Doctoral Grade Fellowship, Clinical Oncology Sector, Faculdade de Medicina do ABC, Santo André, Brazil.
Faculty of Medicine, Monash University, Mildura, Australia.
Ann Oncol. 2017 Sep 1;28(9):2256-2263. doi: 10.1093/annonc/mdx305.
Immune checkpoint inhibitors improve outcomes compared with chemotherapy in lung cancer. Tumor PD-L1 receptor expression is being studied as a predictive biomarker. The objective of this study was to assess the cost-effectiveness and economic impact of second-line treatment with nivolumab, pembrolizumab, and atezolizumab with and without the use of PD-L1 testing for patient selection.
We developed a decision-analytic model to determine the cost-effectiveness of PD-L1 assessment and second-line immunotherapy versus docetaxel. The model used outcomes data from randomized clinical trials (RCTs) and drug acquisition costs from the United States. Thereafter, we used epidemiologic data to estimate the economic impact of the treatment.
We included four RCTs (2 with nivolumab, 1 with pembrolizumab, and 1 with atezolizumab). The incremental quality-adjusted life year (QALY) for nivolumab was 0.417 among squamous tumors and 0.287 among non-squamous tumors and the incremental cost-effectiveness ratio (ICER) were $155 605 and $187 685, respectively. The QALY gain in the base case for atezolizumab was 0.354 and the ICER was $215 802. Compared with treating all patients, the selection of patients by PD-L1 expression improved incremental QALY by up to 183% and decreased the ICER by up to 65%. Pembrolizumab was studied only in patients whose tumors expressed PD-L1. The QALY gain was 0.346 and the ICER was $98 421. Patient selection also reduced the budget impact of immunotherapy.
The use of PD-L1 expression as a biomarker increases cost-effectiveness of immunotherapy but also diminishes the number of potential life-years saved.
免疫检查点抑制剂可改善肺癌患者的预后，优于化疗。目前正在研究肿瘤 PD-L1 受体表达作为预测生物标志物。本研究旨在评估在不进行 PD-L1 检测的情况下，以及在进行 PD-L1 检测的情况下，二线使用纳武利尤单抗、帕博利珠单抗和阿特珠单抗进行治疗的成本效益和经济影响。
我们开发了一个决策分析模型，以确定 PD-L1 评估和二线免疫治疗与多西他赛相比的成本效益。该模型使用了随机临床试验 (RCT) 的结果数据和美国的药物获取成本。此后，我们使用流行病学数据来估计治疗的经济影响。
我们纳入了四项 RCT（两项使用纳武利尤单抗，一项使用帕博利珠单抗，一项使用阿特珠单抗）。纳武利尤单抗在鳞状肿瘤中的增量质量调整生命年 (QALY) 为 0.417，在非鳞状肿瘤中的增量 QALY 为 0.287，增量成本效果比 (ICER) 分别为 155605 美元和 187685 美元。在基础病例中，阿特珠单抗的 QALY 获益为 0.354，ICER 为 215802 美元。与治疗所有患者相比，通过 PD-L1 表达选择患者可使增量 QALY 提高多达 183%，并使 ICER 降低多达 65%。帕博利珠单抗仅在肿瘤表达 PD-L1 的患者中进行了研究。QALY 获益为 0.346，ICER 为 98421 美元。患者选择也降低了免疫治疗的预算影响。
使用 PD-L1 表达作为生物标志物可提高免疫治疗的成本效益，但也减少了潜在的生命年节省数量。
