Chimeric antigen receptor T-cell (CAR-T) therapy tisagenlecleucel may lead to gains in survival in children with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) that align with the price of the medication, a cost-effectiveness analysis conducted by The Institute for Clinical and Economic Review determined.1 The current list price for tisagenlecleucel is $475,000. The analysis results were published online October 8, 2018, in JAMA Pediatrics.

Patient survival outcomes were extracted from 3 studies (B2202, B2205J, and B2101J) and extrapolated over patient lifetime horizon to determine expected costs and outcomes from administration of tisagenlecleucel compared with treatment with clofarabine. Life-years gained, quality-adjusted life-years (QALYs) gained, and incremental costs per life-year and QALY gained were predicted for both tisagenlecleucel and clofarabine.

The analysis predicted that 40% of pediatric patients who initiate treatment with tisagenlecleucel will be long-term survivors, whereas only 10% of those who receive clofarabine will. The expected total discounted cost was $667,000 for tisagenlecleucel and $337, 000 for clofarabine. Tisagenlecleucel had discounted life-years gained of 10.34 years and 9.28 QALYs gained, whereas clofarabine had discounted life-years gained of 2.43 years and 2.10 QALYs gained. The incremental cost-effectiveness ratio was predicted to be $46,000 per QALY gained for tisagenlecleucel compared with clofarabine.

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“With the evidence available at this time, tisagenlecleucel seems to be priced in alignment with benefits observed over a patient lifetime horizon,” the study authors wrote. “The findings are sensitive to the long-term benefit forecasting, but based on currently available evidence and extrapolation assumptions, the cost-effectiveness likely is between $37,000 and $78,000 over a patient lifetime horizon.”

The study authors cautioned, however, that “considerable uncertainty” remains around the long-term benefit of tisagenlecleucel because there is limited available evidence.


  1. Whittington MD, McQueen RB, Ollendorf DA, et al. Long-term survival and value of chimeric antigen receptor T-cell therapy for pediatric patients with relapsed or refractory leukemia [published online October 8, 2018]. JAMA Pediatr. doi: 10.1001/jamapediatrics.2018.2530