Presentation (CTI)

OBJECTIVES: To comprehensively quantify the economic burden of haemophilia B (HB) in Europe by synthesizing recent evidence on healthcare costs and resource utilization. This review evaluates the impact of disease severity, treatment regimens, inhibitor status, and age on financial and healthcare resource outcomes.
METHODS: A systematic literature review was conducted across MEDLINE, Embase, and the Tufts CEA Registry for European studies published between 2015 and 2025. Studies reporting costs or healthcare resource utilization (HRU) in HB were included. Data were extracted and categorized by disease severity, treatment modality, and cost components
RESULTS: Fourteen studies (sample sizes ranging from 31 to 1,331) were included. HB-related direct costs were primarily driven by severity: annual costs for severe HB (€177,213-€206,997) were nearly tenfold higher than moderate HB (€17,796-€41,525). Clotting factor replacement therapy (CFRT) was the dominant driver, representing >95% of direct costs in severe cases. Prophylaxis annual costs (€130,000-€191,539) significantly exceeded on-demand treatment (€6,506-€11,353). The presence of inhibitors increased costs more than threefold (€134,032 vs. €40,318). HRU was substantial: 77.7% of patients required GP consultations, 56.8% saw specialists, and up to 27.0% were hospitalized. Informal care for severe HB averaged 11.85 hours/week. Paediatric annual costs (€104,618) were notably higher than adult costs (€43,173), reflecting higher prophylaxis adoption.
CONCLUSIONS: HB imposes a substantial economic burden in Europe, predominantly driven by severe disease and prophylaxis. While CFRT is the primary cost component, high HRU and significant caregiver burden underscore a major societal impact. These findings highlight a critical need for therapeutic strategies that reduce treatment intensity and long-term complications to alleviate the total economic strain on healthcare systems and society.