After extrapolating trial results over a patient lifetime horizon, we estimated that more than 40% of pediatric patients who undergo leukapheresis in preparation for tisagenlecleucel therapy will be considered long-term survivors, compared with approximately 10% of pediatric patients considered long-term survivors after receiving comparator therapy. With the evidence available at this time, tisagenlecleucel seems to be priced in alignment with benefits observed over a patient lifetime horizon. 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. As payers are negotiating their coverage and reimbursement of tisagenlecleucel, they should consider the uncertainties in the evidence and the assumptions made in this analysis. Novel payment models consistent with the present evidence may reduce the risk and uncertainty in long-term value and be more closely aligned with ensuring high-value care. Financing cures in the United States is challenging owing to the high up-front price, rapid uptake, and uncertainty in long-term outcomes31,32; however, innovative payment models are an opportunity to address some of these challenges and to promote patient access to novel and promising therapies.
