目的:以日本孤儿药医保准入为例，探讨我国在孤儿药医保准入中应用真实世界数据（Real-World Data, RWD）的方法和路径，以期为我国构建科学、高效的孤儿药医保准入路径提供政策启示。方法:系统梳理RWD在我国罕见病药品医保准入中的应用现状及应用难点，分析与总结日本孤儿药准入中应用RWD相关的指南与规范文件、应用案例。结果:日本已形成体系化的RWD应用路径。在应用层面，RWD主要用于经济性评估，包括海外上市后监测数据、医保数据库及海外观察性研究数据，但其应用仍面临人群代表性等挑战。在规范层面，日本建立了严谨的治理框架，强调研究设计的科学性、数据源的可靠性验证以及数据处理全过程的可追溯性。结论:构建统一的RWD平台，推动数据资源整合与标准化;在孤儿药领域，特别注重评估RWD的适用性，包括数据源的代表性和关键变量的覆盖度;完善数据治理框架，建立覆盖数据全生命周期的可追溯机制，以保障数据源的可靠性与证据生成的规范性。
Abstract
Objective:This paper takes Japan’s reimbursement access for orphan drugs as an example to explore the methods and pathways for applying Real-World Data (RWD) in China’s reimbursement access for orphan drugs, providing policy insights for establishing a scientific and efficient reimbursement access pathway for orphan drugs in China. Methods:This study systematically reviewed the current application status and application difficulties of RWD in the medical insurance access of rare disease drugs in China, analyzed and summarized the guidelines and regulatory documents, as well as application cases related to the use of RWD in the access of orphan drugs in Japan. Results:Japan has established a systematic pathway for the application of RWD. At the application level, RWD is mainly used for economic evaluation, including overseas post-marketing surveillance data, healthcare security databases, and overseas observational research data. However, its application still faces challenges such as population representativeness. At the regulatory level, Japan has established a rigorous governance framework, emphasizing the scientific nature of research design, the reliability verification of data sources, and the traceability of the entire data processing process. Conclusion:It is necessary to build a unified RWD platform to promote data resource integration and standardization. In the field of orphan drugs, special attention should be paid to evaluating the applicability of RWD, including the representativeness of data sources and the coverage of key variables. It is also important to improve the data governance framework and establish a traceability mechanism covering the entire lifecycle of data to ensure the reliability of data sources and the normativity of evidence generation.
关键词
孤儿药 /
医保准入 /
真实世界数据 /
日本
Key words
orphan drugs /
reimbursement access /
real-world data /
Japan
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基金
中国药品监督管理研究会2025年度研究课题“数据价值链视角下我国罕见病药物真实世界数据质量监管制度优化研究”（2025-Y-Y-008）。
