Incentive Denmark Aps, Holte, Denmark.
Gilead Sciences Sweden AB, Solna, Sweden.
J Med Econ. 2023 Jan-Dec;26(1):1303-1317. doi: 10.1080/13696998.2023.2260689. Epub 2023 Nov 2.
Our study aimed to evaluate the cost-effectiveness of the chimeric antigen receptor (CAR) T-cell therapy, axicabtagene ciloleucel (axi-cel), compared to standard of care (SOC) in Sweden for second-line (2L) treatment of adult transplant-intended diffuse large B-cell lymphoma (DLBCL) patients who relapse within 12 months from completion of, or are refractory to (early r/r), first-line (1L) chemoimmunotherapy.
Cost-effectiveness was assessed using a three-state partitioned survival model. Mixture cure models were used to extrapolate time-to-event data from the ZUMA-7 trial (NCT03391466) beyond the observational period. Sensitivity and scenario analyses were performed to test the robustness of the base case results, including an analysis that assumed no switching to off-protocol CAR T-cell therapy in subsequent lines in the SOC arm.
The model estimated an incremental cost-effectiveness ratio (ICER) of SEK 534,704 (EUR 50,303) per quality-adjusted life year (QALY) gained over a lifetime horizon of 50 years, with an incremental cost of SEK 812,944 (EUR 76,479) and incremental QALY of 1.52 for axi-cel compared with SOC. The probabilistic sensitivity analysis showed that axi-cel was cost-effective in 73% of the simulations when assuming a willingness-to-pay threshold of SEK 1,000,000 (EUR 94,077) per QALY. The ICER was SEK 694,351 (EUR 65,313) in the scenario analysis where the costs and effects of treatment switching were not included.
2L treatment with axi-cel in transplant-intended DLBCL patients with early r/r after completing 1L chemoimmunotherapy was cost-effective compared to SOC in a Swedish setting. Administering axi-cel in 2L is cost-effective as it enhances the possibility of curing more patients, resulting in not just a survival advantage, but also a reduction in the burden on quality of life and cost of subsequent therapy. This will be advantageous to both patients and society.
我们的研究旨在评估嵌合抗原受体（CAR）T 细胞疗法 axicabtagene ciloleucel（axi-cel）与标准治疗（SOC）相比，在瑞典二线（2L）治疗完成后 12 个月内复发或对一线（1L）化疗免疫治疗早期难治（early r/r）的成人移植意向弥漫性大 B 细胞淋巴瘤（DLBCL）患者中的成本效益。
使用三状态分区生存模型评估成本效益。混合治愈模型用于从 ZUMA-7 试验（NCT03391466）的观察期之外推断时间至事件数据。进行敏感性和情景分析以测试基础案例结果的稳健性，包括假设 SOC 臂中后续线不再使用协议外 CAR T 细胞治疗的分析。
该模型估计在 50 年的生命周期内，每获得 1 个质量调整生命年（QALY）的增量成本效益比（ICER）为瑞典克朗 534704 （欧元 50303），axi-cel 与 SOC 相比，增量成本为瑞典克朗 812944（欧元 76479），增量 QALY 为 1.52。概率敏感性分析表明，当假设愿意支付的瑞典克朗 100 万（欧元 94077）的 QALY 阈值时，axi-cel 在 73%的模拟中具有成本效益。在未包括治疗转换的成本和效果的情景分析中，ICER 为瑞典克朗 694351（欧元 65313）。
在瑞典，与 SOC 相比，早期 r/r 完成 1L 化疗免疫治疗后的移植意向 DLBCL 患者接受 axi-cel 二线治疗具有成本效益。在 2L 中使用 axi-cel 是具有成本效益的，因为它增加了治愈更多患者的可能性，不仅带来了生存优势，而且降低了生活质量负担和后续治疗成本。这对患者和社会都有利。
