CAR T-Cell Therapy Cost-Effective for Pediatric ALL

A study published in JAMA Pediatrics found that tisagenlecleucel, a chimeric antigen receptor (CAR) T-cell therapy that costs $475,000 per treatment, likely improves survival in pediatric patients with leukemia “and seems to be priced in alignment with these benefits,” according to the authors. The results indicated that more than 40% of pediatric patients who undergo leukapheresis in preparation for tisagenlecleucel therapy will experience long-term survival compared with 10% of those who receive a comparator therapy—clofarabine in this study.

The researchers designed a decision analytic model to extrapolate trial evidence to a patient lifetime horizon. They assessed long-term survival outcomes for patients <25 years with relapsed/refractory B-cell acute lymphocytic leukemia from three studies.

Tisagenlecleucel had a total discounted cost of $667,000, with discounted life-years gained of 10.34 years and 9.28 quality-adjusted life-years (QALYs) gained. Clofarabine had a total discounted cost of approximately $337,000, with discounted life-years gained of 2.43 years and 2.10 QALYs gained.

This resulted in an incremental cost-effectiveness ratio of approximately $42,000 per life-year gained and approximately $46,000 per QALY gained for tisagenlecleucel.

“This study suggests that payers and innovators should develop novel payment models that reduce the risk and uncertainty around long-term value and provide safeguards to ensure high-value care,” the researchers concluded.

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Source: JAMA Pediatrics