Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
Global Health Outcomes, Bristol-Myers Squibb, Princeton, NJ, USA.
J Med Econ. 2021 Jan-Dec;24(1):79-86. doi: 10.1080/13696998.2020.1857960.
In August 2018, the US FDA granted accelerated approval for nivolumab in small cell lung cancer (SCLC) that has progressed after platinum-based chemotherapy and at least one other line of therapy. The objective of this study was to evaluate the cost-effectiveness of nivolumab vs. usual care as third-line (3 L) therapy for patients with recurrent SCLC (rSCLC) from the health payer perspective. Given the potential for a meaningful fraction of treated patients to achieve long-term response to nivolumab, we also assessed the impact of using mixture cure modeling (MCM) vs. parametric survival modeling on survival estimates and cost-effectiveness from the US Medicare payer perspective.
We created a partitioned survival decision model to assess the cost-effectiveness of 3 L nivolumab vs. usual care in rSCLC, based on observed US treatment patterns. Using this approach, we assessed the impact of extrapolating long-term survival from the CheckMate 032 trial, using both MCM and standard parametric curve fits. Nivolumab survival, resource use, and Grade 3/4 adverse event rates were derived from CheckMate 032. Usual care survival, resource use, and costs were derived from an analysis of patients receiving 3 L treatment for rSCLC in the SEER-Medicare registry. We applied 2020 Wholesale Acquisition Cost for drugs and 2020 CMS reimbursement for procedures. Utilities were derived from the literature. We estimated life years (LY), quality-adjusted life years (QALYs), and costs over a lifetime horizon.
MCM and parametric survival model extrapolations resulted in 0.43 versus 0.38 more LYs, 0.34 versus 0.30 more QALYs, and $69,308 versus $61,336 more expenditure for nivolumab vs. usual care, respectively. The costs per QALY gained using mixture cure versus parametric survival modeling were $204,386 and $207,431, respectively.
Mixture cure modeling was equivalent compared to parametric modeling in estimating the cost-effectiveness of nivolumab-based therapy due to the small fraction of patients achieving a long-term response with nivolumab (12.9%).
2018 年 8 月，美国食品药品监督管理局（FDA）批准纳武利尤单抗用于铂类化疗后和至少一种其他治疗线进展的小细胞肺癌（SCLC）的加速批准。本研究的目的是从支付者的角度评估纳武利尤单抗与常规护理作为复发性 SCLC（rSCLC）三线（3L）治疗的成本效益。鉴于接受治疗的患者中可能有相当一部分患者对纳武利尤单抗有长期反应的潜力，我们还评估了使用混合治愈模型（MCM）与参数生存模型对从美国医疗保险支付者角度对生存估计和成本效益的影响。
我们创建了一个分区生存决策模型，根据美国的治疗模式，评估 rSCLC 中 3L 纳武利尤单抗与常规护理的成本效益。使用这种方法，我们评估了从 CheckMate 032 试验中推断长期生存的影响，同时使用 MCM 和标准参数曲线拟合。纳武利尤单抗的生存、资源使用和 3/4 级不良事件发生率源自 CheckMate 032 试验。常规护理的生存、资源使用和成本源自 SEER-Medicare 登记处接受 rSCLC 三线治疗的患者分析。我们应用了 2020 年药品的批发采购成本和 2020 年 CMS 对程序的报销。效用来自文献。我们在终生时间范围内估计了生命年（LY）、质量调整生命年（QALY）和成本。
MCM 和参数生存模型推断分别导致纳武利尤单抗与常规护理相比，LY 增加 0.43 年，QALY 增加 0.34 年，支出增加 69308 美元。使用混合治愈模型与参数生存模型进行成本效益分析的成本每 QALY 增加分别为 204386 美元和 207431 美元。
由于纳武利尤单抗治疗的长期反应患者比例较小（12.9%），混合治愈模型在估计纳武利尤单抗为基础的治疗成本效益方面与参数模型相当。
