OBJECTIVES: To estimate, from a UK payer perspective, the budget impact (BI) of a new recombinant activated factor VII (rFVIIa), eptacog beta (EB) in the treatment of spontaneous bleeding events (BE) and surgical bleedings (SB) in hemophilia A (HA) and B (HB) adult patients with inhibitors (PWI).

METHODS: A 5-year time horizon budget impact model was developed to estimate the financial consequences of giving access to the new rFVIIa (EB) to PWI currently treated with bypassing agents (BPAs), rFVIIa (eptacog alfa, EA) or an activated prothrombin complex concentrate. The model considered that some patients can be treated with prophylactic Non-Replacement Therapy (i.e. emicizumab). The model assumed similar efficacy between BPAs. Only drug acquisition costs are included as they represent majority of the total cost. The Mean Total Dose (MTD) of the current EA is assumed to be 20% higher than for EB for similar efficacy and same price per mg. Sensitivity analyses were performed.

RESULTS: Between 2022 and 2026, a maximum of 114 patients of 228 PWI are expected to be treated with the new rFVIIa (EB). From a UK payer perspective, considering a price parity between EA and EB and a 50% market share uptake over 3 years, a total £10.4 million saving is expected over 5 years. The introduction of EB would reduce expenditure by 7.1%. Savings are mainly driven by HA patients (84% of BI) and are distributed equally between the two indications (BE and SB, 49% and 51% respectively). BI is mainly sensitive to MTD and prices for EA and EB.

CONCLUSIONS: This study represents the first BI analysis that estimates the financial impact of EB for the UK. Considering as the only difference between EA and EB being a lower MTD for EB, adopting EB might result in more than £10.4 million savings over a 5-year time horizon.