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Cost-effectiveness of chimeric antigen receptor T-cell therapy in pediatric relapsed/refractory B-cell acute lymphoblastic leukemia

Journal of the National Cancer Institute Dec 20, 2018

Sarkar RR, et al. - Researchers developed a microsimulation model to assess the cost-effectiveness of chimeric antigen receptor T-cell (CAR-T) therapy among pediatric patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL). Study participants included pediatric patients who received either CAR-T therapy or standard of care and were compared with respect to costs, quality of life (health utility), complications, and survival. Incremental cost-effectiveness ratios (ICERs) under $100,000 per quality-adjusted life-year (QALY) were considered cost effective. Model's uncertainty was assessed using one-way and probabilistic sensitivity analyses. The investigators observed an increase in overall cost by $528,200 and improvement in effectiveness by 8.18 QALYs with CAR-T therapy, resulting in an ICER of $64,600/QALY vs standard of care. With CAR-T, a 76.0% 1-year survival was assumed by the base model, although CAR-T was no longer cost effective if this decreased to 57.8%. At a willingness to pay of $100,000/QALY, CAR-T was demonstrated to be cost effective in 94.8% of iterations in probabilistic sensitivity analysis. Overall, CAR-T therapy may afford a cost-effective option for pediatric relapsed/refractory B-ALL.

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