Cost-Effectiveness of Axicabtagene Ciloleucel for Adult Patients With Relapsed or Refractory Follicular Lymphoma in the United States

IF 4.9 2区 医学 Q1 ECONOMICS Value in Health Pub Date : 2024-08-01 DOI:10.1016/j.jval.2024.04.003
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Abstract

Objectives

The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) follicular lymphoma (FL) demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared with standard of care (SOC) to manage r/r FL patients who have had at least 2 prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective.

Methods

A 3-state partitioned-survival cost-effectiveness model was developed with a lifetime horizon. Patient-level analyses of the 36-month ZUMA-5 (axi-cel) and SCHOLAR-5 (SOC) studies were used to extrapolate progression-free and overall survivals. After 5 years of survival, an estimated 40% of the modeled population was assumed to experience long-term remission based on literature. Results include the incremental cost-effectiveness ratio (ICER) measured as incremental cost per quality-adjusted life year (QALY) gained. One-way sensitivity analysis, probabilistic sensitivity analysis, and scenario analyses were performed. All outcomes were discounted 3% per year.

Results

Axi-cel led to an increase of 4.28 life-years, 3.64 QALYs, and a total cost increase of $321 192 relative to SOC, resulting in an ICER of $88 300 per QALY. Across all parameters varied in the one-way sensitivity analysis, the ICER varied between $133 030 and $67 277. In the probabilistic sensitivity analysis, axi-cel had a 99% probability of being cost-effective across 5000 iterations using a $150 000 willingness-to-pay threshold.

Conclusions

Given the robustness of the model results and sensitivity analyses, axi-cel is expected to be a cost-effective treatment in 3L+ r/r FL.

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美国治疗复发或难治性滤泡性淋巴瘤成人患者的阿昔单抗西乐葆的成本效益。
目的最近进行的一项阿昔卡巴替林西洛昔洛韦(axicabtagene ciloleucel,axi-cel)治疗复发/难治性滤泡性淋巴瘤(FL)的单臂试验(ZUMA-5)结果显示,接受治疗的患者中持久应答率高且毒性可耐受。为了量化axi-cel与标准疗法(SOC)相比对既往接受过至少2种系统疗法(3L+)的复发性/难治性滤泡性淋巴瘤患者的治疗价值,我们从美国第三方支付机构的角度建立了一个成本效益模型。对 ZUMA-5(axi-cel)和 SCHOLAR-5(SOC)为期 36 个月的研究进行了患者层面的分析,以推断无进展生存期和总生存期。根据文献资料,在 5 年生存期后,估计有 40% 的建模人群会出现长期缓解。结果包括增量成本效益比 (ICER),即每获得质量调整生命年 (QALY) 的增量成本。进行了单向敏感性分析、概率敏感性分析和情景分析。结果相对于SOC,Axi-cel可增加4.28个生命年、3.64个质量调整生命年,总成本增加321 192美元,每质量调整生命年的ICER为88 300美元。在单向敏感性分析中,在所有参数变化的情况下,ICER 在 133 030 美元到 67 277 美元之间变化。结论鉴于模型结果和敏感性分析的稳健性,在3L+ r/r FL中,axi-cel有望成为一种具有成本效益的治疗方法。
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来源期刊
Value in Health
Value in Health 医学-卫生保健
CiteScore
6.90
自引率
6.70%
发文量
3064
审稿时长
3-8 weeks
期刊介绍: Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.
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