真实世界数据用于药品医保决策的国内外政策分析  

Policies for use of real-world data in drug reimbursement decision

作　　者：黄涛 李灿[1] 黄先琴 李黄倩玉 吴晶[3] 毛宗福[4,5] 史录文[1,2] 管晓东[1,2] HUANG Tao;LI Can;HUANG Xianqin;LI Huangqianyu;WU Jing;MAO Zongfu;SHI Luwen;GUAN Xiaodong(Pharmacy Administration and Clinical Pharmacy,Peking University,Beijing 100191,P.R.China;International Research Center for Medicinal Administration,Peking University,Beijing 100191,P.R.China;School of Pharmaceutical Science and Technology,Tianjin University,Tianjin 300072,P.R.China;School of Public Health,Wuhan University,Wuhan 430071,P.R.China;Global Health Institute,Wuhan University,Wuhan 430071,P.R.China)

机构地区：[1]北京大学药事管理与临床药学系,北京100191 [2]北京大学医药管理国际研究中心,北京100191 [3]天津大学药物科学与技术学院,天津300072 [4]武汉大学公共卫生学院,武汉430071 [5]武汉大学全球健康研究中心,武汉430071

出　　处：《中国循证医学杂志》2024年第5期510-515,共6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：目的 分析真实世界证据用于医保准入的流程与模式,为开展支持药品医保准入的真实世界研究,完善相关数据及标准体系建设提供参考与借鉴。方法 通过综述国内外主要药品监管机构相关政策法规、指导原则及文献综述,梳理和分析真实世界证据用于医保准入的适用条件、应用范围、框架指南和模式。结果 主要发达国家和部分亚洲国家的卫生经济学评估部门都已采纳真实世界证据用于评估药品的疗效和安全性。真实世界证据用于医保准入的应用范围包括:疾病负担测算、描述疾病治疗过程和药品使用情况、评估药品的有效性和安全性、评估临床随机试验的外推性、评估患者报告结局等指标、验证替代指标与临床终点关系、以及预算影响分析等。包括英国和美国在内的部分发达国家已经发布开展真实世界研究用于医保准入的指南或框架,用于规范纳入药物、研究设计、数据收集和报告结果。目前真实世界证据用于医保准入的模式可分为三种:附条件报销、按效果付费和再评估。结论 当前国内外真实世界数据用于医保准入的经验已较为丰富,迫切需要完善真实世界数据标准和体系,制定相应的药物准入范围和流程,以适应更快更高质量的医药产业发展,满足临床患者需求。Objective To analyze the limitations and challenges for the use of real-world data in the decision making of drug reimbursement through literature review and provide standard process and guideline for the real-world study supporting drug reimbursement.Methods By summarizing the relevant policies,regulations,and guiding principles of major drug regulatory agencies worldwide,the study analyzed the applicable conditions,framework,and reimbursement mode for using real-world evidence in the decision making of drug reimbursement.Results The study found that the health technology assessment departments of major developed countries and Asian countries have used real-world evidence to evaluate the drug efficacy and safety.The application scope of real-world data for reimbursement decision included describing the treatment process of the disease,assessing economic burden,verifying economic models,and evaluating the efficacy and safety of drugs.Some developed countries including the United Kingdom and the United States had released guidelines or frameworks of the real-world study for reimbursement decision.The process and framework of using real-world data in reimbursement decision could be divided into three models:coverage with evidence development,outcome-based contract,and re-assessment.Conclusion Real-world data has been widely used in the process of health technology assessment.To adapt to the development of the pharmaceutical industry and to meet the needs of clinical patients,it is urgent to standardize the process of collecting real-world data and formulate the scope and process of using real-world data in the reimbursement process.

关 键 词：真实世界 医保准入 政策 流程 模式 

分 类 号：R95[医药卫生—药学]