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嵌合抗原受体 T 细胞疗法治疗血液系统恶性肿瘤和实体瘤的经济学评价:系统评价。

Economic Evaluations of Chimeric Antigen Receptor T-Cell Therapies for Hematologic and Solid Malignancies: A Systematic Review.

机构信息

Clinical Epidemiology Program, Ottawa Hospital Research Institute, General Campus, Ottawa, ON, Canada; School of Epidemiology and Public Health, University of Ottawa, Ottawa, ON, Canada; Pharmacoepidemiology and Statistics Research Center, Faculty of Pharmacy, Chiang Mai University, Chiang Mai, Thailand.

Clinical Epidemiology Program, Ottawa Hospital Research Institute, General Campus, Ottawa, ON, Canada.

出版信息

Value Health. 2024 Aug;27(8):1149-1173. doi: 10.1016/j.jval.2024.04.004. Epub 2024 Apr 17.

Abstract

OBJECTIVES

This study aimed to systematically review evidence on the cost-effectiveness of chimeric antigen receptor T-cell (CAR-T) therapies for patients with cancer.

METHODS

Electronic databases were searched in October 2022 and updated in September 2023. Systematic reviews, health technology assessments, and economic evaluations that compared costs and effects of CAR-T therapy in patients with cancer were included. Two reviewers independently screened studies, extracted data, synthesized results, and critically appraised studies using the Philips checklist. Cost data were presented in 2022 US dollars.

RESULTS

Our search yielded 1809 records, 47 of which were included. Most of included studies were cost-utility analysis, published between 2018 and 2023, and conducted in the United States. Tisagenlecleucel, axicabtagene ciloleucel, idecabtagene vicleucel, ciltacabtagene autoleucel, lisocabtagene maraleucel, brexucabtagene autoleucel, and relmacabtagene autoleucel were compared with various standard of care chemotherapies. The incremental cost-effectiveness ratio (ICER) for CAR-T therapies ranged from $9424 to $4 124 105 per quality-adjusted life-year (QALY) in adults and from $20 784 to $243 177 per QALY in pediatric patients. Incremental cost-effectiveness ratios were found to improve over longer time horizons or when an earlier cure point was assumed. Most studies failed to meet the Philips checklist due to a lack of head-to-head comparisons and uncertainty surrounding CAR-T costs and curative effects.

CONCLUSIONS

CAR-T therapies were more expensive and generated more QALYs than comparators, but their cost-effectiveness was uncertain and dependent on patient population, cancer type, and model assumptions. This highlights the need for more nuanced economic evaluations and continued research to better understand the value of CAR-T therapies in diverse patient populations.

摘要

目的

本研究旨在系统回顾嵌合抗原受体 T 细胞(CAR-T)疗法治疗癌症患者的成本效益证据。

方法

2022 年 10 月检索电子数据库,并于 2023 年 9 月更新。纳入比较癌症患者 CAR-T 治疗成本和效果的系统评价、卫生技术评估和经济评估。两名评审员独立筛选研究、提取数据、综合结果,并使用飞利浦清单对研究进行批判性评估。成本数据以 2022 年美元呈现。

结果

我们的搜索共产生 1809 条记录,其中 47 条被纳入。大多数纳入的研究为成本效用分析,发表于 2018 年至 2023 年期间,在美国进行。Tisagenlecleucel、axicabtagene ciloleucel、idecabtagene vicleucel、ciltacabtagene autoleucel、lisocabtagene maraleucel、brexucabtagene autoleucel 和 relmacabtagene autoleucel 与各种标准护理化疗进行了比较。成人中 CAR-T 疗法的增量成本效益比(ICER)范围为每质量调整生命年(QALY)9424 至 4124105 美元,儿科患者中每 QALY 为 20784 至 243177 美元。研究发现,随着时间的延长或假设更早的治愈点,增量成本效益比会提高。由于缺乏头对头比较以及对 CAR-T 成本和疗效的不确定性,大多数研究未达到飞利浦清单的要求。

结论

与对照相比,CAR-T 疗法更昂贵,产生了更多的 QALYs,但它们的成本效益不确定,取决于患者人群、癌症类型和模型假设。这突出表明需要更细致的经济评估和持续研究,以更好地了解 CAR-T 疗法在不同患者人群中的价值。

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