Long-term survival and value of chimeric antigen receptor T-cell therapy for paediatric patients with relapsed or refractory leukaemia

JAMA Pediatrics

8 October 2018 - What is the long-term survival and value of tisagenlecleucel for paediatric patients with relapsed or refractory leukaemia?

In this decision-analytic modelling study using de-identified data, cost-effectiveness analysis generated an incremental cost-effectiveness ratio between $37 000 and $78 000 per quality-adjusted life-year gained over a patient lifetime horizon, with more than 40% of those initiating tisagenlecleucel treatment becoming long-term survivors.

Tisagenlecleucel seems to be priced in alignment with its clinical benefits, although its value depends on assumptions around long-term survival.

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Michael Wonder

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Michael Wonder