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帕博利珠单抗联合阿昔替尼作为晚期肾细胞癌一线治疗的成本效果分析。

Cost-effectiveness of pembrolizumab with axitinib as first-line treatment for advanced renal cell carcinoma.

机构信息

Analysis Group Inc., Boston, MA, USA.

Merck & Co. Inc., Kenilworth, NJ, USA.

出版信息

Curr Med Res Opin. 2020 Sep;36(9):1507-1517. doi: 10.1080/03007995.2020.1799771. Epub 2020 Aug 8.

Abstract

OBJECTIVE

Pembrolizumab/axitinib significantly prolonged overall survival (OS) and progression-free survival (PFS), and increased objective response rate versus sunitinib in the phase III trial KEYNOTE-426 among previously untreated patients with advanced renal cell carcinoma (RCC). This study assessed the cost-effectiveness of pembrolizumab/axitinib versus other first-line treatments of advanced RCC from a US public healthcare payer perspective.

METHODS

A partitioned survival model with three states (progression-free, progressed, death) evaluated lifetime costs and quality-adjusted life-years (QALYs) for pembrolizumab/axitinib and other first-line regimens: sunitinib, pazopanib and avelumab/axitinib in the overall population; and sunitinib, cabozantinib and nivolumab/ipilimumab in the subgroup with intermediate/poor prognostic risk. Costs of treatments, adverse events and medical resources were estimated. OS, PFS and treatment duration were extrapolated using parametric models fitted to KEYNOTE-426 data and hazard ratios from network meta-analyses. Utilities were derived through mixed-effects regressions of KEYNOTE-426 EuroQol-5 Dimensions-3 Levels data.

RESULTS

In the overall population, pembrolizumab/axitinib was associated with incremental cost-effectiveness ratios (ICERs) of $95,725/QALY versus sunitinib and $128,210/QALY versus pazopanib, and was dominant (lower cost, higher effectiveness) versus avelumab/axitinib, with incremental QALY gains of 2.73, 2.40 and 1.80 versus these therapies, respectively. In the intermediate/poor-risk subgroup, base-case ICERs for pembrolizumab/axitinib were $101,030/QALY versus sunitinib, $6989/QALY versus cabozantinib, and $130,934/QALY versus nivolumab/ipilimumab, with incremental QALY gains of 2.62, 1.78 and 1.06 versus these therapies.

CONCLUSIONS

In this economic evaluation, pembrolizumab/axitinib was associated with higher life expectancy and QALYs and, based on typical willingness-to-pay thresholds of $150,000-$180,000/QALY, was found cost-effective versus other first-line treatments for advanced RCC in the US.

摘要

目的

在未经治疗的晚期肾细胞癌(RCC)患者的 III 期 KEYNOTE-426 试验中,与舒尼替尼相比,派姆单抗/阿昔替尼显著延长了总生存期(OS)和无进展生存期(PFS),并提高了客观缓解率。本研究从美国公共医疗保健支付者的角度评估了派姆单抗/阿昔替尼与其他晚期 RCC 一线治疗药物相比的成本效益。

方法

采用三状态(无进展、进展、死亡)分区生存模型,评估了派姆单抗/阿昔替尼与其他一线方案(舒尼替尼、帕唑帕尼和avelumab/axitinib )在总人群中的终生成本和质量调整生命年(QALY);以及在中/差预后风险亚组中(舒尼替尼、卡博替尼和纳武单抗/伊匹单抗)的治疗方案。治疗费用、不良事件和医疗资源均进行了评估。OS、PFS 和治疗持续时间通过使用 KEYNOTE-426 数据和网络荟萃分析中的风险比拟合的参数模型进行外推。效用通过 KEYNOTE-426 EuroQol-5 维度-3 水平数据的混合效应回归得出。

结果

在总人群中,与舒尼替尼相比,派姆单抗/阿昔替尼的增量成本效益比(ICER)为 95725 美元/QALY,与帕唑帕尼相比为 128210 美元/QALY,与avelumab/axitinib 相比具有优势(成本更低,效果更高),分别相对于这些治疗方案获得了 2.73、2.40 和 1.80 个 QALY 的增量。在中/差风险亚组中,派姆单抗/阿昔替尼的基础病例 ICER 分别为 101030 美元/QALY 与舒尼替尼,6989 美元/QALY 与卡博替尼,130934 美元/QALY 与纳武单抗/伊匹单抗,相对于这些治疗方案,分别获得了 2.62、1.78 和 1.06 个 QALY 的增量。

结论

在这项经济评估中,与其他一线治疗药物相比,派姆单抗/阿昔替尼与更高的预期寿命和 QALY 相关,并且基于典型的 15 万至 18 万美元/QALY 的意愿支付阈值,在美国对晚期 RCC 具有成本效益。

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