Dhanji Nishit, Decimoni Tassia Cristina, Dyer Matthew T D, May Jessica R, van de Wetering Gijs, Petersohn Svenja, Nickel Katharina, Silva Amanda, Muniz David Q B, Casagrande D Oliveira Ana Paula
Modeling & Meta-Analysis, OPEN Health, Oxford, UK.
Health Economics and Outcomes Research, Bristol Myers Squibb, São Paulo, SP, Brazil.
J Med Econ. 2023 Jan-Dec;26(1):1108-1121. doi: 10.1080/13696998.2023.2252716.
Nivolumab plus ipilimumab (NIVO + IPI) and pembrolizumab plus axitinib (PEM + AXI) have demonstrated significant clinical benefits as first-line (1 L) treatments for intermediate/poor-risk advanced renal cell carcinoma (aRCC) patients. This study aimed to assess the cost-effectiveness of NIVO + IPI versus PEM + AXI from a Brazilian private healthcare system perspective, utilizing a novel approach to estimate comparative efficacy between the treatments.
A three-state partitioned survival model (progression-free, progressed, and death) was developed to estimate costs, life-years (LYs), quality-adjusted LYs (QALYs), and the incremental cost-utility ratio (ICUR) over a 40-year time horizon. In the absence of head-to-head comparisons between NIVO + IPI and PEM + AXI, clinical data for NIVO + IPI was obtained from CheckMate 214 (NCT02231749) and for PEM + AXI from KEYNOTE-426 (NCT02853331). A matching-adjusted indirect comparison was conducted to account for the imbalance of treatment effect modifiers between the trials. Patient characteristics, resource use, health state utilities, and costs were based on Brazilian-specific sources. Costs and health outcomes were both discounted by 5% annually in line with Brazilian guidelines. The robustness of the results was evaluated through extensive sensitivity analysis and scenario analyses.
When comparing the versus OS, PFS, and TTD curves there was no noteworthy difference. NIVO + IPI was associated with cost savings (R$ 350,232), higher LYs (5.54 vs. 4.61), and QALYs (4.74 vs. 3.76) versus PEM + AXI, resulting in NIVO + IPI dominating PEM + AXI. Key model drivers were the treatment duration for PEM, NIVO, and AXI. NIVO + IPI remained dominant in all scenario analyses, which indicated that model results were robust to alternative modelling inputs or assumptions.
This analysis shows that NIVO + IPI is estimated to be a life-extending and potentially cost-saving 1 L treatment option when compared with PEM + AXI for intermediate/poor-risk a RCC patients in the Brazilian private healthcare system.
纳武利尤单抗联合伊匹木单抗（NIVO+IPI）和帕博利珠单抗联合阿昔替尼（PEM+AXI）已被证明作为中/低风险晚期肾细胞癌（aRCC）患者的一线（1L）治疗具有显著的临床益处。本研究旨在从巴西私立医疗系统的角度评估NIVO+IPI与PEM+AXI的成本效益，采用一种新方法来估计两种治疗方法之间的比较疗效。
建立了一个三状态分区生存模型（无进展、进展和死亡），以估计40年时间范围内的成本、生命年（LYs）、质量调整生命年（QALYs）和增量成本效用比（ICUR）。由于缺乏NIVO+IPI与PEM+AXI之间的直接比较，NIVO+IPI的临床数据来自CheckMate 214（NCT02231749），PEM+AXI的临床数据来自KEYNOTE-426（NCT02853331）。进行了匹配调整间接比较，以考虑试验之间治疗效果修饰因素的不平衡。患者特征、资源使用、健康状态效用和成本基于巴西特定来源。根据巴西指南，成本和健康结果均按每年5%进行贴现。通过广泛的敏感性分析和情景分析评估结果的稳健性。
比较总生存期（OS）、无进展生存期（PFS）和至疾病进展时间（TTD）曲线时，没有显著差异。与PEM+AXI相比，NIVO+IPI可节省成本（350,232雷亚尔），具有更高的生命年（5.54对4.61）和质量调整生命年（4.74对3.76），导致NIVO+IPI优于PEM+AXI。关键模型驱动因素是PEM、NIVO和AXI的治疗持续时间。NIVO+IPI在所有情景分析中均保持优势，这表明模型结果对替代建模输入或假设具有稳健性。
该分析表明，在巴西私立医疗系统中，对于中/低风险aRCC患者，与PEM+AXI相比，估计NIVO+IPI是一种可延长生命且可能节省成本的1L治疗选择。
