Taking chimeric antigen receptor T-Cell(CAR-T)drugs as an example,this study systematically analyzes the application of real-world evidence(RWE)in the managed access agreements for high-value innovative drugs.It focuses on three aspects:the basic information of the agreements,the requirements for real-world data collection,and the analysis of real-world data and its impact on health insurance access decisions.During the Cancer Drugs Fund(CDF)reimbursement period in the UK,clinical trials for tisagenlecleucel,axicabtagene ciloleucel,and brexucabtagene autoleucel were extended.Real-world studies were conducted using the Systemic Anti-Cancer Therapy(SACT)dataset and the Blueteq system.The RWE obtained helped to validate or update key uncertainties,such as overall survival and proportion of patients receiving intravenous immunoglobin.As a result,tisagenlecleucel(for B-cell acute lymphoblastic leukaemia)and axicabtagene ciloleucel were included in the reimbursement drug list based on clearer evidence of efficacy and economic evaluation outcomes.The UK's experience provides a reference for China to explore the use of RWE to support the reimbursement of innovative drugs.
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