Division of Medical Oncology, Department of Medicine, University of Washington, Seattle Cancer Care Alliance, Fred Hutchinson Cancer Research Center, Seattle, WA; Division of Oncology, Department of Medicine, Stanford University, Palo Alto, CA.
Division of Urology, Department of Surgery, The University of Texas Medical Branch, Galveston, TX.
Urol Oncol. 2021 Oct;39(10):732.e9-732.e16. doi: 10.1016/j.urolonc.2021.03.004. Epub 2021 Mar 23.
Multiple single-arm clinical trials showed promising pathologic complete response rates with neoadjuvant immune checkpoint inhibitors (ICIs) in muscle-invasive bladder cancer. We conducted a cost-effectiveness analysis comparing neoadjuvant ICIs with cisplatin-based chemotherapy (CBC).
We applied a decision analytic simulation model with a health care payer perspective to compare neoadjuvant ICIs vs. CBC. For the primary analysis we compared pembrolizumab with ddMVAC. We performed a secondary analysis with gemcitabine/cisplatin as CBC and exploratory analyses with atezolizumab or nivolumab/ipilimumab as ICI. We input pathologic complete response rates from trials or meta-analysis and costs from average sales price. Outcomes of interest included costs, 2-year recurrence-free survival (RFS), and incremental cost-effectiveness ratio (ICER) of cost per 2-year RFS. A threshold analysis estimated a price reduction for ICI to be cost-effective and one-way and probabilistic sensitivity analyses were performed.
The incremental cost of pembrolizumab compared with ddMVAC was $8,041 resulting in an incremental improvement of 1.5% in 2-year RFS for an ICER of $522,143 per 2-year RFS. A 21% reduction in cost of pembrolizumab would render it more cost-effective with an ICER of $100,000 per 2-year RFS. GC required an 89% pembrolizumab cost reduction to achieve an ICER of $100,000 per 2-year RFS. Atezolizumab appeared to be more cost-effective than ddMVAC.
ICIs were not cost-effective as neoadjuvant therapies, except when atezolizumab was compared with ddMVAC. Randomized clinical trials, larger sample sizes and longer follow-up are required to better understand the value of ICIs as neoadjuvant treatments.
多项单臂临床试验显示,新辅助免疫检查点抑制剂(ICI)在肌层浸润性膀胱癌中具有有前景的病理完全缓解率。我们进行了一项成本效益分析,比较了新辅助ICI 与顺铂为基础的化疗(CBC)。
我们采用了一种基于医疗保健支付者视角的决策分析模拟模型,比较了新辅助ICI 与 CBC。在主要分析中,我们将 pembrolizumab 与 ddMVAC 进行了比较。我们进行了次要分析,以 gemcitabine/cisplatin 作为 CBC,并进行了探索性分析,以 atezolizumab 或 nivolumab/ipilimumab 作为 ICI。我们从试验或荟萃分析中输入病理完全缓解率,并从平均销售价格中输入成本。感兴趣的结果包括成本、2 年无复发生存率(RFS)和每 2 年 RFS 的增量成本效益比(ICER)。阈值分析估计了 ICI 降价的成本效益,还进行了单因素和概率敏感性分析。
与 ddMVAC 相比,pembrolizumab 的增量成本为 8041 美元,导致 2 年 RFS 增加了 1.5%,每 2 年 RFS 的 ICER 为 522143 美元。如果 pembrolizumab 的成本降低 21%,则其成本效益更高,每 2 年 RFS 的 ICER 为 10 万美元。GC 需要降低 89%的 pembrolizumab 成本,才能达到每 2 年 RFS 10 万美元的 ICER。与 ddMVAC 相比,atezolizumab 似乎更具成本效益。
ICI 作为新辅助治疗方法并不具有成本效益,除非将 atezolizumab 与 ddMVAC 进行比较。需要进行随机临床试验、更大的样本量和更长的随访,以更好地了解 ICI 作为新辅助治疗的价值。