OBJECTIVES: Patients with the FMS-like tyrosine kinase 3 gene mutation (FLT3mut+) form of acute myeloid leukemia (AML) experience poor clinical outcomes. Limited treatment options result in many patients receiving supportive care, placing a considerable economic burden on healthcare systems. This study aimed to estimate the direct costs associated with the treatment of relapsed or refractory (R/R) FLT3mut+ AML in the Chilean public health system.

METHODS: The economic model included adult patients with confirmed R/R FLT3mut+ AML diagnosis who were already covered by the Chilean public health system. Fundamental health resource items, frequency of usage, and quantities were estimated from the literature, clinical practice guidelines, and semi-structured interviews with five Chilean clinical experts. Tariffs for cost valuation were based on the CENABAST public purchasing system, public hospital tariffs, and Chilean diagnosis related group fees for relevant clinical events. Costs were adjusted by inflation and converted to United States Dollars. In the economic model, costs were calculated per patient for the year 2024, and literature and local statistics were used to estimate the cost of illness for the overall target population for two scenarios: conservative (lower cost estimate) and likely (higher cost estimate).

RESULTS: The overall cost of illness of R/R FLT3mut+ AML in Chile in 2024 was calculated to be $6,698,826 and $6,880,121 for the conservative and likely scenarios. There were an estimated 114 new patients per year, and a per capita cost of $58,762 and $60,352 for the conservative and likely scenarios. Medication costs were estimated to be 67% of the overall cost of illness, followed by hematopoietic stem cell transplantation-related (13%), adverse event (8%), and post-progression AML care costs (8%).

CONCLUSIONS: This cost of illness economic model demonstrated that R/R FLT3mut+ AML conveys a considerable resource and economic burden in the Chilean public health system.