OBJECTIVES The objective of this study was to assess the cost-utility of using MIDOS + SoC for the treatment of newly-diagnosed FLT3 mutation-positive acute myeloid leukemia (AML) in adults in Colombia over a life time horizon.

METHODS An adaptation of a partition survival model was used to estimate the effectiveness and costs associated with routine care of the disease, administration of AML treatment, adverse events of each treatment scheme included in the model, stem cell transplantation (SCT), and second-line treatments. The model has a Markov of discrete times and cycles of 28 days, which considers the progression to the main health states of a patient diagnosed with AML: induction, consolidation, relapse, complete remission, SCT, and death. From the phase III RATIFY study, we extracted data on overall survival (OS), complete remission and the frequency of adverse events. Health states utility data were obtained from international evidence. Health resource use was determined with the collaboration of a clinical expert. The costs were obtained from the SISMED 2017 report (Q1-Q4) and the ISS 2001 tariff manual (+ 30%).

RESULTS An incremental cost-effectiveness ratio of $ 164,359,267 COP was estimated, with a cost difference of $ 202,921,254 COP and 1.23 additional QALYs for midostaurin. The main variables that drive uncertainty in the results are the OS hazard ratio with MIDOS and the SCT rate.

CONCLUSIONS Taking the cost-effectiveness threshold recommended by WHO of 1 to 3 GDP per capita (COP $ 56,484,299.7), the combination of MIDOS + SoC in patients with FLT3+ AML is not cost-effective. However, a gain of 1.23 years of QALYs was observed for midostaurin, and lower costs for routine care and mortality were observed in the group of patients who started treatment with MIDOS.