Toranomon Hospital, Minato-ku, Tokyo, Japan.
Kindai University, Osakasayama, Osaka, Japan.
J Gastroenterol. 2019 Jun;54(6):558-570. doi: 10.1007/s00535-019-01554-0. Epub 2019 Feb 20.
Lenvatinib demonstrated a treatment effect on overall survival by the statistical confirmation of non-inferiority to sorafenib for the first-line treatment of uHCC. The objective of this study was to evaluate the cost-effectiveness of lenvatinib compared with sorafenib for patients with uHCC in Japan.
A partitioned-survival model was developed to estimate the cost-effectiveness of lenvatinib versus sorafenib when treating uHCC patients over a lifetime horizon and considering total public healthcare expenditure. Efficacy and safety data were extracted from the REFLECT trial. Utility values were derived from the European Quality-of-Life 5-Dimension Questionnaire, conducted with patients enrolled in the REFLECT trial. Direct medical costs, such as primary drug therapy, outpatient visits, diagnostic tests, hospitalization, post-progression therapy, and adverse-event treatments, were included. Cost parameters unavailable in the clinical trial or publications were obtained based on the consolidated clinical standards from a Delphi panel of four Japanese medical experts.
For lenvatinib versus sorafenib, the incremental cost was - 406,307 Japanese Yen (JPY), and the incremental life years and quality-adjusted life years (QALYs) were 0.27 and 0.23, respectively. Thus, lenvatinib dominated sorafenib, due to the mean incremental cost-effectiveness ratio falling in the fourth quadrant, conferring more benefit at lower costs compared with sorafenib. The probabilistic sensitivity analysis showed that 81.3% of the simulations were favorable to lenvatinib compared with sorafenib, with a payer's willingness-to-pay-per-QALY of 5 million JPY.
Lenvatinib was cost-effective compared with sorafenib for the first-line treatment of uHCC in Japan.
仑伐替尼在索拉非尼的一线治疗中表现出优于索拉非尼的总生存治疗效果，通过统计学确认。本研究的目的是评估仑伐替尼与索拉非尼相比在日本治疗 uHCC 患者的成本效益。
开发了一个分区生存模型，以估计在终生范围内用仑伐替尼治疗 uHCC 患者的成本效益，并考虑总公共医疗支出。从 REFLECT 试验中提取疗效和安全性数据。效用值来自欧洲生活质量 5 维度问卷，该问卷是在参加 REFLECT 试验的患者中进行的。直接医疗费用，如主要药物治疗、门诊就诊、诊断测试、住院、进展后治疗和不良事件治疗，均包括在内。临床试验或出版物中未提供的成本参数是根据来自四名日本医学专家的 Delphi 小组的综合临床标准获得的。
仑伐替尼与索拉非尼相比，增量成本为-406307 日元（JPY），增量寿命年和质量调整寿命年（QALY）分别为 0.27 和 0.23。因此，仑伐替尼优于索拉非尼，因为平均增量成本效益比落入第四象限，与索拉非尼相比，成本更低，效益更高。概率敏感性分析表明，与索拉非尼相比，81.3%的模拟结果对仑伐替尼有利，支付者每 QALY 的意愿支付金额为 500 万日元。
仑伐替尼在日本作为 uHCC 的一线治疗药物具有成本效益。
