Lifetime economic burden of hemophilia using a nationwide real-world healthcare data.

BACKGROUND: Patients with hemophilia require lifelong treatment, but the real-world lifetime economic burden of hemophilia remains unclear. This study aims to estimate the lifetime economic burden of hemophilia using real-world data, accounting for cost variation by disease phase and over time. METHODS: Male patients with hemophilia A (PwHa) or B (PwHb) recorded in South Korea's Health Insurance Review and Assessment (HIRA) database from 2007 to 2022 were included. Survival was estimated using the rolling extrapolation method. A phase-specific costing approach was applied, distinguishing three phases: before hemophilic arthropathy (BH), after hemophilic arthropathy (AH), and 1 year before death (BD). Transition probabilities from BH to AH were calculated based on incidence rates of hemophilic arthropathy. Phase-specific annual costs were modeled using generalized estimating equations (GEEs), and predicted costs were multiplied by phase-specific probabilities from birth to estimated life expectancy to derive lifetime costs. RESULTS: Estimated life expectancy for PwHa (n = 2,624) and PwHb (n = 664) in South Korea between 2007-2022 was 76.13 and 77.54 years, respectively. The incidence rate of hemophilic arthropathy was 0.090 cases/person-year [95% confidence interval, 0.084-0.097] for PwHa and 0.080 [0.070-0.092] for PwHb, yielding transition probabilities from BH to AH of 0.086 (PwHa) and 0.077 (PwHb). Mean annual costs were highest in the BD phase, followed by AH and BH phases: PwHa (BH: $11,331; AH: $27,271; BD: $27,985); PwHb (BH: $15,567; AH: $38,659; BD: $55,985). Compared to PwHa, PwHb incurred 1.37 and 1.42 times higher BH and AH costs, respectively (p < 0.05). Estimated lifetime cost was 1.76 times higher for PwHb ($22.25 million) than PwHa ($12.62 million) in hypothetical patients born in 2000. CONCLUSIONS: Hemophilia imposes a substantial economic burden across the lifespan, with PwHb incurring higher lifetime costs than PwHa. These real-world estimates can support more informed resource allocation and planning for hemophilia care.