临床试验加载药物经济学评价专家共识(2024版)  

Expert consensus on pharmacoeconomic evaluations alongside clinical trials(2024 edition)

作　　者：中国药学会药物经济学专业委员会 吴晶[2] 刘国恩[3] 吴斌[4] 王海银 方宇[6] 李洪超[7] 张田甜[8] 金雪晶 官海静 胡明[11] 贺小宁 郭武栋 席晓宇[7] 黄卫东[14] 韩晟[15] 谢诗桐 Pharmacoeconomics Committee of the Chinese Pharmaceutical Association;Wu Jing;Liu Guoen(不详;School of Pharmaceutical Science and Technology,Faculty of Medicine,Tianjin University,Tianjin 300072,China;Institute for Global Health and Development,National School of Development,Peking University,Beijing 100091,China)

机构地区：[1]不详 [2]天津大学医学部药物科学与技术学院,天津300072 [3]北京大学全球健康发展研究院,北京100091 [4]上海交通大学医学院附属胸科医院药剂科 [5]上海卫生和健康发展研究中心 [6]西安交通大学药学院 [7]中国药科大学国际医药商学院 [8]暨南大学药学院 [9]北京中医药大学中医学院 [10]首都医科大学附属北京天坛医院药学部 [11]四川大学华西药学院 [12]天津大学医学部药物科学与技术学院 [13]国家卫生健康委卫生发展研究中心 [14]哈尔滨医科大学卫生管理学院 [15]北京大学医药管理国际研究中心

出　　处：《中华医学杂志》2024年第40期3736-3744,共9页National Medical Journal of China

摘　　要：药物经济学评价作为指导医药资源优化配置的技术工具,其证据已被逐步应用到国家基本医疗保险药品目录调整等国家医疗卫生决策中。随着药品上市、医保准入等决策进度不断加快,医疗卫生决策对药物经济学评价的需求也不断提升,在药品临床试验期间加载药物经济学评价设计,已成为重要研究方向与循证证据来源。为了提升此研究领域的规范性,本共识从中国医疗卫生体系与决策环境出发,围绕如何在临床试验中加载药物经济学评价这一主题,形成涵盖研究设计、数据收集与管理、数据分析以及方法与结果报告全流程的推荐意见。共识内容从目标人群、对照的选择、样本量的计算、医疗资源使用数据的收集、健康相关生命质量数据的收集、成本的分析、健康结果的分析、缺失和删失数据的处理等18个方面,形成24个方法学与实践操作的核心推荐意见,以期为相关领域研究者和决策者提供参考。As a technical tool to guide the optimal allocation of healthcare resources,pharmacoeconomic evaluation has been gradually applied to national healthcare decisions,including the adjustment of the National Reimbursement Drug List in China.With the acceleration of drug market approval and access to insurance coverage,the demand for pharmacoeconomic evaluation in healthcare decision‑making is also increasing.Designing a pharmacoeconomic evaluation alongside clinical trials has become an important research direction and the source of evidence.In order to facilitate the standardization of this research field,the current consensus focuses on the topic of how to conduct a pharmacoeconomic evaluation alongside clinical trials from the Chinese healthcare system and decision‑making context.The consensus group reconciled expert opinions and developed recommendations covering the entire process of study design,data collection and management,data analysis,and methods and results reporting.A total of 24 recommendations regarding to methodology and practice were developed from eighteen aspects including target population,selection of comparators,calculation of sample size,data collection of healthcare resource utilization,data collection of health‑related quality of life,cost analysis,health outcome analysis,addressing the missing and censored data,etc.,with the aim to provide reference for scholars and decision makers in this research filed.

关 键 词：临床试验 药物经济学评价 专家共识 

分 类 号：TP3[自动化与计算机技术—计算机科学与技术]