Costs of Hemophilia With Inhibitors Are High, Warranting Better Management | AMCP Annual 2025

An analysis of costs and healthcare resource utilization found that patients with hemophilia A or B with inhibitors (HAwI and HBwI) incurred substantial costs, with pharmacy and medical costs representing the highest contributing factor, according to a poster presented at the annual meeting of the Academy of Managed Care Pharmacy (AMCP) in Houston.

The study was conducted using the Komodo Health Claims Database on data from January 1, 2016, to December 31, 2023. During the analysis, most patients experienced at least one bleeding event. Moreover, all-cause costs were found to be twice as high for those with HAwI compared with HBwI. In both groups, the healthcare utilization was high, with nearly two-thirds requiring an emergency visit and a quarter to a third requiring an inpatient stay.

"People with hemophilia and inhibitors incur a substantial economic burden," the study authors, who are from Novo Nordisk, wrote in their poster. "There is an urgent need for improved prophylactic options and targeted interventions to optimize patient care and mitigate costs."

Hemophilia is an X chromosome-linked congenital bleeding disorder with joint damage as a consequence of the disease. As an X-linked disorder, it affects males much more often than females. Hemophilia A is more common than hemophilia B. The treatment for hemophilia has been factor replacement therapy for the missing clotting factors VIII and IX; however, in some patients, antibodies develop that inhibit clotting factors VIII and IX, limiting the efficacy of the replacement therapy. They are referred to as inhibitors. At the time of the study, the authors noted, there were no additional prophylactic treatments approved for HBwI and only one approved for those with HAwI.

Since the study was conducted, however, two new agents have gained approval. In December 2024, the FDA approved Novo Nordisk's Alhemo (concizumab) to prevent or reduce frequent bleeding episodes for patients with HAwI and HBwI. In March 2025, the FDA approved Sanofi's Qfitlia (fitusiran) for both types of hemophilia. The agent is approved to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age or older with or without the patient being on a factor VIII or IX inhibitor.

Study Details

For the study presented at AMCP, the investigators sought to examine the full economic impact of hemophilia with inhibitors in the United States using claims data for 84 patients with HAwI and 16 with HBwI. The median age of those with HAwI was 23.0, and it was 21.8 years for those with HBwI. Most of the enrolled patients were male, and 33% of those with HAwI and 50% of those with HBwI reported joint-related problems. Patients were well distributed across the United States.

At least one bleeding event was experienced by 69% of those with HAwI and 75% of those with HBwI. For the HAwI group, 49% were on prophylaxis. Of those on a prophylaxis (n = 41), at least one bleed was experienced by 49% of patients. The annual bleed rate (ABR) was 1.3 for HAwI and 1.8 for HBwI. For those with HAwI on prophylaxis who experienced one or more bleeds, the ABR was 1.6.

For the HAwI group, the annual mean length of stay (LOS) was six days for the 26% of patients who required inpatient care. All patients with HAwI required an outpatient visit during the study period, and 61% required an emergency visit.

For the HBwI group, the mean LOS was 13 days for the 38% of patients requiring inpatient care. In this group, 94% of patients were also treated in the outpatient setting, and 63% required an emergency visit.

The mean all-cause costs for those with HAwI was $359,304, and the mean pharmacy costs were $206,479. For these patients, pharmacy costs represented 57% of the total costs. For those with HBwI, the mean all-cause costs were $156,789, and the medical costs were $152,784, representing 97% of the total costs.