目的: 分析药品医保准入中用药剂量不确定性的类型,提出测算优化路径。方法: 通过政策文件和文献分析,从年治疗费用测算视角对剂量不确定性进行分类,并比较不同国家在剂量假设与证据要求方面的主要做法。结果: 剂量不确定性可归纳为固定剂量递增型、体重/体表面积依赖型、疗程不固定型、适应证/亚组分层型和联合用药减量型五类。国际经验表明,应依据目标人群和真实治疗路径确定剂量假设,并通过情景分析、分层核算及上市后监测实现动态校核。结论: 建议建立分类识别和分层测算规则,强化经济性评估环节的剂量精细化要求,并配套动态校核与支付联动机制,以提升医保准入评估的科学性与可执行性。
Abstract
Objective: The paper analyzes the types of dosing uncertainty in drug medical insurance access and proposes optimized pathways for cost estimation. Methods: Based on policy documents and literature analysis, dosing uncertainty was categorized from the perspective of annual treatment cost estimation, and major practices regarding dosing assumptions and evidence requirements in different countries were compared. Results: Dosing uncertainty can be classified into five types: fixed-dose escalation, weight/body surface area dependence, variable treatment duration, indication/subgroup stratification, and dose reduction due to combination therapy. International experience suggests that dosing assumptions should be determined based on the target population and real treatment pathways, with dynamic validation through scenario analysis, stratified accounting, and post-marketing surveillance. Conclusion: It is recommended to establish rules for type-based identification and stratified estimation, strengthen dosing precision requirements in economic evaluation, and implement dynamic validation linked to payment mechanisms, so as to enhance the scientific rigor and feasibility of medical insurance access assessment.
关键词
医保准入 /
用药剂量 /
年治疗费用 /
测算偏差 /
分类框架
Key words
medical insurance access /
drug dosing /
annual treatment cost /
estimation bias /
classification framework
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参考文献
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基金
国家自然科学基金面上项目“‘时空—政策—经济’三元耦合驱动下国谈抗肿瘤药物可及性跨尺度机制解析与分层干预策略研究”（72574147）;上海市科学技术委员会“上海市创新药械准入效果评估与影响力提升路径研究”（25692113900）;上海市卫生健康委临床研究专项资助项目“人工智能病理用于乳腺癌新辅助治疗的精准管理策略研究”（20254Y0065）
