早期 HER2 阳性乳腺癌曲妥珠单抗辅助使用奈拉替尼的成本效益分析。
Cost-Effectiveness Analysis of Adjuvant Neratinib Following Trastuzumab in Early-Stage HER2-Positive Breast Cancer.
机构信息
The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, University of Washington, Seattle.
Department of Surgery, University of Washington School of Medicine, and Fred Hutchinson Cancer Research Center, Seattle, Washington.
出版信息
J Manag Care Spec Pharm. 2019 Oct;25(10):1133-1139. doi: 10.18553/jmcp.2019.25.10.1133.
BACKGROUND
Disease-free survival (DFS) in early-stage human epidermal growth factor receptor 2 (HER2)-positive breast cancer is significantly greater with the addition of neratinib after adjuvant trastuzumab versus no additional therapy. However, it remains uncertain whether these survival gains represent good value for the money, given the substantial cost of neratinib.
OBJECTIVE
To evaluate clinical and economic implications of adding neratinib after adjuvant trastuzumab based on results from the phase III ExteNET trial.
METHODS
A 3-state Markov model was developed to estimate the cost-effectiveness of neratinib for women with early-stage (I-III) HER2-positive breast cancer. Five-year recurrence rates were derived from the ExteNET trial. Mortality and recurrence rates after 5 years were derived from the HERceptin Adjuvant (HERA) trial. Outcomes included life-years, quality-adjusted life-years (QALYs), and direct medical expenditures. The analysis was performed from a payer perspective over a lifetime horizon. One-way sensitivity and probabilistic analyses were conducted to evaluate uncertainty.
RESULTS
Total cost of neratinib following adjuvant trastuzumab was $317,619 versus $152,812 for adjuvant trastuzumab alone. A gain of 0.4 QALYs (15.7 vs. 15.3) and 0.1 years of projected life expectancy (18.3 vs. 18.2) favored neratinib after trastuzumab versus trastuzumab alone. The neratinib cost per QALY gained was $416,106. At standard willingness-to-pay thresholds of $50,000, $100,000, and $200,000 per QALY gained, neratinib has a probability of 2.8%, 16.7%, and 33.9% of cost-effectiveness, respectively. The cost per QALY gained in a scenario analysis only including patients with hormone-receptor positive disease was $275,311.
CONCLUSIONS
Based on 5-year data from ExteNET, neratinib following adjuvant trastuzumab is not projected to be cost-effective, even among those patients shown to derive the greatest clinical benefit. Future analyses should reassess the cost-effectiveness associated with neratinib treatment as trial data mature.
DISCLOSURES
No outside funding supported this study. Roth reports consulting fees from Genentech. Steuten reports grants from AstraZeneca, EMD Serono, and Genomic Health, along with personal fees from Agendia, unrelated to this study. The other authors have no conflicts of interest in connection with this study.
背景
与不进行额外治疗相比，在曲妥珠单抗辅助治疗后添加奈拉替尼可显著提高早期人表皮生长因子受体 2（HER2）阳性乳腺癌的无病生存期（DFS）。然而，鉴于奈拉替尼的高昂成本，这些生存获益是否代表物有所值仍不确定。
目的
根据 III 期 ExteNET 试验的结果，评估在曲妥珠单抗辅助治疗后添加奈拉替尼的临床和经济意义。
方法
建立了一个 3 状态马尔可夫模型，以评估早期（I-III 期）HER2 阳性乳腺癌患者使用奈拉替尼的成本效益。5 年复发率源自 ExteNET 试验。5 年后的死亡率和复发率源自 HERceptin Adjuvant（HERA）试验。结果包括寿命年、质量调整寿命年（QALY）和直接医疗费用。分析从支付者的角度在终身时间范围内进行。进行了单因素敏感性和概率分析以评估不确定性。
结果
曲妥珠单抗辅助治疗后添加奈拉替尼的总成本为 317619 美元，而仅使用曲妥珠单抗辅助治疗的总成本为 152812 美元。与仅使用曲妥珠单抗相比，奈拉替尼在曲妥珠单抗治疗后可使 QALY 增加 0.4（15.7 对 15.3），预期寿命延长 0.1 年（18.3 对 18.2）。与单独使用曲妥珠单抗相比，使用奈拉替尼每获得一个 QALY 的成本为 416106 美元。在标准的意愿支付阈值为 50000 美元、100000 美元和 200000 美元/QALY 时，奈拉替尼的成本效益分别为 2.8%、16.7%和 33.9%。在仅包括激素受体阳性疾病患者的情景分析中，奈拉替尼每获得一个 QALY 的成本为 275311 美元。
结论
根据 ExteNET 的 5 年数据，即使在从曲妥珠单抗治疗中获益最大的患者中，曲妥珠单抗辅助治疗后添加奈拉替尼也预计不会具有成本效益。随着试验数据的成熟，未来的分析应重新评估与奈拉替尼治疗相关的成本效益。
披露
本研究无外部资金支持。Roth 报告有来自 Genentech 的咨询费。Steuten 报告有来自 AstraZeneca、EMD Serono 和 Genomic Health 的拨款，以及与本研究无关的 Agendia 的个人酬金。其他作者与本研究无利益冲突。
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