Department of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL, United States.
Janssen Scientific Affairs, LLC, a Johnson & Johnson company, Horsham, PA, United States.
Front Immunol. 2024 Aug 21;15:1408892. doi: 10.3389/fimmu.2024.1408892. eCollection 2024.
Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor T-cell therapy approved for patients with relapsed/refractory multiple myeloma (RRMM). In the phase 3 trial, CARTITUDE-4 (NCT04181827), cilta-cel demonstrated improved efficacy vs. standard of care (SOC; daratumumab plus pomalidomide and dexamethasone [DPd] or pomalidomide plus bortezomib and dexamethasone [PVd]) with a ≥ complete response (≥CR) rate of 73.1% vs. 21.8%.
A cost-per-responder model was developed to assess the value of cilta-cel and SOC (87% DPd and 13% PVd) based on the CARTITUDE-4 trial data from a US mixed payer perspective (76.7% commercial, 23.3% Medicare). The model was developed using progression-free survival (PFS), overall survival (OS), and ≥CR endpoints from CARTITUDE-4 over a period of 25.4 months. Inpatient stays, outpatient visits, drug acquisition, administration, and monitoring costs were included. The base-case model assumed an inpatient setting for each cilta-cel infusion; another scenario included 30% outpatient and 70% inpatient infusions. Costs of managing grade 3-4 adverse events (AEs) and grade 1-4 cytokine release syndrome and neurotoxicity were included. Subsequent therapy costs were incurred after disease progression; terminal care costs were considered upon death events. Outcomes included total cost per treated patient, total cost per complete responder, and cost per month in PFS between cilta-cel and SOC. Costs were adjusted to 2024 US dollars.
Total cost per treated patient, total cost per complete responder, and total cost per month in PFS were estimated at $704,641, $963,941, and $30,978 for cilta-cel, respectively, and $840,730, $3,856,559, and $42,520 for SOC over the 25.4-month period. Cost drivers included treatment acquisition costs before progression and subsequent treatment costs ($451,318 and $111,637 for cilta-cel; $529,795 and $265,167 for SOC). A scenario analysis in which 30% of patients received an outpatient infusion (assuming the same payer mix) showed a lower cost per complete responder for cilta-cel ($956,523) than those with an infusion in the inpatient setting exclusively.
This analysis estimated that cost per treated patient, cost per complete responder, and cost per month in PFS for cilta-cel were remarkably lower than for DPd or PVd, highlighting the substantial clinical and economic benefit of cilta-cel for patients with RRMM.
Ciltacabtagene autoleucel(cilta-cel)是一种嵌合抗原受体 T 细胞疗法,已获批准用于治疗复发/难治性多发性骨髓瘤(RRMM)患者。在 3 期临床试验 CARTITUDE-4(NCT04181827)中,cilta-cel 与标准治疗(SOC;daratumumab 联合泊马度胺和地塞米松[DPd]或泊马度胺联合硼替佐米和地塞米松[PVd])相比,疗效更佳,完全缓解率(≥CR)为 73.1%,而 21.8%。
基于 CARTITUDE-4 试验数据,从美国混合支付者的角度(76.7%商业,23.3%医疗保险),建立了一个基于每个反应者的成本模型,以评估 cilta-cel 和 SOC(87% DPd 和 13% PVd)的价值。该模型使用了 CARTITUDE-4 试验的无进展生存期(PFS)、总生存期(OS)和≥CR 终点,时间为 25.4 个月。包括住院和门诊就诊、药物获得、管理和监测成本。基础模型假设每个 cilta-cel 输注均为住院设置;另一种方案包括 30%的门诊和 70%的住院输注。包括 3-4 级不良事件(AE)和 1-4 级细胞因子释放综合征和神经毒性的管理成本。疾病进展后会产生后续治疗成本;在发生死亡事件时会考虑临终护理成本。结果包括每例治疗患者的总成本、每例完全缓解者的总成本和 PFS 期间每月的成本。成本调整为 2024 年的美元。
在 25.4 个月的时间内,cilta-cel 的每例治疗患者总成本、每例完全缓解者总成本和 PFS 期间每月总成本分别估计为 704641 美元、963941 美元和 30978 美元,SOC 分别为 840730 美元、3856559 美元和 42520 美元。成本驱动因素包括治疗前进展的治疗获取成本和后续治疗成本(cilta-cel 为 451318 美元和 111637 美元;SOC 为 529795 美元和 265167 美元)。在一项 30%的患者接受门诊输注的方案分析中(假设支付者组合相同),cilta-cel 的每例完全缓解者成本低于完全住院输注的成本(956523 美元)。
本分析估计,cilta-cel 的每例治疗患者总成本、每例完全缓解者总成本和 PFS 期间每月总成本明显低于 DPd 或 PVd,这突出了 cilta-cel 为 RRMM 患者带来的显著临床和经济效益。