HEOR, Parexel International, London, UK.
Worldwide Health Economics & Outcomes Research, Bristol Myers Squibb, Uxbridge, UK.
J Med Econ. 2021 Jan-Dec;24(1):1124-1133. doi: 10.1080/13696998.2021.1974763.
Present cost-effectiveness analysis of nivolumab monotherapy vs. commonly prescribed third-line (3 L+) treatment in small cell lung cancer (SCLC).
A three health states partitioned survival model (progression-free, progressed disease, and death; US payer perspective) was developed. The systematic literature review identified no randomized controlled or single-arm trials with separate outcomes for 3 L + SCLC patients. Topotecan was chosen as a comparator because it is frequently prescribed in real-world practice for 3 L SCLC. Clinical inputs for topotecan were derived from the Flatiron database with inclusion/exclusion criteria matched to patients treated with 3 L + nivolumab in CheckMate 032. Intravenous (IV) and oral topotecan clinical efficacy were assumed equivalent. Base-case analysis used a 20-year lifetime horizon. An annual discount rate of 3.0% for costs and outcomes was applied. Uncertainty was assessed using sensitivity analyses adjusted for key parameters.
Incremental cost per quality-adjusted life-year (QALY) gained with nivolumab was US$153,312 vs. IV topotecan and US$123,003 vs. oral topotecan, respectively. When results were disaggregated, nivolumab-related costs were mainly driven by drug acquisition costs, and topotecan-related costs were primarily due to adverse event treatment. Mean overall survival (OS) was 21.69 months with nivolumab and 5.80 months with IV or oral topotecan. More favorable outcomes were found by the landmark response analyses. Deterministic sensitivity analyses showed that changes to the discount rate for costs and outcomes and body weight had the greatest impacts on results.
Included use of real-world data for OS outcomes associated with 3 L topotecan, use of second-line topotecan data for progression-free survival, and no indirect costs.
Based on the literature on willingness-to-pay for a QALY in metastatic cancer, nivolumab monotherapy might represent a cost-effective option for 3 L + treatment of SCLC compared with IV and oral topotecan. Sensitivity analysis using response-based methods yielded further favorable cost-effectiveness estimates.
本项成本效益分析旨在比较纳武利尤单抗单药治疗与小细胞肺癌（SCLC）三线（3L+）治疗的常用方案。
采用三状态分区生存模型（无进展、进展疾病和死亡；美国支付者视角）进行分析。系统文献回顾未发现 3L+SCLC 患者有单独结局的随机对照或单臂试验。拓扑替康被选为对照药物，因为在现实实践中，拓扑替康常用于 3L SCLC 的治疗。拓扑替康的临床数据来自 Flatiron 数据库，其纳入/排除标准与接受纳武利尤单抗单药治疗的 CheckMate 032 研究中的 3L+纳武利尤单抗患者相匹配。静脉（IV）和口服拓扑替康的临床疗效假定相同。基础案例分析采用 20 年的生命周期。成本和结局的年贴现率为 3.0%。采用调整关键参数的敏感性分析评估不确定性。
与 IV 拓扑替康相比，纳武利尤单抗每增加一个质量调整生命年（QALY）的增量成本为 153312 美元，与口服拓扑替康相比为 123003 美元。当结果进行细分时，纳武利尤单抗相关成本主要由药物获取成本驱动，而拓扑替康相关成本主要由不良事件治疗所致。纳武利尤单抗的总生存期（OS）为 21.69 个月，IV 或口服拓扑替康为 5.80 个月。通过里程碑式反应分析发现了更有利的结果。确定性敏感性分析表明，成本和结局贴现率以及体重的变化对结果影响最大。
包括使用真实世界数据评估 3L 拓扑替康的 OS 结局，使用二线拓扑替康数据评估无进展生存期，以及未考虑间接成本。
基于转移性癌症 QALY 的支付意愿文献，与 IV 和口服拓扑替康相比，纳武利尤单抗单药治疗可能是 SCLC 三线治疗的一种具有成本效益的选择。使用基于反应的方法进行敏感性分析得出了更有利的成本效益估计。
