmaceutical clinical research.

关 键 词：真实世界数据 临床价值评价 医保决策 技术规范 

分 类 号：R95[医药卫生—药学]