Department of Public Health, School of Medicine, International University of Health and Welfare, Narita, Chiba, Japan.
Department of Gastroenterology and Hepatology, Kindai University Faculty of Medicine, Osakasayama, Osaka, Japan.
Value Health Reg Issues. 2021 May;24:82-89. doi: 10.1016/j.vhri.2020.05.009. Epub 2021 Jan 30.
Hepatocellular carcinoma (HCC) is one of the leading causes of cancer-related mortality in Japan. Prognosis is poor, and until recently sorafenib was the only treatment option available for patients with unresectable disease. Lenvatinib is the first therapy to demonstrate noninferiority to sorafenib. An analysis was conducted using clinical data from Japanese patients in the phase III REFLECT trial to assess the cost-effectiveness of lenvatinib versus sorafenib for first-line treatment of unresectable HCC in Japan.
A partitioned survival model was implemented adopting the perspective of the Japanese healthcare system, with costs and outcomes modeled over a lifetime horizon and using a discount rate of 2%, as per Japanese guidelines. Population data from the Japanese subpopulation of REFLECT were used to extrapolate outcomes, and costs and resource use were based on Japanese sources. The Japanese tariff was applied to EQ-5D data collected during the REFLECT clinical trial to obtain utility values reflecting the preferences of the Japanese population.
Compared with sorafenib, lenvatinib is dominant because it is associated with a reduction in incremental costs of ¥156 799 and incremental quality-adjusted life-years of 0.31. These results were robust to changes in key assumptions, and probabilistic outcomes aligned with deterministic outcomes.
Given the use of Japan-specific data in the cost-effectiveness model, it is expected that the use of lenvatinib as a first-line treatment in Japan will be associated with cost savings and improved clinical outcomes versus sorafenib for patients with unresectable HCC.
肝细胞癌（HCC）是日本癌症相关死亡的主要原因之一。预后较差，直到最近，索拉非尼一直是不可切除疾病患者的唯一治疗选择。仑伐替尼是第一种被证明与索拉非尼非劣效的疗法。使用 III 期 REFLECT 试验中日本患者的临床数据进行了一项分析，以评估仑伐替尼与索拉非尼在日本作为不可切除 HCC 一线治疗的成本效益。
采用日本医疗保健系统的观点实施了分割生存模型，成本和结果在终生范围内建模，并按照日本指南使用 2%的贴现率。REFLECT 研究的日本亚组人群数据用于推断结果，成本和资源使用基于日本来源。根据 REFLECT 临床试验中收集的 EQ-5D 数据，应用日本关税将其转换为反映日本人群偏好的效用值。
与索拉非尼相比，仑伐替尼具有优势，因为它与增量成本降低 156799 日元和增量质量调整生命年增加 0.31 相关。这些结果对关键假设的变化具有稳健性，概率结果与确定性结果一致。
鉴于成本效益模型中使用了日本特定的数据，预计仑伐替尼在日本作为一线治疗不可切除 HCC 的使用将与索拉非尼相比，在节省成本和改善患者临床结果方面具有优势。
