Abstract：
UNASSIGNED: Patients with previously treated microsatellite instability-high (MSI-H)/mismatch repair deficient (dMMR) tumours have limited chemotherapeutic treatment options. Pembrolizumab received approval from the EMA in 2022 for the treatment of colorectal, endometrial, gastric, small intestine, and biliary MSI-H/dMMR tumour types. This approval was supported by data from the KEYNOTE-164 and KEYNOTE-158 clinical trials. This study evaluated the cost-effectiveness of pembrolizumab compared with standard of care (SoC) for previously treated MSI-H/dMMR solid tumours in line with the approved EMA label from a UK healthcare payer perspective.
UNASSIGNED: A multi-tumour partitioned survival model was built consisting of pre-progression, progressed disease, and dead health states. Pembrolizumab survival outcomes were extrapolated using Bayesian hierarchical models (BHMs) fitted to pooled data from KEYNOTE-164 and KEYNOTE-158. Comparator outcomes were informed by published sources. Tumour sites were modelled independently and then combined, weighted by tumour site distribution. A SoC comparator was used to formulate the overall cost-effectiveness result with pembrolizumab as the intervention. SoC comprised a weighted average of the comparators by tumour site based on market share. Drug acquisition, administration, adverse events, monitoring, subsequent treatment, end-of-life costs, and testing costs were included. Sensitivity and scenario analyses were performed, including modelling pembrolizumab efficacy using standard parametric survival models.
UNASSIGNED: Pembrolizumab, at list price, was associated with £129,469 in total costs, 8.30 LYs, and 3.88 QALYs across the pooled tumour sites. SoC was associated with £28,222 in total costs, 1.14 LYs, and 0.72 QALYs across the pooled tumour sites. This yields an incremental cost-effectiveness ratio (ICER) of £32,085 per QALY. Results were robust to sensitivity and scenario analyses.
UNASSIGNED: This model demonstrates pembrolizumab provides a valuable new alternative therapy for UK patients with MSH-H/dMMR cancer at the cost of £32,085 per QALY, with confidential discounts anticipated to improve cost-effectiveness further.
UNASSIGNED: A multi-tumour partitioned survival model was built consisting of pre-progression, progressed disease, and dead health states. Pembrolizumab survival outcomes were extrapolated using Bayesian hierarchical models (BHMs) fitted to pooled data from KEYNOTE-164 and KEYNOTE-158. Comparator outcomes were informed by published sources. Tumour sites were modelled independently and then combined, weighted by tumour site distribution. A SoC comparator was used to formulate the overall cost-effectiveness result with pembrolizumab as the intervention. SoC comprised a weighted average of the comparators by tumour site based on market share. Drug acquisition, administration, adverse events, monitoring, subsequent treatment, end-of-life costs, and testing costs were included. Sensitivity and scenario analyses were performed, including modelling pembrolizumab efficacy using standard parametric survival models.
UNASSIGNED: Pembrolizumab, at list price, was associated with £129,469 in total costs, 8.30 LYs, and 3.88 QALYs across the pooled tumour sites. SoC was associated with £28,222 in total costs, 1.14 LYs, and 0.72 QALYs across the pooled tumour sites. This yields an incremental cost-effectiveness ratio (ICER) of £32,085 per QALY. Results were robust to sensitivity and scenario analyses.
UNASSIGNED: This model demonstrates pembrolizumab provides a valuable new alternative therapy for UK patients with MSH-H/dMMR cancer at the cost of £32,085 per QALY, with confidential discounts anticipated to improve cost-effectiveness further.
摘要：
■先前治疗过的微卫星不稳定性高（MSI-H）/错配修复缺陷（dMMR）肿瘤的患者化疗治疗选择有限。Pembrolizumab于2022年获得EMA批准用于治疗结直肠，子宫内膜,胃,小肠和胆道MSI-H/dMMR肿瘤类型。这项批准得到KEYNOTE-164和KEYNOTE-158临床试验数据的支持。这项研究评估了pembrolizumab与以前治疗的MSI-H/dMMR实体瘤标准（SoC）相比的成本效益，符合英国医疗保健支付者角度批准的EMA标签。
■建立了一个多肿瘤分区的生存模型，包括进展前，疾病进展和死亡的健康状况。使用符合KEYNOTE-164和KEYNOTE-158的合并数据的贝叶斯分层模型（BHM）外推Pembrolizumab生存结果。比较结果由公布的来源告知。肿瘤部位独立建模，然后合并，按肿瘤部位分布加权。使用SoC比较器来制定pembrolizumab作为干预措施的总体成本效益结果。SoC包括基于市场份额的肿瘤部位的比较器的加权平均值。获取药物,administration,不良事件,监测,后续治疗,报废成本，测试费用也包括在内。进行了敏感性和情景分析，包括使用标准参数生存模型对帕博利珠单抗的疗效进行建模。
■Pembrolizumab，在标价下，与129,469英镑的总成本相关，8.30LYs,和3.88QALY跨越汇集的肿瘤部位。SoC的总成本为28,222英镑，1.14LYs和0.72QALYs跨越汇集的肿瘤部位。这产生了每QALY32,085英镑的增量成本效益比（ICER）。结果对敏感性和情景分析是稳健的。
■该模型表明，pembrolizumab为英国MSH-H/dMMR癌症患者提供了一种有价值的新替代疗法，每个QALY的费用为32,085英镑，保密折扣预计将进一步提高成本效益。
■建立了一个多肿瘤分区的生存模型，包括进展前，疾病进展和死亡的健康状况。使用符合KEYNOTE-164和KEYNOTE-158的合并数据的贝叶斯分层模型（BHM）外推Pembrolizumab生存结果。比较结果由公布的来源告知。肿瘤部位独立建模，然后合并，按肿瘤部位分布加权。使用SoC比较器来制定pembrolizumab作为干预措施的总体成本效益结果。SoC包括基于市场份额的肿瘤部位的比较器的加权平均值。获取药物,administration,不良事件,监测,后续治疗,报废成本，测试费用也包括在内。进行了敏感性和情景分析，包括使用标准参数生存模型对帕博利珠单抗的疗效进行建模。
■Pembrolizumab，在标价下，与129,469英镑的总成本相关，8.30LYs,和3.88QALY跨越汇集的肿瘤部位。SoC的总成本为28,222英镑，1.14LYs和0.72QALYs跨越汇集的肿瘤部位。这产生了每QALY32,085英镑的增量成本效益比（ICER）。结果对敏感性和情景分析是稳健的。
■该模型表明，pembrolizumab为英国MSH-H/dMMR癌症患者提供了一种有价值的新替代疗法，每个QALY的费用为32,085英镑，保密折扣预计将进一步提高成本效益。
