KTE-X19 CAR T 疗法治疗美国成人复发/难治性套细胞淋巴瘤的成本效益分析。

Cost-effectiveness for KTE-X19 CAR T therapy for adult patients with relapsed/refractory mantle cell lymphoma in the United States.

机构信息

Pharmerit - An OPEN Health Company, York, UK.

L. S. Skaggs Research Institute, The University of Utah College of Pharmacy, Salt Lake City, UT, USA.

出版信息

J Med Econ. 2021 Jan-Dec;24(1):421-431. doi: 10.1080/13696998.2021.1894158.

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 relapsed/refractory (R/R) mantle cell lymphoma (MCL) patients from a US healthcare perspective.

MATERIALS AND METHODS

A three-state partitioned-survival model (pre-progression, post-progression, and death) with a cycle length of 1 month was used to extrapolate progression-free and overall survival (OS) over a lifetime horizon. Due to the long tail of the OS curve, OS was modeled applying a mixture-cure methodology, using the assumption that patients whose disease had not progressed after 5 years experienced long-term remission. Population inputs were derived from the ZUMA-2 trial. This was also the source of KTE-X19 efficacy and safety data, while this data was obtained from the literature for SoC. Costs and resource use inputs were derived from the published literature and publicly available data sources. Health state utilities were derived from the ZUMA-2 trial (NCT02601313), applying the US tariff. Adverse event disutilities were derived from the published literature. Costs and health outcomes were discounted at 3% per year. The model estimated expected life years (LY), quality-adjusted life years (QALY), and total costs for KTE-X19 vs SoC. Deterministic and probabilistic sensitivity analyses were performed.

RESULTS

Median survival was 9.71 years for KTE-X19 and 2.13 for SoC. Discounted LYs, QALYs, and lifetime costs were 8.99, 7.39, and $693,832 for KTE-X19 vs 4.47, 3.65, and $574,263 for SoC, respectively. The KTE-X19 vs SoC cost per QALY was $31,985. The most influential model parameter was the utility for patients with long-term remission. At a willingness-to-pay threshold of $150,000 per QALY, the probability that KTE-X19 was cost-effective was 99%.

CONCLUSION

The treatment of R/R MCL with KTE-X19 presents a potentially cost-effective alternative to the current SoC, deriving its value from incremental survival and health-related quality-of-life benefits.

摘要

目的

本研究旨在从美国医疗保健的角度评估 KTE-X19 对比标准治疗(SoC)治疗复发/难治性(R/R)套细胞淋巴瘤(MCL)患者的成本效果。

材料和方法

采用三状态分区生存模型(进展前、进展后和死亡),周期长度为 1 个月,以推断终生的无进展生存(PFS)和总生存(OS)。由于 OS 曲线的长尾,采用混合治愈方法对 OS 进行建模,假设疾病在 5 年后未进展的患者经历长期缓解。人群输入来自 ZUMA-2 试验。这也是 KTE-X19 疗效和安全性数据的来源,而 SoC 的数据则来自文献。成本和资源使用输入来自已发表的文献和公开数据来源。健康状态效用来自 ZUMA-2 试验(NCT02601313),采用美国关税。不良事件失能来自已发表的文献。成本和健康结果以每年 3%贴现。该模型估计 KTE-X19 与 SoC 的预期寿命(LY)、质量调整生命年(QALY)和总费用。进行了确定性和概率敏感性分析。

结果

KTE-X19 的中位生存为 9.71 年,SoC 为 2.13 年。KTE-X19 的贴现 LY、QALY 和终生成本分别为 8.99、7.39 和 693832 美元,SoC 分别为 4.47、3.65 和 574263 美元。KTE-X19 与 SoC 的每 QALY 成本为 31985 美元。模型中最具影响力的参数是长期缓解患者的效用。在愿意支付每 QALY 150000 美元的阈值下,KTE-X19 具有成本效益的概率为 99%。

结论

KTE-X19 治疗 R/R MCL 可能是目前 SoC 的一种具有成本效益的替代方案,其价值来自于生存和健康相关生活质量的增量获益。

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