OBJECTIVES: Hemophilia A (HA) is a rare congenital disorder caused by an inherited deficiency in blood clotting factor VIII, predominantly affecting males. Patients with HA face increased risks of bleeding following surgery or trauma, and spontaneous bleeding can occur. Approximately 30% of HA patients develop neutralizing alloantibodies (inhibitors) against Factor VIII, leading to complications and significantly increasing costs. Our main objective is to assess the budgetary impact of treating the Hemophilia pediatric population using Emicizumab in the Egyptian healthcare system over a 5-year time horizon.

METHODS:

A static budget impact model was conducted from a payer perspective to assess the Emicizumab in 240 Hemophilia A pediatric patients with inhibitors versus the traditional therapies including Factor VIII replacement, activated prothrombin complex concentrate (APCC), recombinant activated FVII (rFVIIa), and immune tolerance induction (ITI). Emicizumab is projected to treat 100% of pediatric patients. Direct medical costs were measured and captured from the unified procurement authority in Egypt. It includes the drug acquisition costs, follow-up, physician visits, and hospitalization due to bleeding events. One-way sensitivity analyses were performed.

RESULTS:

Significant cost savings were associated with Emicizumab over 5 years. The annual total costs for the current scenario were estimated to range between $554,017,231 to $2,770,086,156 from year 1 to year 5. While for the new scenario, the annual total costs were estimated to range between $224,520,210 to $1,122,601,051 from year 1 to year 5. The incremental cost reduction was estimated at 59% annually compared to traditional therapies.

CONCLUSIONS:

The budget impact analysis demonstrates the substantial cost savings with Emicizumab for Hemophilia A pediatric patients in Egypt. The adoption of Emicizumab significantly reduces total healthcare costs, demonstrating its economic and medical value.