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.
Key words
real-world data /
real-world evidence /
medical insurance access /
database classification /
evidence level
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