目的：借鉴国际经验,构建我国本土化真实世界数据库分类体系,分析不同类型真实世界数据（RWD）的收集情况并探讨其证据等级,为我国医保准入中RWD的应用提供参考。方法：以英国国家卫生与临床优化研究所（NICE）在2025年5月前通过高度专业化技术评估（HST）的26种罕见病药品为样本,系统分析其在NICE、加拿大药品与健康技术署（CADTH）和美国临床与经济评论研究所（ICER）的医保准入评估报告中RWD的应用情况,进一步构建数据库分类。结合案例分析探讨不同数据库的数据收集情况,并基于特征比较及评估机构考量因素分析,深入讨论医保准入中的真实世界证据（RWE）等级框架。结果：NICE、CADTH和ICER分别有96%、62%和19%的药品使用RWD。研究结合国内外指南,构建了医院信息系统数据、医保支付数据等9类数据库,并遴选了9种典型药品案例,详述其数据收集、整理和组织等关键要素。特征分析显示,不同类型的数据来源在数据结构与质量方面存在显著差异。评分结果显示,医院信息系统、医保支付与官方卫生健康行业数据的证据等级较高;登记研究、自然人群队列、文献研究数据的证据等级中等;患者报告结局数据及专家调研数据的证据等级较低。研究发现,所有类型的数据库均在药品评估中被企业或评估机构使用,但是13种药品中企业使用的16次RWD（涉及5类数据库）由于数据适用性或证据等级较低,被评估机构提出异议。结论：本研究初步构建了我国本土化的真实世界数据库分类体系及其证据等级框架,可为RWD在医保准入中的应用提供参考。未来需进一步完善规范和多方论证,以提升RWD的决策价值。
Abstract
Objective: The paper draws on international experience to establish a localized real-world database classification system, and preliminarily explore the collection status and evidence levels of different types of real-world data (RWD), providing reference for the application of RWD in China's medical insurance access. Methods: Using 26 orphan drugs approved by the National Institute for Health and Care Excellence (NICE) through the Highly Specialised Technology (HST) evaluations before May 2025 as samples, this study systematically analyzed the use of RWD in the health insurance access assessment reports of NICE, the Canadian Agency for Drugs and Technologies in Health (CADTH), and the Institute for Clinical and Economic Review (ICER), and further developed a database classification framework. Case studies were conducted to examine data collection across different databases. Based on characteristic comparisons and the evaluators’ considerations, the study further explored the evidence grading framework for real-world evidence (RWE) in medical insurance access. Results: Among drugs assessed by NICE, CADTH, and ICER, 96%, 62%, and 19%, respectively, incorporated RWD. Guided by both domestic and international frameworks, this study constructed 9 categories of databases, including hospital information system data and health insurance claim data, and selected 9 representative drug cases to detail the key processes of data collection, curation, and organization. Feature analysis indicated substantial differences in data structure and quality across database types. The grading results showed that hospital information system data, health insurance claim data, and official health authority data exhibited high evidence levels; registry databases, general population cohort, and literature-derived data were rated as moderate; while patient-reported outcomes and expert survey data were associated with lower evidence levels. Although all database types were utilized by manufacturers or HTA agencies in drug assessments, the study identified that, for 13 drugs, 16 data sources spanning 5 database categories were questioned by HTA agencies due to limited applicability or low evidence quality. Conclusions: This study preliminarily established a China-specific real-world database classification system and its evidence grading framework, which can provide reference for the application of RWD in medical insurance access. Further standardization and multi-party validation are needed in the future to enhance the decision-making value of RWD.
关键词
真实世界数据 /
真实世界证据 /
医保准入 /
数据库分类 /
证据等级
Key words
real-world data /
real-world evidence /
medical insurance access /
database classification /
evidence level
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