肿瘤细胞治疗研究
英文原题：Cost-effectiveness of treating relapsed or refractory 3L+ follicular lymphoma with axicabtagene ciloleucel vs mosunetuzumab in the United States.
本研究发现，在通常引用的 $150,000/QALY 美国支付意愿阈值下，与 mosun 相比，axi-cel 具有成本效益，且在一系列考虑参数不确定性的敏感性分析中结果稳健。
引言：美国食品药品监督管理局近期批准了多种用于三线及后线（3L+）复发/难治性（r/r）滤泡性淋巴瘤（FL）的新疗法，包括抗 CD19 CAR-T 疗法 axicabtagene ciloleucel（axi-cel）及 CD20×CD3 T 细胞衔接双特异性单克隆抗体 mosunetuzumab（mosun）。本研究旨在从美国第三方支付方角度，评估 axi-cel 相较 mosun 治疗 3L+ r/r FL 患者的成本效果。方法：采用包含无进展、疾病进展和死亡三个状态的分区生存模型，在假设的美国成人队列（年龄 18 岁）中比较两种治疗终身时间范围内的成本。采用 ZUMA-5 和 GO29781 试验数据估算无进展生存期（PFS）及总生存期（OS）。通过将风险比（HR）应用于 axi-cel 生存曲线，对 mosun 生存情况进行建模。PFS 的 HR 值通过匹配调整间接比较（MAIC）估算，依据 mosun 模拟个体患者数据及调整后的 axi-cel 数据，以校正试验人群差异。开展单因素敏感性分析（OWSA）和概率敏感性分析（PSA）。情景分析包括：（1）将 mosun HR 应用于加权（调整后的）ZUMA-5 24 个月数据，以尽可能精确反映 MAIC；（2）将 mosun HR 应用于 axi-cel 48 个月随访数据；（3）采用 axi-cel 治疗弥漫大 B 细胞淋巴瘤患者近期健康状态效用值。结果：与 mosun 相比，axi-cel 预计增加 1.82 个生命年（LY）和 1.89 个质量调整生命年（QALY）。axi-cel 患者的 PFS 为 6.42 LY，mosun 为 1.60 LY。无进展状态成本增加 257,113 美元，主要由 axi-cel 一次性治疗费用导致。axi-cel 总增量成本为 204,377 美元，增量成本效果比（ICER）为每增加一个 QALY 108,307 美元。OWSA 得出的 ICER 为 75,624–240,255 美元，其中除两个参数外，其余参数对应 ICER 均低于每 QALY 150,000 美元。PSA 在 5,000 次迭代中显示，按每 QALY 150,000 美元的支付意愿阈值，axi-cel 具有 64% 的成本效果概率。情景一和二的 ICER 分别为 105,353 美元和 102,695 美元。讨论：本研究发现，按照美国常用的每 QALY 150,000 美元支付意愿阈值，axi-cel 相较 mosun 具有成本效果；考虑参数不确定性的多种敏感性分析也显示结果稳健。
INTRODUCTION: Novel therapies for 3L+ relapsed/refractory (r/r) follicular lymphoma (FL) have been approved recently by the US Food and Drug Administration including anti-CD19 CAR-T therapies such as axicabtagene ciloleucel (axi-cel) and CD20 CD3 T-cell-engaging bispecific monoclonal antibodies such as mosunetuzumab (mosun). The objective of this study was to assess the cost-effectiveness of axi-cel compared to mosun in 3L+ r/r FL patients from a US third-party payer perspective. METHODS: A three-state (progression-free, progressed disease, and death) partitioned-survival model was used to compare two treatments over a lifetime horizon in a hypothetical cohort of US adults (age 18) receiving 3L+ treatment for r/r FL. ZUMA-5 and GO29781 trial data were used to inform progression-free survival (PFS) and overall survival (OS). Mosun survival was modeled via hazard ratios (HRs) applied to axi-cel survival curves. The PFS HR value was estimated via a matching-adjusted indirect comparison (MAIC) based on mosun pseudo-individual patient data and adjusted axi-cel data to account for trial populations differences. One-way sensitivity analysis (OWSA) and probabilistic sensitivity analyses (PSA) were conducted. Scenario analyses included: 1) the mosun HRs were applied to the weighted (adjusted) ZUMA-5 24-month data to most exactly reflect the MAIC, 2) mosun HR values were applied to axi-cel 48-month follow-up data, and 3) recent axi-cel health state utility values in diffuse large B-cell lymphoma patients. RESULTS: The analysis estimated increases of 1.82 LY and 1.89 QALY for axi-cel compared to mosun. PFS for axi-cel patients was 6.42 LY vs. 1.60 LY for mosun. Increase of $257,113 in the progression-free state was driven by one-time axi-cel treatment costs. Total incremental costs for axi-cel were $204,377, resulting in an ICER of $108,307/QALY gained. The OWSA led to ICERs ranging from $240,255 to $75,624, with all but two parameters falling below $150,000/QALY. In the PSA, axi-cel had an 64% probability of being cost-effective across 5,000 iterations using a $150,000 willingness-to-pay threshold. Scenarios one and two resulted in ICERs of $105,353 and $102,695, respectively. DISCUSSION: This study finds that axi-cel is cost-effective compared to mosun at the commonly cited $150,000/QALY US willingness-to-pay threshold, with robust results across a range of sensitivity analyses accounting for parameter uncertainty.
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