Department of Hematology-Oncology, Chang Gung Memorial Hospital at Linkou and College of Medicine, Chang Gung University, Taoyuan, Taiwan.
Evidera, London, UK.
Cancer Rep (Hoboken). 2023 Oct;6(10):e1887. doi: 10.1002/cnr2.1887. Epub 2023 Aug 28.
Patients with locally advanced or metastatic urothelial carcinoma have limited treatment options and a poor prognosis. The JAVELIN Bladder 100 trial showed that avelumab as first-line maintenance plus best supportive care significantly prolonged overall survival and progression-free survival versus best supportive care alone in patients with locally advanced or metastatic urothelial carcinoma that had not progressed with first-line platinum-containing chemotherapy.
We assessed whether avelumab plus best supportive care is a cost-effective treatment option versus best supportive care alone in this patient group in Taiwan.
A partitioned survival model was used to estimate the costs and effects of avelumab plus best supportive care versus best supportive care alone over a 20-year time horizon from the perspective of Taiwan's National Health Insurance Administration. Patient-level data from JAVELIN Bladder 100 on efficacy, safety, utility, and time on treatment were analyzed to provide parameters for the model. Log-normal and Weibull distributions were used for overall survival and progression-free survival, respectively. Costs of healthcare resources, drug acquisition, adverse events, and progression were identified through publicly available data sources and clinician interviews. The model estimated total costs, life years, and quality-adjusted life years. In the modeled base case, avelumab plus best supportive care increased survival versus best supportive care alone by 0.79 life years (2.93 vs. 2.14) and 0.61 quality-adjusted life years (2.15 vs. 1.54). The incremental cost-effectiveness ratio for avelumab plus best supportive care versus best supportive care alone was NT$1 827 680. Most (78%) of the probabilistic sensitivity analyses fell below three times the gross domestic product per capita. Scenario analysis indicated that life year and quality-adjusted life year gains were most sensitive to alternative survival extrapolations for both avelumab plus best supportive care and best supportive care alone.
Avelumab first-line maintenance therapy combined with best supportive care was determined as a cost-effective treatment strategy for patients in Taiwan diagnosed with locally advanced or metastatic urothelial carcinoma that had not progressed with platinum-containing chemotherapy.
局部晚期或转移性尿路上皮癌患者的治疗选择有限，预后较差。JAVELIN Bladder 100 试验表明，与单独最佳支持治疗相比，阿维鲁单抗作为一线维持治疗联合最佳支持治疗可显著延长局部晚期或转移性尿路上皮癌患者的总生存期和无进展生存期，这些患者在接受含铂化疗一线治疗后未进展。
我们评估在台湾，对于未接受含铂化疗的局部晚期或转移性尿路上皮癌患者，阿维鲁单抗联合最佳支持治疗与单独最佳支持治疗相比是否是一种更具成本效益的治疗选择。
使用分割生存模型，从台湾全民健康保险署的角度，在 20 年的时间范围内，估算阿维鲁单抗联合最佳支持治疗与单独最佳支持治疗相比的成本和效果。对 JAVELIN Bladder 100 试验中关于疗效、安全性、效用和治疗时间的患者水平数据进行分析，为模型提供参数。对数正态和威布尔分布分别用于总生存期和无进展生存期。通过公开数据源和临床医生访谈确定医疗资源、药物获取、不良事件和进展的成本。模型估计总费用、寿命年和质量调整寿命年。在模型的基本情况下，与单独最佳支持治疗相比，阿维鲁单抗联合最佳支持治疗增加了 0.79 个寿命年（2.93 比 2.14）和 0.61 个质量调整寿命年（2.15 比 1.54）。阿维鲁单抗联合最佳支持治疗与单独最佳支持治疗相比的增量成本-效果比为新台币 182.768 万。概率敏感性分析的大部分（78%）都低于人均国内生产总值的三倍。情景分析表明，与单独最佳支持治疗相比，阿维鲁单抗联合最佳支持治疗的寿命年和质量调整寿命年获益对阿维鲁单抗和单独最佳支持治疗的生存外推的替代方案最为敏感。
对于未接受含铂化疗的局部晚期或转移性尿路上皮癌患者，阿维鲁单抗一线维持治疗联合最佳支持治疗被确定为一种具有成本效益的治疗策略。
