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中国弥漫性大 B 细胞淋巴瘤患者嵌合抗原受体 T 细胞治疗的性价比:来自成本效果分析的证据。

Value for Money of CAR-T Cell Therapy for Patients with Diffuse Large B-cell Lymphoma in China: Evidence from a Cost-Effectiveness Analysis.

机构信息

Department of Science and Education of the Fourth Affiliated Hospital, Center for Health Policy Studies, School of Public Health, Zhejiang University School of Medicine, Hangzhou, China.

Faculty of Medicine, Centre for Health Services Research, Queensland of University, Brisbane, Australia.

出版信息

Appl Health Econ Health Policy. 2023 Sep;21(5):773-783. doi: 10.1007/s40258-023-00817-5. Epub 2023 Jun 25.

Abstract

OBJECTIVE

This research assesses the cost effectiveness of Axicabtagene ciloleucel (Axi-cel), Tisagenlecleucel (Tis-cel), Relmacabtagene autoleucel (Rel-cel) and Lisocabtagene maraleucel (Lis-cel) against standard of care (SOC) for patients with diffuse large B-cell lymphoma (DLBCL) in the first-line setting (1L), second-line setting (2L) and third-line or later setting (3L+).

METHODS

Markov modelling based on a flexible survival model was adopted to evaluate four chimeric antigen receptor T-cell (CAR-T) therapies compared with SOC for patients with diffuse large B-cell lymphoma (DLBCL). The clinical inputs and utility values of the model were derived from the most recent clinical trials and the health care costs from a Chinese provincial clinical center. Costs and quality-adjusted life years (QALYs) were used to derive incremental cost-effectiveness ratios (ICERs) from the Chinese health care system perspective.

RESULTS

The ICER of Axi-cel (1L) versus SOC was approximately Chinese Yuan (CNY) 2,125,311 per QALY. The ICER for Axi-cel (2L), Tis-cel (2L) and Liso-cel (2L)) versus SOC in transplant-eligible patients were approximately CNY363,977, CNY32,066,781 and CNY347,746 per quality-adjusted life year (QALY), respectively. The ICER for Liso-cel (2L) versus SOC in transplant-ineligible patients was approximately CNY1,233,972 per QALY. The ICERs for Axi-cel (3L+), Tis-cel (3L+), Rel-cel (3L+) and Liso-cel (3L+) versus SOC were approximately CNY346,009, CNY654,344, CNY280,964 and CNY436,858 per QALY, respectively. In the scenario analysis using mixture cure models, the long-term survival benefit for CAR-T and SOC groups was found higher, and only Rel-cel (3L+) was found to be cost effective.

CONCLUSION

Our results demonstrated that CAR-T treatments are not cost effective in any-line settings for DLBCL patients at the WHO-recommended willingness-to-pay threshold (CNY257,241 per QALY) in the base-case analysis. Price reduction of CAR-T therapies is the main approach for lowering ICERs and ensuring that the drug costs are proportional to patient health benefits.

摘要

目的

本研究评估了 axi-cel、tisagenlecleucel、relmacabtagene autoleucel 和 lisocabtagene maraleucel 四种嵌合抗原受体 T 细胞(CAR-T)疗法在弥漫性大 B 细胞淋巴瘤(DLBCL)一线治疗(1L)、二线治疗(2L)和三线或更后线治疗(3L+)与标准治疗(SOC)相比的成本效果。

方法

采用基于灵活生存模型的马尔可夫模型,对四种嵌合抗原受体 T 细胞(CAR-T)疗法与 SOC 治疗弥漫性大 B 细胞淋巴瘤(DLBCL)患者进行比较。模型的临床输入和效用值来自最近的临床试验,以及中国省级临床中心的医疗成本。成本和质量调整生命年(QALY)用于从中国卫生保健系统的角度得出增量成本效益比(ICER)。

结果

Axi-cel(1L)与 SOC 相比的 ICER 约为 212.531 元人民币/质量调整生命年(QALY)。在可移植患者中,Axi-cel(2L)、Tis-cel(2L)和 Lisocel(2L)与 SOC 的 ICER 分别约为 363977 元人民币、32066781 元人民币和 347746 元人民币/QALY。在不可移植患者中,Liso-cel(2L)与 SOC 的 ICER 约为 123.3972 元人民币/QALY。Axi-cel(3L+)、Tis-cel(3L+)、Rel-cel(3L+)和 Lisocel(3L+)与 SOC 的 ICER 分别约为 346009 元人民币、654344 元人民币、280964 元人民币和 436858 元人民币/QALY。在使用混合治愈模型的情景分析中,发现 CAR-T 组和 SOC 组的长期生存获益更高,只有 Rel-cel(3L+)具有成本效果。

结论

在基于 WHO 推荐的意愿支付阈值(257241 元人民币/QALY)的基础分析中,我们的结果表明,CAR-T 治疗在 DLBCL 患者的任何一线治疗中都不具有成本效益。降低 CAR-T 疗法的价格是降低 ICER 和确保药物成本与患者健康效益成比例的主要方法。

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