医学
成本效益
化学免疫疗法
造血干细胞移植
肿瘤科
临床试验
美罗华
移植
队列
弥漫性大B细胞淋巴瘤
内科学
质量调整寿命年
淋巴瘤
重症监护医学
风险分析(工程)
作者
Jee H. Choe,Tianzhou Yu,Jeremy S. Abramson,Mohamed Abou‐El‐Enein
出处
期刊:Blood Advances
[American Society of Hematology]
日期:2024-01-23
卷期号:8 (2): 484-496
被引量:1
标识
DOI:10.1182/bloodadvances.2023011793
摘要
Lisocabtagene maraleucel (liso-cel), a chimeric antigen receptor (CAR) T-cell therapy, received the US Food and Drug Administration approval in 2022 for second-line treatment of diffuse large B-cell lymphoma (DLBCL) for patients with refractory disease or early relapse after first-line chemoimmunotherapy. This decision was based on the TRANSFORM study demonstrating improvements in event-free survival with liso-cel compared with standard care. Given the high costs of CAR T-cell therapies, particularly as they transition to second-line treatment, a cost-effectiveness analysis is essential to determine their economic viability. The study used a partitioned survival model with standard parametric functions to evaluate the cost-effectiveness of liso-cel aganist platinum-based chemotherapy followed by high-dose chemotherapy and autologous hematopoietic stem cell transplantation over a lifetime horizon The analysis relied on data from the TRANSFORM and TRANSCEND trials, established literature, and public data sets to calculate the incremental cost-effectiveness ratio (ICER). For a representative cohort of US adults aged 60 years, ICER of liso-cel was $99 669 per quality-adjusted life-year (QALY) from a health care sector perspective and $68 212 per QALY from a societal perspective, confirming its cost-effectiveness at the $100 000 per QALY threshold. Nonetheless, under certain scenarios, liso-cel surpasses this benchmark but remains within the US acceptable range of $150 000 per QALY. A key finding underlines the importance of incorporating productivity losses into such analyses to capture the broader societal values of novel therapies. Although these therapies offer substantial clinical benefits, their high acquisition costs and limited long-term data critically challenge their economic sustainability.
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