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新辅助免疫检查点抑制与顺铂为基础的化疗治疗肌肉浸润性膀胱癌的成本效益分析。

Cost-effectiveness analysis of neoadjuvant immune checkpoint inhibition vs. cisplatin-based chemotherapy in muscle invasive bladder cancer.

机构信息

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.

出版信息

Urol Oncol. 2021 Oct;39(10):732.e9-732.e16. doi: 10.1016/j.urolonc.2021.03.004. Epub 2021 Mar 23.

Abstract

BACKGROUND

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).

METHODS

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.

RESULTS

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.

CONCLUSIONS

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.

摘要

背景

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)。

方法

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。

结果

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。

结论

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。

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