目的: 探讨真实世界数据（Real-World Data, RWD）在多适应证药物新增适应证医保准入评估中的应用价值,为完善我国多适应证药物医保定价机制提供参考。方法: 系统梳理德国、法国、意大利、日本四国在多适应证药物新增适应证医保准入评估中应用RWD的机制与路径,并分析我国现行政策与RWD应用现状。结果: 四国在RWD应用上路径各异,德国通过RWD补充临床证据,支持附加治疗效益再评价与报销金额调整;法国以RWD校准预期使用量,实现支付标准动态调整;意大利以RWD验证基于结局协议中的治疗响应,支撑适应证层面差异化净支付;日本以真实世界销售数据驱动量价联动式定期调价。我国现行“一通用名一支付标准”模式难以反映不同适应证间的临床价值差异,RWD应用亦以费用监测为主,面临“价值评估维度缺位”与“RWD证据支撑不足”双重困境。结论: 建议建立分适应证价值评估与分类管理机制、完善基于价值评估结果的差异化支付标准调整机制、强化RWD在新增适应证定价中的制度化支撑、推进适应证层面医保精细化管理基础设施建设。
Abstract
Objective: The paper attempts to explore the value of Real-World Data (RWD) in the assessment of medical insurance access for new indications of multi-indication drugs, and to provide guidance for improving China’s medical insurance pricing mechanism for such medicines. Methods: A systematic review was conducted of the mechanisms and approaches used in Germany, France, Italy and Japan for applying RWD in the assessment of medical insurance access for new indications of multi-indication drugs, and an analysis was carried out of China’s current policies and the current status of RWD application. Results: The four countries adopted distinct approaches to the application of RWD: Germany utilises RWD to supplement clinical evidence, supporting the re-evaluation of additional therapeutic benefits and adjustments to reimbursement amounts; France uses RWD to calibrate projected usage volumes, enabling dynamic adjustments to payment standards; Italy employs RWD to validate treatment responses as defined in outcome-based agreements, underpinning differentiated net payments at the indication level; and Japan utilises real-world sales data to drive periodic price adjustments through a volume-price linkage mechanism. China’s current “one generic name, one reimbursement standard” model struggles to reflect differences in clinical value across different indications, whilst the application of RWD remains primarily focused on cost monitoring, facing the dual challenges of “a lack of value assessment dimensions” and “insufficient RWD evidence”. Conclusion: It is recommended to establish a mechanism for indication-specific value assessment and categorised management, to refine the mechanism for adjusting differentiated reimbursement standards based on value assessment outcomes, to strengthen institutional support for the use of RWD in pricing new indications, and to advance the development of infrastructure for refined medical insurance management at the indication level.
关键词
真实世界数据 /
多适应证药物 /
新增适应证 /
医保准入 /
国际经验
Key words
real-world data /
multi-indication drugs /
new indication /
medical insurance access /
experience
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