A mainstay of treatment in haemophilia patients with inhibitors (PWI) is recombinant Factor VIIa (rFVIIa) bypassing agent. The FDA recently approved a new rFVIIa (eptacog beta (EB)) variant that may reduce bypassing agent use for on-demand treatment of bleeding events (BEs). The aim of this study was to quantify the clinical and economic value associated with use of EB vs Coagulation rFVIIa eptacog alfa (EA), for on-demand treatment of bleeding episodes (BE) occurring in PWI.

A decision analytic model was developed to compare the utilization, clinical outcomes and costs of EA and EB among hemophilia PWI utilizing on-demand treatment of BEs over the course of one year. Treatment regimens of rFVIIa were based on prescribing information. Clinical efficacy was informed by clinical trial data. Epidemiology and costs for emergency department (ED) visits, hospital admissions, and intravenous-access (IV-access) complications were informed by literature. Drug costs were 2020 wholesale acquisition costs.

In a cohort of 1 million insured males, 5 PWI annually receive on-demand treatment for 54 mild/moderate BEs. If all PWI switch from EA to EB 75μg/kg or to EB 225μg/kg for on-demand use, a health plan would reduce the total costs from $3.7million to $1.7million (54%) or to $2.3million (40%), respectively. The majority of the savings are driven by reduced drug costs of $1.7million (85%) or $1.1million (76%), from the lower acquisition costs of EB and reduced consumption by 48% or 33%, with EB 75μg/kg or 225μg/kg, respectively. The remaining savings were from reduced ED visits, hospitalizations and IV-access complications.

The model suggests use of EB may reduce cost of rFVIIa from lower acquisition costs and fewer vials consumed. Additionally, fewer cases of re-bleeding or unresolved bleeding and fewer IV-accesses with EB therapy results in lower costs of ED visits, hospitalizations and IV-access complications.