OBJECTIVES : To estimate the budget impact of the incorporation of midostaurin for patients newly diagnosed with FLT3-positive AML who are fit to receive stem cell therapy in the BPHS.

METHODS : A budget impact model was developed including three phases of midostaurin chemotherapy (induction with cytarabine plus daunorubicin with/without midostaurin, consolidation with high dose cytarabine with/without midostaurin and maintenance with midostaurin for maximum of 12 cycles) based on time-to-event data from the RATIFY trial. Patients who did not achieve remission transferred to secondary therapy. Duration in each treatment phase was based on an “incidence model” approach and maximum treatment duration of two years. The proportions of patients in each health state were derived from the clinical outcomes of the RATIFY trial: overall survival, time-to-complete-remission, and time-to-stem-cell-transplantation. A five-year time horizon was used. Of the 47,540,061 BPHS beneficiaries, an incidence of 1.1/100,000 cases/habitants, estimated by a local epidemiology study, was used, as well a proportion of 33% for patients who were FLT3-positive, resulting in a baseline target population of 175. An annual population growth rate of 0.64% was also applied for the subsequent years of the model. For the scenario without midostaurin, a mix of standard chemotherapy schemes was adopted, assuming an average cost of treatment. Costs of treatment with midostaurin and secondary therapy were also considered. Estimated market share uptake for midostaurin was assumed ranging from 5% in the first year to 30% in the fifth year.

RESULTS : Over the five-year horizon, the introduction of midostaurin was estimated to cost an additional 10,979,722 USD (1USD=3.97BRL – May/2019), which corresponds to USS 4,619/100,000 beneficiaries per year in the BHPS.

CONCLUSIONS : Incorporation of midostaurin to BPHS leads to limited and predictable budget impact due to a small target patient population, medical cost-offsets justified by fewer patients requiring 2nd induction, and reduction the proportion of patients in relapse.