OBJECTIVES: A cost-effectiveness model was developed to estimate the incremental-cost-effectiveness-ratio (ICER) of MIDO+SOC followed by MIDO monotherapy, compared to SOC in FLT3+AML patients, from a French perspective. Methods were based on Haute Autorité de Santé guidelines and international good research practices for modelling.

METHODS: A partitioned survival model was used and included five health states: AML diagnosis/induction, complete remission, relapse, stem cell transplantation (SCT) and mortality. A lifetime horizon was used, beginning at AML diagnosis/treatment initiation. Based on key opinion leader input, a “cure model” was used, applying natural mortality for overall survival to both treatment arms after trial cut-off. The economic endpoints used in the model were quality-adjusted life years (QALYs) and life years (LYs). No health-related quality-of-life data were available from the RATIFY trial, so utility values were obtained from a systematic literature review. Resource utilization and costs associated with each treatment included drug costs (primary therapy including maintenance MIDO and secondary therapy), SCT costs, routine care costs, adverse event-related costs (Grade 3-4, ≥5% of patients), and mortality costs.

RESULTS: In the RATIFY trial and after extrapolation, MIDO showed benefits for overall survival compared to SOC. This translated into LY gains, with MIDO-treated patients gaining 1.12 LYs versus SOC. When applying utility values to the different model health states, MIDO-treated patients gained 1.23 QALYs versus SOC. The ICER for MIDO versus SOC was €68,781 per LY and €62,305 per QALY. Deterministic and probabilistic sensitivity analyses generally showed consistency with basecase findings. When additional scenarios were explored, ICERs were most sensitive to variations in time horizon and discount rate.

CONCLUSIONS: With limited treatments in FLT3+AML, MIDO represents a clinically significant advancement in the management of newly diagnosed AML. In the economic analysis, MIDO add-on and subsequent maintenance therapy was associated with gains in LYs and QALYs versus SOC alone.