BresMed Health Solutions Ltd, Sheffield, UK.
BresMed Netherlands, Utrecht, The Netherlands.
J Med Econ. 2021 Jan-Dec;24(1):675-688. doi: 10.1080/13696998.2021.1917140.
There is limited published evidence for the cost-effectiveness of treatments for unresectable or metastatic endometrial cancer (mEC). The objective of this analysis was to assess the cost-effectiveness of pembrolizumab versus chemotherapy for previously treated unresectable or mEC, in women whose tumors have deficient mismatch repair (dMMR) or high microsatellite instability (MSI-H). The analysis was carried out from a US healthcare payer perspective.
A lifetime partitioned survival model comprising three health states (progression-free, progressed disease and death) was constructed. Chemotherapy was represented by single-agent paclitaxel or doxorubicin. Overall survival, progression-free survival and time on treatment data for pembrolizumab were obtained from a Phase II clinical study that included women with previously treated dMMR/MSI-H unresectable or mEC (KEYNOTE-158, NCT02628067). Survival data for chemotherapy were obtained from a published Phase III study for previously treated advanced endometrial cancer. Costs included were drug acquisition and administration, health-state, end-of-life, and adverse event management. Costs were presented in 2019 US$. Outcomes were calculated as quality-adjusted life-years (QALYs), using EQ-5D data from KEYNOTE-158. Model results were tested extensively in deterministic and probabilistic sensitivity analyses.
Results demonstrated that pembrolizumab is a highly cost-effective treatment option when compared with chemotherapy, with estimated deterministic and probabilistic incremental cost-effectiveness ratios (ICERs) of $58,165 and $57,668 per QALY gained, respectively. Pembrolizumab was associated with a large QALY and life-year gain per person versus chemotherapy over the model time horizon (deterministic 4.68 life year gain, 3.80 QALYs), with the majority of QALYs accrued in the progression-free health state.
The key limitation of the analysis was the lack of comparative effectiveness data for pembrolizumab versus chemotherapy.
Pembrolizumab is a highly cost-effective treatment option when compared with chemotherapy for women with previously treated dMMR/MSI-H unresectable or mEC. Results were robust to the changes in parameters and assumptions explored.
针对不可切除或转移性子宫内膜癌（mEC），目前仅有有限的治疗有效性和成本效益证据。本分析旨在评估在既往治疗的不可切除或 mEC 且存在错配修复缺陷（dMMR）或高微卫星不稳定性（MSI-H）的女性中，与化疗相比，pembrolizumab 的成本效益。该分析从美国医疗保健支付者的角度进行。
构建了一个终身分割生存模型，包含三个健康状态（无进展、疾病进展和死亡）。化疗由单药紫杉醇或多柔比星代表。pembrolizumab 的总生存、无进展生存和治疗时间数据来自一项包括既往治疗的 dMMR/MSI-H 不可切除或 mEC 女性的 II 期临床试验（KEYNOTE-158，NCT02628067）。化疗的生存数据来自一项已发表的针对既往治疗的晚期子宫内膜癌的 III 期研究。包括药物获取和管理、健康状态、生命终末期和不良事件管理的成本。成本以 2019 年美元表示。结果以使用 KEYNOTE-158 的 EQ-5D 数据计算的质量调整生命年（QALY）表示。模型结果在确定性和概率敏感性分析中进行了广泛测试。
结果表明，与化疗相比，pembrolizumab 是一种极具成本效益的治疗选择，其估计的确定性和概率增量成本效益比（ICER）分别为每获得一个 QALY 增加 58,165 美元和 57,668 美元。与化疗相比，pembrolizumab 每例患者的 QALY 和生命年获益都较大，在模型时间范围内（确定性为 4.68 年生命获益，3.80 QALYs），大部分 QALYs 发生在无进展健康状态。
分析的主要局限性是缺乏 pembrolizumab 与化疗相比的疗效数据。
与化疗相比，pembrolizumab 是一种极具成本效益的治疗选择，适用于既往治疗的 dMMR/MSI-H 不可切除或 mEC 女性。结果对所探讨的参数和假设的变化具有稳健性。
