UK – Cost-effectiveness of pediatric leukemia CAR T-cell therapy studied

But long-term outcomes key; report follows NICE thumbs-down in adult DLBCL

Despite its $475,000 per-patient price tag, the survival gains and cost per life-year gained with tisagenlecleucel (Kymriah) in pediatric B-cell acute lymphoblastic leukemia (ALL) were both in line with commonly accepted thresholds, a cost-effectiveness analysis found.

Using conservative assumptions of survival in these patients, the incremental cost-effectiveness ratio for the chimeric antigen receptor (CAR) T-cell therapy would be between $37,000 and $78,000 per quality-adjusted life year (QALY) gained, reported Melanie Whittington, PhD, of the University of Colorado Anschutz Medical Campus in Aurora, and colleagues.

By extrapolating trial results of tisagenlecleucel, the researchers estimated that about four in 10 pediatric patients who initiated the CAR T-cell therapy could be considered long-term survivors compared with about one in 10 treated with the comparator, clofarabine.

« With the evidence available at this time, tisagenlecleucel seems to be priced in alignment with benefits observed over a patient lifetime horizon, » the authors wrote iJAMA Pediatrics, while noting that considerable uncertainty of the drug’s long-term benefit remains due to the limited available evidence.

Tisagenlecleucel was the first FDA-approved gene therapy, indicated for use in pediatric patients with relapsed or refractory B-cell ALL. The one-time cost of the treatment is the most expensive in cancer care…