Cost per Responder Analysis of Patients with Lenalidomide-Refractory Multiple Myeloma Who Received Cilta-Cel from the Cartitude-4 Trial

Introduction Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor-T cell (CAR-T) immunotherapy approved for patients (pts) 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 an overall response rate (ORR) of 84.6% vs 67.3% and ≥complete response (CR) of 73.1% vs 21.8%. A cost per responder model was developed to assess the value of cilta-cel and SOC within the CARTITUDE-4 trial from a US mixed payer perspective incorporating efficacy and total treatment costs. Methods We conducted a CPR analysis from a mixed payer perspective (76.7% commercial, 23.3% Medicare) comparing the direct medical cost per pt receiving cilta-cel versus SOC (87% DPd and 13% PVd, per trial distribution; 25.4 months). The model was developed using progression-free survival (PFS), overall survival (OS), time to next treatment, ≥CR, and ORR endpoints from CARTITUDE-4. A standard parametric approach extrapolated PFS and OS beyond the trial period. Resource use, drug acquisition, administration, and monitoring costs were included. The base case assumed the inpatient setting for each CAR-T infusion; another scenario included outpatient infusion (70% inpatient, 30% outpatient). Costs of managing grade 3-4 adverse events (AEs) and grade 1-4 AEs for cytokine release syndrome and neurotoxicity were included. Subsequent therapy costs were assigned after disease progression; terminal care costs were considered upon death events. Outcomes included total cost per treated pt, total cost per complete responder, and cost per month in PFS between cilta-cel and SOC. Costs were adjusted to 2023 US dollars. Results Total cost (PFS and post-progression survival [PPS] combined) per treated pt was estimated as $704,602 for cilta-cel and $793,796 for SOC over 25.4 months.Treatment acquisition and subsequent treatment costs were key total cost drivers during PFS and PPS, respectively ($451,318 and $108,636 for cilta-cel; $483,159 and $257,776 for SOC). Total costs per complete responder and total costs per month in PFS were estimated to be lower for cilta-cel than SOC (Figure). Scenario analysis with some pts receiving outpatient CAR-T infusion led to lower cost per complete responder and cost per month in PFS for cilta-cel ($956,290 and $30,841, respectively) than full inpatient CAR-T. Conclusions CPR analysis of CARTITUDE-4 data estimated that cost per complete responder and cost per month in PFS for cilta-cel were remarkably lower than DPd or PVd primarily driven by treatment acquisition and subsequent treatment costs demonstrating the substantial clinical and economic benefit cilta-cel offers for pts with RRMM.