(228) Resource utilization and economic burden amongst a cohort of patients with hemophilia without inhibitors in the United States

Background: : Hemophilia is a rare bleeding disorder with complex manifestations and potentially expensive treatment options for patients with hemophilia A or B without inhibitors (PwHAwoI or PwHBwoI). Patients with hemophilia experience significant economic burden associated with their disease and its management.

Objective: : To assess hemophilia-related costs and healthcare resource utilization (HCRU) in PwHAwoI or PwHBwoI in the United States (US).

Methods: : This was a descriptive, non-interventional, retrospective cohort study of medical and pharmacy claims data (Komodo Health claims database). The study period ran from Jan 1, 2016, to Dec 31, 2023. The cohort includes PwHAwoI and PwHBwoI on non-prophylactic and prophylactic treatment regimens, identified from medication claims and defined as 6 consecutive prescriptions with >7 days’ supply and no gaps of >60 days.

Results: : Data was collated from 6606 patients, well-distributed across the US with 17–37% in each geographical quadrant. The 5095 PwHAwoI and 1511 PwHBwoI had a median age of 25.5 and 26.2 years, respectively. Almost all patients were male (91% HAwoI and 90% HBwoI). Joint related problems were reported for many patients (36% HAwoI and 35% HBwoI).

Mean annualized bleeding rate (ABR) was 1.1 in both groups. 58% of PwHAwoI (n=2967) and 59% (n=887) of PwHBwoI experienced ≥1 bleeding event. Of the 1449 (28%) PwHAwoI receiving prophylaxis, bleeds were experienced by 52% (n=752), with a mean ABR among those with ≥1 bleed of 1.8. Of the 271 (18%) PwHBwoI on prophylaxis, bleeds were experienced by 55% (n=150), with a mean ABR of 2.6.

On an annualized basis, patients demonstrated significant hemophilia-related HCRU. 14% of PwHAwoI were hospitalized (mean (SD) duration of 3.0 (7.5) days), 97% had outpatient visits and 39% visited the emergency department (ED). 17% of PwHBwoI were hospitalized (mean (SD) duration of 2.6 (4.5) days), 97% had outpatient visits and 38% visited the ED.

Mean all-cause total costs (PwHAwoI, $133,527; PwHBwoI, $85,778) were largely composed of pharmacy costs for HAwoI (54%) and medical costs for HBwoI (77%).

Conclusions: : Patients with hemophilia without inhibitors incur substantial economic burden. There is an urgent need for improved prophylactic options and targeted interventions to optimize patient care and mitigate costs.

Sponsorship:: Novo Nordisk