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从美国视角评估布加替尼在未经ALK 抑制剂治疗的 ALK 阳性非小细胞肺癌成年患者中的成本效果。

The cost-effectiveness of brigatinib in adult patients with ALK inhibitor-naive ALK-positive non-small cell lung cancer from a US perspective.

机构信息

Takeda UK Ltd, London, UK.

Takeda Pharmaceuticals U.S.A., Inc., Lexington, MA.

出版信息

J Manag Care Spec Pharm. 2022 Sep;28(9):970-979. doi: 10.18553/jmcp.2022.28.9.970.

Abstract

The discovery of specific oncogenic drivers in non-small cell lung cancer (NSCLC) has led to the development of highly targeted anaplastic lymphoma kinase tyrosine kinase inhibitors (ALKis). Brigatinib is a next-generation ALKi associated with prolonged progression-free survival in patients with ALKi-naive + NSCLC. To estimate the cost-effectiveness of brigatinib compared with crizotinib and alectinib in patients with ALKi-naive + NSCLC, from a US payer perspective. A lifetime area under the curve-partitioned survival model with 4 health states was used to evaluate the relative cost-effectiveness of brigatinib in the ALKi-naive + NSCLC setting. Brigatinib was compared with crizotinib within a cost-effectiveness framework and compared with alectinib in a cost-comparison framework, where all efficacy outcomes were assumed equal. The efficacy of brigatinib and crizotinib was informed by the ALTA-1L trial, and an indirect treatment comparison was performed to inform the efficacy of brigatinib vs alectinib owing to a lack of head-to-head data. Costs were derived from public sources. The main outcomes of the model were total costs, quality-adjusted life-years (QALYs), life-years, and incremental cost-effectiveness ratios. Univariate and probabilistic sensitivity analyses, in addition to multiple scenario analyses, were conducted to assess the robustness of the model outcomes. The improved outcomes observed in ALTA-1L translated into QALY gains (+0.97) in the comparison of brigatinib vs crizotinib. The superior efficacy profile was associated with increased time on treatment with brigatinib, which drove the increase in costs vs crizotinib (+$210,519). The resulting base-case incremental cost-effectiveness ratio was $217,607/QALY gained. Compared with alectinib, brigatinib was associated with a cost difference of -$8,546. Sensitivity analysis suggested that extrapolation of overall survival, the assumptions relating to time on treatment, and subsequent therapy costs were the most influential determinants of results. Probabilistic sensitivity analysis suggested brigatinib had the highest probability of being cost-effective beyond willingness-to-pay thresholds of $236,000 per QALY vs crizotinib and alectinib. At list prices and under base-case assumptions in the current analysis, brigatinib was associated with cost-savings vs alectinib, and QALY gains but at higher costs vs crizotinib. Additional research into the real-world efficacy of ALKis is warranted to further understand the comparative cost-effectiveness of these therapies. Ms Cranmer and Ms Kearns are employees of Takeda UK Ltd. Dr Young is a former employee of Takeda Pharmaceuticals America, Inc. Dr Humphries is an employee of Takeda Pharmaceuticals U.S.A., Inc. Mr Trueman is an employee of Source Health Economics, the consultancy company that provided health economic and writing services. This work was funded by ARIAD Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited. Work by Source Health Economics was funded by ARIAD Pharmaceuticals, Inc. Professional medical writing assistance was provided by Phillipa White, of Source Health Economics, and funded by ARIAD Pharmaceuticals, Inc.

摘要

在非小细胞肺癌 (NSCLC) 中发现特定的致癌驱动因素,导致了高度靶向间变性淋巴瘤激酶酪氨酸激酶抑制剂 (ALKi) 的发展。布加替尼是一种下一代 ALKi,与 ALKi-初治+ NSCLC 患者的无进展生存期延长相关。 从美国支付者的角度估计布加替尼与克唑替尼和阿来替尼在 ALKi-初治+ NSCLC 患者中的成本效益。使用具有 4 种健康状态的终生分区生存模型来评估布加替尼在 ALKi-初治+ NSCLC 环境中的相对成本效益。在成本效益框架内将布加替尼与克唑替尼进行比较,并在成本比较框架内将其与阿来替尼进行比较,假设所有疗效结果均相等。布加替尼和克唑替尼的疗效来自 ALTA-1L 试验,由于缺乏头对头数据,进行了间接治疗比较以告知布加替尼与阿来替尼的疗效。成本来自公共资源。模型的主要结果是总成本、质量调整生命年 (QALY)、生命年和增量成本效益比。进行了单变量和概率敏感性分析,以及多种情景分析,以评估模型结果的稳健性。 在 ALTA-1L 中观察到的改善结果转化为布加替尼与克唑替尼比较时 QALY 的增加 (+0.97)。优越的疗效特征与布加替尼治疗时间的延长有关,这导致与克唑替尼相比成本增加 (+210,519 美元)。基础案例的增量成本效益比为每 QALY 增加 217,607 美元。与阿来替尼相比,布加替尼的成本差异为-8,546 美元。敏感性分析表明,总生存的外推、与治疗时间相关的假设以及随后的治疗成本是结果的最具影响力的决定因素。概率敏感性分析表明,布加替尼在超过每 QALY 236,000 美元的意愿支付阈值方面,比克唑替尼和阿来替尼更有可能具有成本效益。 在当前分析的标价和基础案例假设下,布加替尼与阿来替尼相比具有成本节约,与克唑替尼相比 QALY 增加,但成本更高。需要进一步研究 ALKi 的真实世界疗效,以进一步了解这些疗法的比较成本效益。 Cranmer 女士和 Kearns 女士是武田英国有限公司的员工。杨博士曾是武田制药美国公司的员工。Humphries 博士是武田制药美国公司的员工。特鲁曼先生是源健康经济学的员工,该咨询公司提供健康经济学和写作服务。这项工作由 ARIAD 制药公司(Takeda 制药公司的全资子公司)资助。源健康经济学的工作由 ARIAD 制药公司资助。ARIAD 制药公司为菲利普斯怀特(Phillipa White)提供专业的医学写作协助,菲利普斯怀特来自源健康经济学,费用由 ARIAD 制药公司支付。

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