在英国，接受过 BTKi 治疗后复发/难治性套细胞淋巴瘤患者中，KTE-X19 CAR T 疗法与真实世界标准护理的成本效益分析。

Cost-effectiveness analysis of KTE-X19 CAR T therapy versus real-world standard of care in patients with relapsed/refractory mantle cell lymphoma post BTKi in England.

机构信息

OPEN Health, Rotterdam, The Netherlands.

Lymphoma Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, United States.

出版信息

J Med Econ. 2022 Jan-Dec;25(1):730-740. doi: 10.1080/13696998.2022.2079317.

DOI:10.1080/13696998.2022.2079317

PMID:35611697

Abstract

AIMS

The objective of this study is to estimate the cost-effectiveness of KTE-X19 versus standard of care (SoC) in the treatment of patients with relapsed/refractory (R/R) mantle cell lymphoma (MCL) post-Bruton tyrosine kinase inhibitor (BTKi) treatment from a UK healthcare perspective.

MATERIALS AND METHODS

A three-state partitioned survival model (pre-progression, post-progression and death) with a cycle length of one month was used to extrapolate progression-free and overall survival over a lifetime horizon. Population inputs along with KTE-X19 (brexucabtagene autoleucel) efficacy and safety data were derived from the single-arm trial ZUMA-2 (NCT02601313). The composition of SoC was informed by a literature-based meta-analysis, SoC efficacy data were obtained from the SCHOLAR-2 real-world study. Survival was modelled using standard parametric curves for SoC and a mixture-cure methodology for KTE-X19. It was assumed that patients whose disease had not progressed after five years experienced long-term remission. Costs, resource use and utility, and adverse event disutility inputs were obtained from published literature and publicly available data sources. An annual discount rate of 3.5% was applied to costs and health outcomes. Modelled outcomes for KTE-X19 and SoC included expected life years (LY), quality-adjusted life years (QALY) and total costs. Deterministic and probabilistic sensitivity analyses and scenario analyses were performed.

RESULTS

Estimated median survival was 5.96 years for KTE-X19 and 1.38 for SoC. Discounted LYs, QALYs and lifetime costs were 8.27, 5.99 and £385,765 for KTE-X19 versus 1.98, 1.48 and £79,742 for SoC, respectively. The KTE-X19 versus SoC cost per QALY was £67,713 and the cost per LY was £48,645. Influential scenario analyses use alternative KTE-X19 survival curves and discount rates, and shorter time horizons.

CONCLUSION

Considering the survival and quality of life benefits compared to SoC, KTE-X19 for R/R MCL appears as a cost-effective treatment in the real-world UK setting.

摘要

目的

本研究旨在从英国医疗保健的角度，评估 KTE-X19 相较于标准治疗（SoC）用于治疗接受布鲁顿酪氨酸激酶抑制剂（BTKi）治疗后复发/难治性（R/R）套细胞淋巴瘤（MCL）患者的成本效果。

材料与方法

采用三状态分区生存模型（进展前、进展后和死亡），以一个月为一个周期，对终生的无进展生存期和总生存期进行外推。人群输入以及 KTE-X19（brexucabtagene autoleucel）的疗效和安全性数据源自单臂试验 ZUMA-2（NCT02601313）。SoC 的组成部分来源于基于文献的荟萃分析，SoC 的疗效数据来自 SCHOLAR-2 真实世界研究。采用标准参数曲线对 SoC 进行生存建模，对 KTE-X19 采用混合治愈方法进行建模。假设五年后疾病未进展的患者可长期缓解。成本、资源利用和效用以及不良事件失能的输入均来源于已发表的文献和公开可用的数据源。对成本和健康结果采用了 3.5%的年度贴现率。KTE-X19 和 SoC 的模型结果包括预期寿命（LY）、质量调整生命年（QALY）和总成本。进行了确定性和概率敏感性分析以及情景分析。

结果

KTE-X19 的估计中位生存期为 5.96 年，SoC 为 1.38 年。经贴现后，KTE-X19 的 LY、QALY 和终生成本分别为 8.27、5.99 和 385765 英镑，SoC 分别为 1.98、1.48 和 79742 英镑。KTE-X19 相较于 SoC 的每 QALY 成本为 67713 英镑，每 LY 成本为 48645 英镑。敏感性分析中，采用了替代的 KTE-X19 生存曲线和贴现率，以及更短的时间范围。