OBJECTIVES : To demonstrate potential budget impact of high-cost therapies with near-term indication expansion in the United States.

METHODS : We developed a budget impact model for tisagenlecleucel, a chimeric antigen receptor T-cell (CAR-T) therapy currently indicated for the treatment of relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) patients ≤25 years old. Tisagenlecleucel ’s manufacturer has submitted for FDA approval to treat relapsed/refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. For ALL, we used results from a recent cost-effectiveness analysis to estimate the net undiscounted costs of therapy with tisagenlecleucel compared to clofarabine chemotherapy over five years, assuming a cost of $475,000 and payment only on treatment response at one-month post-infusion. For ALL and DLBCL, we used current US epidemiological data to estimate the number of patients eligible for tisagenlecleucel. For DLBCL, we assumed that tisagenlecleucel has the same clinical effectiveness as an approved CAR-T (axicabtagene ciloleucel, when compared to palliative chemotherapy) in the DLBCL population, 50% market share in this indication, and the same price and payment structure as in the ALL population.

RESULTS : Our analysis estimates 617 new pediatric ALL cases eligible for treatment with tisagenlecleucel each year. Assuming all eligible patients received treatment with tisagenlecleucel rather than clofarabine, the estimated total budget impact is approximately $230.3 million per year, 25% of ICER’s potential budget impact threshold of $915 million per year. The annual eligible population for DLBCL is approximately 10 times greater (6,223). If tisagenlecleucel were approved for DLBCL, its annual total budget impact would be approximately $1.7 billion for both populations, exceeding the ICER annual budget impact threshold by approximately 86%.

CONCLUSIONS : If used for both indications, tisagenlecleucel would need to be priced substantially lower to remain within ICER’s budget threshold. An indication-specific pricing approach should be considered when pricing tisagenleceucel for future indications.