Centre for Research on Health and Social Care Management, SDA Bocconi School of Management, Bocconi University, Milan, Italy.
Centre for Research on Health and Social Care Management, SDA Bocconi School of Management, Bocconi University, Milan, Italy.
Clin Ther. 2020 May;42(5):830-847. doi: 10.1016/j.clinthera.2020.03.012. Epub 2020 Apr 27.
The aim of this study was to evaluate the cost-effectiveness and net monetary benefit of durvalumab consolidation therapy compared with no consolidation therapy after chemoradiotherapy in patients with stage III non-small cell lung cancer with programmed cell death 1 ligand 1 expression ≥1% from the Italian National Health Service perspective.
We developed a 12-month decision tree combined with a lifetime cohort Markov model in which patients were assigned to receive durvalumab consolidation therapy or active follow-up (Italian standard of care) after chemoradiotherapy to compare cost-effectiveness and net monetary benefit of the two strategies during a 40-year period. Clinical outcomes data were obtained from the respective clinical trials and extrapolated using survival analysis; cost data were derived from Italian official sources and relevant real-world studies. The incremental cost-effectiveness ratio, incremental cost-utility ratio, and incremental net monetary benefit were computed and compared against a 16,372 € per quality-adjusted life-year (QALY) willingness-to-pay threshold. We performed deterministic sensitivity analysis and probabilistic sensitivity analysis to assess how uncertainty affected results; we also performed scenario analyses to compare results under different pricing settings.
In the base-case scenario, during a 40-year period, the total costs for patients treated with durvalumab consolidation therapy and active follow-up were €59,860 and €49,840 respectively; life-years gained were 3.47 and 3.31, respectively; and QALYs gained were 2.73 and 2.50, respectively, with an incremental cost-effectiveness ratio of €62,131 per life-year, an incremental cost-utility ratio of €42,322 per QALY, and an incremental net monetary benefit of €-6,144. We found that durvalumab was cost-effective (incremental net monetary benefit = 0) when a discount of 13% and 30% on its official price was applied, considering all other drugs priced according to official or maximum selling prices, respectively. Results were most sensitive to the progression-free survival rate for durvalumab and active follow-up, health utility in progression-free state, and price of subsequent treatments.
Our analysis indicates that durvalumab consolidation is cost-effective when a discount is applied on its official price. These results suggest that durvalumab may deliver an incremental health benefit with a contained upfront cost during a 40-year period, from the Italian National Health Service perspective, providing added value in a potentially curative care setting.
本研究旨在从意大利国家卫生服务体系的角度，评估与放化疗后无巩固治疗相比，PD-L1 表达≥1%的 III 期非小细胞肺癌患者接受 durvalumab 巩固治疗的成本效益和净货币收益。
我们构建了一个 12 个月的决策树，并结合了一个终生队列 Markov 模型，其中患者在放化疗后被分配接受 durvalumab 巩固治疗或主动随访（意大利标准护理），以比较两种策略在 40 年内的成本效益和净货币收益。临床结局数据来自各自的临床试验，并使用生存分析进行外推；成本数据来自意大利官方来源和相关真实世界研究。计算增量成本效果比、增量成本效用比和增量净货币收益，并与 16372 欧元/QALY 的意愿支付阈值进行比较。我们进行了确定性敏感性分析和概率敏感性分析，以评估不确定性对结果的影响；我们还进行了情景分析，以比较不同定价环境下的结果。
在基线情况下，在 40 年内，接受 durvalumab 巩固治疗和主动随访的患者的总费用分别为 59860 欧元和 49840 欧元；获得的生命年分别为 3.47 年和 3.31 年；获得的 QALYs 分别为 2.73 年和 2.50 年，增量成本效果比为每年 62131 欧元，增量成本效用比为每年 42322 欧元，增量净货币收益为-6144 欧元。我们发现，当对其官方价格进行 13%和 30%的折扣时，durvalumab 具有成本效益（增量净货币收益=0），同时考虑到所有其他按官方或最高销售价格定价的药物。结果对 durvalumab 和主动随访的无进展生存期率、无进展状态下的健康效用以及后续治疗的价格最为敏感。
我们的分析表明，当对 durvalumab 的官方价格进行折扣时，durvalumab 巩固治疗具有成本效益。这些结果表明，从意大利国家卫生服务体系的角度来看，durvalumab 在潜在的可治愈治疗环境中，在 40 年内提供了额外的健康效益，同时控制了前期成本，可能具有成本效益。
