Use of Cost per Responder Models for CAR-T Therapies in Relapsed or Refractory Multiple Myeloma


Martin T1, Usmani SZ2, Joseph N3, Crivera C4, Valluri S5, Jackson CC6, Cohen L7, Singh S7, Jagannath S8
1UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA, USA, 2Memorial Sloan Kettering Cancer Center, New York, NY, USA, 3Janssen Scientific Affairs, LLC, Phoenixville, PA, USA, 4Janssen Scientific Affairs, LLC, Horsham, PA, USA, 5Janssen Global Services, LLC, Raritan, NJ, USA, 6Janssen R&D, Raritan, NJ, USA, 7Eversana Life Science Services, Burlington, ON, Canada, 8Icahn School of Medicine at Mount Sinai, New York, NY, USA

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.

Conference/Value in Health Info

2022-05, ISPOR 2022, Washington, DC, USA




Economic Evaluation


Genetic, Regenerative and Curative Therapies

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