OBJECTIVES: Management of hemophilia A has undergone a paradigm shift with the introduction of routine prophylaxis as a means of increasing plasma FVIII levels to prevent spontaneous bleeding and resulting joint damage. BAY 94-9027 is a prolonged–half-life site-specifically PEGylated recombinant factor VIII (rFVIII) product. The PROTECT VIII study showed that BAY 94-9027 provides effective protection against bleeds and is well tolerated with twice-weekly, every-5-day, and every-7-day prophylaxis in patients with severe hemophilia A. This study compares the rFVIII utilization in international units of BAY 94-9027 with other extended duration or standard-acting recombinant FVIII products used for prophylactic treatment of hemophilia A in Sweden.

METHODS: A rFVIII utilization analysis was conducted from the Swedish perspective using a utilization model. The analysis assumed equivalent efficacy between treatments. Prophylaxis utilization was informed by products’ summary of product characteristics. Swedish weight data were also retrieved to calculate the utilization for the population of interest. The model time horizon was 68 years. Patients entered the model aged 12 years, and underwent a prophylaxis dose reduction at age 20, in accordance with expert opinion. The model outcome was lifetime factor utilization.

RESULTS: BAY 94-9027 consumes the fewest units of clotting factor over a lifetime horizon, compared to extended duration products (ranging from 15% to 32% in the scenario chosen) and compared to standard-acting recombinant FVIII products (ranging from 10% to 44% in the scenario chosen). In a scenario where a goal of zero bleeds is achieved, BAY 94-9027 is also associated with the lowest utilization.

CONCLUSIONS: Assuming no difference in clinical outcomes (efficacy, safety, inhibitor incidence), treatment with BAY 94-9027 utilizes the fewest units of clotting factor over a lifetime, compared to relevant other treatments in Sweden. This has implications for healthcare budgets.