OBJECTIVES: Chimeric antigen receptor T-cell (CAR-T) therapies targeting the B-cell maturation antigen are a new class of therapy for treating Relapsed or Refractory Multiple Myeloma (RRMM). As CAR-T therapies are introduced, payers will need to balance the higher efficacy, upfront costs, and total treatment costs when evaluating potential value. We developed a cost per responder (CPR) model which incorporates efficacy and total treatment cost to evaluate the value of RRMM CAR-T therapies (ciltacabtagene autoleucel [cilta-cel] and idecabtagene vicleucel [ide-cel]).

METHODS: Without available head-to-head trial data, unanchored matching adjusted indirect comparisons (MAIC) were used to evaluate the comparative efficacy of cilta-cel and ide-cel in the CPR model. MAIC results indicated that cilta-cel was associated with statistically significant improved overall response rate (odds ratio [OR]: 94.93 [95% confidence interval [CI]: 21.86, 412.25; p<.0001]), complete response or better rate (OR: 5.65 [95% CI: 2.51, 12.69; p<.0001]) and progression-free survival (PFS) (hazard ratio: 0.37 [95% CI: 0.24, 0.59; p<.0001]) when compared with ide-cel. To estimate total treatment costs, the CPR analysis also included the cost of apheresis, bridging therapy, CAR-T acquisition and administration, supportive care and monitoring, adverse event management, and any costs associated with delivery of inpatient or outpatient clinical services. A time horizon of approximately 31 months was used based on follow-up times available from the CARTITUDE-1 and KarMMa clinical trials.

RESULTS: Preliminary results indicate that ide-cel is associated with an approximate cost per overall responder of $763,000, cost per complete responder of $1,710,000, and cost per month in PFS of $50,000. Corresponding results for cilta-cel will be generated closer to the PDUFA date (February 28, 2022) and presented at ISPOR 2022.

CONCLUSIONS: CPR models have significant potential to support payers in evaluating the value of newer, more innovative RRMM therapies by incorporating information on both treatment efficacy and total costs.