■Ciltacabtageneautoleucel(cilta-cel)是一种嵌合抗原受体T细胞疗法,已批准用于复发性/难治性多发性骨髓瘤(RRMM)患者。在第三阶段的试验中,CARTITUDE-4(NCT04181827),cilta-cel表现出改善的疗效与护理标准(SOC;达拉图单抗+泊马度胺和地塞米松[DPd]或泊马度胺+硼替佐米和地塞米松[PVd]),≥完全缓解率(≥CR)为73.1%21.8%。
■根据美国混合付款人的CARTITUDE-4试验数据,开发了每个响应者成本模型,以评估cilta-cel和SOC(87%DPd和13%PVd)的价值(76.7%商业,23.3%的医疗保险)。该模型是使用无进展生存期(PFS)开发的,总生存期(OS),在25.4个月内来自CARTITUDE-4的≥CR终点。住院,门诊就诊,药物收购,administration,监测费用也包括在内。基本情况模型假定每次cilta-cel输注均为住院设置;另一种情况包括30%的门诊和70%的住院输注。包括管理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成本。疾病进展后发生后续治疗费用;临终护理费用在死亡事件时考虑。结果包括每位接受治疗的患者的总费用,每个完整响应者的总成本,以及cilta-cel和SOC之间的每月PFS成本。成本调整为2024美元。
■每位接受治疗的患者的总费用,每个完整响应者的总成本,PFS每月的总费用估计为704,641美元、963,941美元和30,978美元,分别,在25.4个月期间,SOC分别为840,730美元、3,856,559美元和42,520美元。成本驱动因素包括进展前的治疗获取成本和随后的治疗成本(西塔塞尔为451,318美元和111,637美元;SOC为529,795美元和265,167美元)。其中30%的患者接受门诊输液(假设相同的付款人组合)的情景分析显示,与仅在住院环境中进行输液的患者相比,cilta-cel的每个完整应答者的成本较低(956,523美元)。
■该分析估计每位接受治疗的患者的费用,每个完整响应者的成本,Cilta-cel的PFS每月成本显着低于DPd或PVd,强调cilta-cel对RRMM患者的实质性临床和经济效益。
UNASSIGNED: 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%.
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: 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.