Department of Health and Welfare Services, Center for Outcomes Research and Economic Evaluation for Health, National Institute of Public Health, 2-3-6, Wako, Saitama, 351-0104, Japan.
Meiji Pharmaceutical University, Tokyo, Japan.
Clin Drug Investig. 2022 Mar;42(3):253-262. doi: 10.1007/s40261-022-01124-y. Epub 2022 Mar 1.
Trastuzumab is a standard care as adjuvant chemotherapy (AdjCT) for patients with human epidermal growth factor receptor 2 (HER2)-positive primary breast cancer (BC) in Japan. However, no reports have evaluated its economics for patients with HER2-positive BC over 70 years of age. The objective of this study was to evaluate the cost-effectiveness of HER2-targeted trastuzumab + chemotherapy in Japan, comparing it with trastuzumab monotherapy.
A three-state-partitioned survival model was developed to evaluate the cost-effectiveness of trastuzumab + chemotherapy versus trastuzumab monotherapy for AdjCT in elderly patients with HER2-positive BC. We derived the efficacy data, utilities, and costs of both arms from individual patient data in the RESPECT trial (NCT01104935) and published studies. The costs and quality-adjusted life years (QALYs) were discounted at 2% per annum using a payer perspective. The respective cost estimates were reported in 2019 Japanese Yen (JPY) or US dollars (US$). The primary outcome was the incremental cost-effectiveness ratio (ICER). We assured robustness with deterministic and probabilistic sensitivity analyses.
The cost per patient for trastuzumab + chemotherapy was JPY 14.6 million (US$137,000), and their QALYs were 9.308, compared with JPY 14.2 million (US$131,000) and 9.101, respectively, for trastuzumab monotherapy. The ICER of trastuzumab + chemotherapy versus trastuzumab monotherapy was JPY 2.7 milllion/QALY (US$17,200/QALY). The ICER for trastuzumab with chemotherapy varied from "Dominant" to "Dominated" in one-way sensitivity analysis.
The base-case analysis suggests that AdjCT with trastuzumab + chemotherapy is likely to be a cost-effective choice for patients with HER2-positive BC aged 70 years or older. However, the sensitivity analysis suggested uncertainty regarding the cost-effectiveness of trastuzumab + chemotherapy.
曲妥珠单抗是日本人表皮生长因子受体 2（HER2）阳性原发性乳腺癌（BC）辅助化疗（AdjCT）的标准治疗方法。然而，尚无报告评估曲妥珠单抗对 70 岁以上 HER2 阳性 BC 患者的经济学价值。本研究旨在评估曲妥珠单抗联合化疗治疗日本 HER2 阳性 BC 患者的成本效果，与曲妥珠单抗单药治疗进行比较。
建立了一个三状态分区生存模型，以评估曲妥珠单抗联合化疗与曲妥珠单抗单药治疗老年 HER2 阳性 BC 患者 AdjCT 的成本效果。我们从 RESPECT 试验（NCT01104935）和已发表的研究中的个体患者数据中获得了这两种方案的疗效数据、效用值和成本。采用支付者视角，按照每年 2%的贴现率对成本和质量调整生命年（QALYs）进行贴现。相应的成本估计以 2019 年日元（JPY）或美元（USD）报告。主要结果是增量成本效果比（ICER）。我们通过确定性和概率敏感性分析来确保稳健性。
曲妥珠单抗联合化疗的每位患者成本为 1460 万日元（13.7 万美元），QALYs 为 9.308，而曲妥珠单抗单药治疗的相应成本为 1420 万日元（13.1 万美元）和 9.101。曲妥珠单抗联合化疗与曲妥珠单抗单药治疗的 ICER 为 2700 万日元/QALY（17200 美元/QALY）。在单项敏感性分析中，曲妥珠单抗联合化疗的 ICER 从“占优”变为“劣势”。
基于模型的分析表明，对于 70 岁或以上的 HER2 阳性 BC 患者，曲妥珠单抗联合化疗的 AdjCT 可能是一种具有成本效果的选择。然而，敏感性分析表明，曲妥珠单抗联合化疗的成本效果存在不确定性。
