A Cost-Effectiveness Analysis of Axicabtagene Ciloleucel versus Tisagenlecleucel in the Treatment of Diffuse Large B-cell Lymphoma Based on a Real-World French Registry

IF 3.4 3区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL
Markqayne Ray, Jean-Gabriel Castaigne, Alexandra Zang, Anik Patel, Elizabeth Hancock, Nicholas Brighton, Emmanuel Bachy
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引用次数: 0

Abstract

Introduction

Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are chimeric antigen receptor T-cell therapies that were evaluated in third and later line (3L+) relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) in the ZUMA-1 and JULIET trials, respectively. As of October 2021, the DESCAR-T registry included 729 French patients with 3L+ r/r DLBCL who received axi-cel or tisa-cel. Using these data, propensity score matching was used to conduct an adjusted comparison between axi-cel and tisa-cel. Axi-cel was associated with statistically significant improvements in overall survival (OS) and progression-free survival (PFS), and significantly more frequent Grade ≥ 3 immune effector cell-associated neurotoxicity syndrome (ICANS), compared with tisa-cel. There was no significant difference in Grade ≥ 3 cytokine release syndrome (CRS). The current analysis assessed the cost-effectiveness of axi-cel versus tisa-cel in the treatment of 3L+ r/r DLBCL using propensity score-matched data from the DESCAR-T registry.

Methods

A partitioned survival model was used to extrapolate costs and quality-adjusted life years (QALYs) over a lifetime. Survival curves for PFS and OS were based on independent mixture cure models fitted to digitized Kaplan–Meier data for the propensity score-matched DESCAR-T populations. Average duration of intensive care unit stays for each of axi-cel and tisa-cel in DESCAR-T were used to inform adverse event costs. Selected parametric survival distributions were based on clinical expert validation. Utility values were derived from ZUMA-1, and costs were obtained from French registries and published sources. List prices were used for both axi-cel and tisa-cel. Costs and outcomes were discounted at an annual rate of 2.5%.

Results

Axi-cel is associated with an incremental cost-effectiveness ratio of €15,520 per QALY compared with tisa-cel.

Conclusion

Based on explicit willingness-to-pay thresholds applied in Europe, axi-cel is expected to be a cost-effective use of healthcare resources in real-world clinical settings compared with tisa-cel in 3L+ r/r DLBCL.

基于法国真实世界登记的Axicabtagene Ciloleucel与Tisagenlecleucel治疗弥漫大B细胞淋巴瘤的成本效益分析》(Axicabtagene Ciloleucel versus Tisagenlecleucel in the Treatment of Diffuse Large B-cell Lymphoma based on a Real-World French Registry)。
导言:Axicabtagene ciloleucel(axi-cel)和tisagenlecleucel(tisa-cel)是嵌合抗原受体T细胞疗法,分别在ZUMA-1和JULIET试验中对三线及三线以上(3L+)复发或难治性(r/r)弥漫大B细胞淋巴瘤(DLBCL)进行了评估。截至2021年10月,DESCAR-T登记处纳入了729名接受axi-cel或tisa-cel治疗的3L+ r/r DLBCL法国患者。利用这些数据,倾向得分匹配法对axi-cel和tisa-cel进行了调整比较。与tisa-cel相比,Axi-cel的总生存期(OS)和无进展生存期(PFS)均有统计学意义上的显著改善,但≥3级免疫效应细胞相关神经毒性综合征(ICANS)的发生率明显更高。≥3级细胞因子释放综合征(CRS)没有明显差异。本次分析利用DESCAR-T登记处的倾向评分匹配数据,评估了axi-cel与tisa-cel治疗3L+ r/r DLBCL的成本效益:方法:采用分区生存模型推断一生中的成本和质量调整生命年(QALYs)。PFS和OS的生存曲线基于独立混合治愈模型,该模型拟合了倾向得分匹配的DESCAR-T人群的数字化Kaplan-Meier数据。DESCAR-T中axi-cel和tisa-cel在重症监护室的平均住院时间用于计算不良事件成本。选定的参数生存分布基于临床专家验证。效用值来自ZUMA-1,成本来自法国登记处和公开资料。axi-cel和tisa-cel均采用清单价格。成本和结果按2.5%的年贴现率折算:结果:与 tisa-cel 相比,Axi-cel 的增量成本效益比为每 QALY 15,520 欧元:结论:根据欧洲明确的支付意愿阈值,在现实世界的临床环境中,对于3L+ r/r DLBCL患者,与tisa-cel相比,axi-cel使用医疗资源具有成本效益。
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来源期刊
Advances in Therapy
Advances in Therapy 医学-药学
CiteScore
7.20
自引率
2.60%
发文量
353
审稿时长
6-12 weeks
期刊介绍: Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.
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