A budget impact analysis of gilteritinib for the treatment of relapsed or refractoryFLT3^mut+acute myeloid leukemia in a US health plan

When it comes to drug access, should children be considered small adults? Countering coverage denials of FLT3 inhibitors in children with FLT3‐ITD AML

“…Because pediatric AML displays a lower mutational burden, it could be argued that FLT3 inhibitors may even be more effective in children than in adults 27 . From a fiscal standpoint, they are also cost‐effective 52 compared to HSCT or salvage chemotherapy 53 . In the context of relatively rare diseases, such as pediatric FLT3‐ITD AML, use of a standard based on “best available evidence” or “reasonable probability” may be more appropriate than requiring phase III, randomized controlled trial‐based proof of efficacy.…”