Division of Haematology-Oncology, Department of Internal Medicine, Chang Gung Memorial Hospital at Linkou, Chang Gung University College of Medicine, Taoyuan, Taiwan.
College of Medicine, Chang Gung University, Taoyuan, Taiwan.
Cancer Rep (Hoboken). 2021 Dec;4(6):e1399. doi: 10.1002/cnr2.1399. Epub 2021 May 2.
Metastatic Merkel cell carcinoma (mMCC) has traditionally been managed with palliative chemotherapy regimens or best supportive care (BSC). Avelumab, a novel anti-programmed death-ligand 1 (PD-L1) human monoclonal antibody for mMCC treatment, is being studied in the pivotal JAVELIN Merkel 200 trial.
Incorporating trial results, this analysis aimed to evaluate the cost-utility of avelumab in Taiwan.
A de novo partitioned-survival model with three key health states related to survival (progression-free disease, progressed disease, and death) was applied in this study. The data of clinical efficacy, safety, and patient utilities were obtained from the JAVELIN Merkel 200 trial, literature review, and Taiwanese clinical expert opinion. Cost-utility analysis was performed, and results were presented as cost per quality-adjusted life year (QALY) gained. For treatment-naïve patients, the incremental cost-effectiveness ratios (ICERs) for avelumab vs BSC and avelumab vs chemotherapy were US$44885.06 and US$42993.06 per QALY gained, respectively. As to treatment-experienced mMCC patients, avelumab was associated with ICERs of US$27243.06 (vs BSC)/US$26557.43 (vs chemotherapy) per QALY gained. All ICERs remained consistently within the willingness-to-pay (WTP) threshold of US$53,333.33 per QALY gained.
This study demonstrated avelumab to be a cost-effective treatment option for both treatment-experienced and treatment-naïve mMCC patients with very poor prognosis in Taiwan.
转移性 Merkel 细胞癌（mMCC）传统上采用姑息性化疗方案或最佳支持治疗（BSC）进行治疗。avelumab 是一种用于 mMCC 治疗的新型抗程序性死亡配体 1（PD-L1）人源单克隆抗体，正在关键的 JAVELIN Merkel 200 试验中进行研究。
结合试验结果，本分析旨在评估 avelumab 在台湾的成本效益。
本研究采用了一个新的分割生存模型，该模型有三个与生存相关的关键健康状态（无疾病进展、疾病进展和死亡）。临床疗效、安全性和患者效用数据来自 JAVELIN Merkel 200 试验、文献回顾和台湾临床专家意见。进行了成本效用分析，并以每获得一个质量调整生命年（QALY）的成本（增量成本效益比，ICER）表示结果。对于初治患者，avelumab 与 BSC 和化疗相比的增量成本效益比（ICER）分别为每 QALY 增加 44885.06 美元和 42993.06 美元。对于有治疗经验的 mMCC 患者，avelumab 与 BSC 和化疗相比的 ICER 分别为每 QALY 增加 27243.06 美元和 26557.43 美元。所有 ICER 均保持在台湾每 QALY 增加 53333.33 美元的意愿支付（WTP）阈值内。
本研究表明，avelumab 对于台湾预后极差的初治和有治疗经验的 mMCC 患者是一种具有成本效益的治疗选择。
