Objective: The paper attempts to analyze the practical experience of the United Kingdom, Canada, and the United States in applying real-world data (RWD) to health insurance access (reimbursement) decision-making, and, in light of China's current policy context, propose a construction pathway for an RWD-driven comprehensive value assessment system for pharmaceuticals to inform the improvement of China's reimbursement policy. Methods: Twenty-six orphan drugs that had been included in the UK National Health Service (NHS) through the Highly Specialised Technologies (HST) evaluations conducted by the National Institute for Health and Care Excellence (NICE) before May 2025 were selected as the study samples. The reimbursement assessment reports issued by NICE, the Canadian Agency for Drugs and Technologies in Health (CADTH), and the US Institute for Clinical and Economic Review (ICER) were analyzed. Combining specific application cases, the study examined the use of RWD from two perspectives—companies (P1) and assessment agencies (P2)—to explore the specific purposes, data sources, and assessment agencies' considerations of RWD in addressing uncertainties related to effectiveness (D1), cost (D2), population and market share (D3). Results: RWD was used in the reimbursement assessments of 96%, 62%, and 19% of the drugs evaluated by NICE, CADTH, and ICER, respectively. Its primary applications were in evaluating four dimensions of pharmaceutical value: economic efficiency, effectiveness, safety, and accessibility. Specifically, RWD was employed to address three main categories of uncertainty: effectiveness uncertainty (D1), cost uncertainty (D2), and population and market share uncertainty (D3) . The main purposes included providing data on disease management costs, patient outcomes, and target populations. RWD was most widely applied in addressing cost (D2) and effectiveness (D1) uncertainties. All types of RWD were used by both companies (P1) and assessment agencies (P2), though some datasets were challenged by assessment agencies due to concerns about their applicability or low evidence level. Conclusion: The application of RWD in reimbursement decision-making has been increasingly adopted by assessment agencies. However, differences remain in the degree of acceptance across different purposes and RWD types. Overall, RWD plays a crucial role in the reimbursement process, yet its application still requires clarification of specific use scenarios, alignment of data-source grading with appropriate purposes, and the establishment of standardized guidelines to enhance scientific rigor and standardization of its application.
Key words
real-world data /
real-world evidence /
medical insurance admission /
comprehensive value assessment of drugs
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