基于价值的价格的resmetirom 代谢功能障碍相关的脂肪性肝病
Phuc Le1, Srinivasan Dasarathy2,3, William H Herman4,5
1Center for Value-Based Care Research, Primary Care Institute, Cleveland Clinic, Cleveland, Ohio.
JAMA network open
|June 27, 2025
概括
由于价格高昂,resmetirom是一种新的MASH治疗方法,可能不具有成本效益. 它的价值在很大程度上取决于患者的坚持和代谢功能障碍相关的脂肪肝炎 (MASH) 和纤维化症的长期结果.
科学领域:
- 新型MASH治疗药物的药理经济学.
- 对肝病治疗的卫生技术评估.
背景情况:
- 雷斯米蒂罗姆是FDA批准用于MASH和纤维化 (F2-F3) 的第一个药物.
- 高昂的药物成本对患者获取药物构成潜在障碍.
研究的目的:
- 为了估计与标准护理 (SoC) 相比,resmetirom的终身成本效益.
- 为了确定resmetirom的价格门,将其视为具有成本效益.
主要方法:
- 使用一种基于代理的状态过渡微模拟模型.
- 该模型包含了14种健康状况,包括MASH和纤维化阶段.
- 分析了美国200,000名患有MASH和纤维化 (F2-F3) 的成年人的模拟队列.
主要成果:
- 复合药对每位患者产生了0.26个QALY的增长.
- 增量成本效益比 (ICER) 为140,134美元/QALY.
- 成本效益对停产率敏感;更高的坚持改善了价值.
结论:
- 在100,000美元/QALY支付意愿门下,Resmetirom的成本效益不佳.
- 为了准确确定经济价值,需要长期有效性数据.
- 模型假设,特别是停产率,对研究结果产生重大影响.
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