Cost-effectiveness analysis of cabozantinib as second-line therapy in advanced hepatocellular carcinoma
Cost-effectiveness analysis of cabozantinib as second-line therapy in advanced hepatocellular carcinoma
复制标题
卡博替尼二线治疗晚期肝细胞癌的成本效果分析
DOI：
10.1111/liv.14257
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发表时间：
2019-10-03
影响因子：
6.7
通讯作者：
Li, Qiu
中科院分区：
文献类型：
--
作者：
Liao, Weiting;Huang, Jiaxing;Li, Qiu
在晚期肝细胞癌（HCC）患者的CELESTIAL试验中，与安慰剂相比，卡博替尼显示出生存率提高，但代价高昂。我们的目的是从美国、英国和中国的支付者的角度调查卡博替尼治疗索拉非尼耐药HCC的成本效益。方法我们建立马尔可夫模型来模拟CELESTIAL试验后接受一线索拉非尼预治疗的患者。计算卡博替尼或最佳支持治疗的质量调整生命年（QRR）和增量成本效果比（ICER）。药品定价来源于红皮书、英国国家处方集、华西医院和文献报道。不良事件、效用权重和状态之间的转换可能性来自已发表的随机III期试验。愿意支付的门槛在美国设定为150 000美元/QALY，在英国设定为70 671美元/QALY（50磅/QALY），在中国设定为26 481美元/QALY（人均GDP的3倍）。确定性和概率敏感性分析，以测试模型的不确定性。结果在基础病例中，卡博替尼治疗的有效性增加了0.13 QALY，导致ICER与美国最佳支持治疗相比为833 497美元/QALY，英国为304 177美元/QALY，中国为156 437美元/QALY。该模型对关于使用卡博替尼和最佳支持性治疗向进展过渡的假设最敏感，该效用与无进展相关。这些结果在一系列情景和敏感性分析（包括确定性和概率性分析）中都是稳健的。结论：从美国、英国或中国的支付者的角度来看，卡博替尼在目前的成本水平下对索拉非尼耐药的HCC患者来说不是一种具有成本效益的治疗选择。要达到传统的成本效益阈值，就必须大幅折扣。
Background In the CELESTIAL trial for patients with advanced hepatocellular carcinoma (HCC), cabozantinib showed improved survival compared with placebo but comes at a price. We aimed to investigate the cost-effectiveness of cabozantinib for sorafenib-resistant HCC from the payer's perspective of the USA, UK and China. Methods We developed Markov models to simulate the patients pre-treated with first-line sorafenib following the CELESTIAL trial. Quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratio (ICER) were calculated for the treatment with cabozantinib or best supportive care. The list price for drugs was acquired from the Red Book, the British National Formulary, West China hospital and reported literature. Adverse events, utilities weights, and transition likelihood between states were sourced from the published randomized phase III trial. A willing-to-pay threshold was set $150 000/QALY in the USA, $70 671/QALY (50 pound 000/QALY) in the UK and $26 481/QALY (3x GDP per capita) in China. Deterministic and probabilistic sensitivity analyses were developed to test the models' uncertainty. Results In the base case, treatment with cabozantinib increased effectiveness by 0.13 QALYs, resulting in an ICER vs best supportive care of $833 497/QALY in the USA, $304 177/QALY in the UK and $156 437/QALY in China. The models were most sensitive to assumptions about transitions to progression with both cabozantinib and best supportive care, the utility associated with being progression free. These results were robust across a range of scenarios and sensitivity analyses, including deterministic and probabilistic analyses. Conclusions Cabozantinib at its current cost would not be a cost-effective treatment option for patients with sorafenib-resistant HCC from the payer's perspective in the USA, UK or China. Substantial discounts are necessary to meet conventional cost-effectiveness thresholds.