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.
Key words
orphan drugs /
reimbursement access /
real-world data /
Japan
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