OBJECTIVES: This systematic literature review (SLR) aimed to investigate the economic burden of hemophilia A and B.
METHODS: Searches were conducted in Embase, MEDLINE, and MEDLINE In-Process for English-language articles published from November 2012 through November 2022. This SLR included studies of patients ≥ 12 years with hemophilia A and B with or without inhibitors. Abstracts and full texts were checked for eligibility by one researcher with a 10% quality check by a second researcher.
RESULTS: After screening, 32 studies were included from 6,057 citations. Heterogeneity across studies limited the comparison of results. However, this SLR found that hemophilia A or B is associated with significant economic burden, and the major drivers of costs were hemophilia-related treatment, severe disease, and inhibitor presence. Across several countries, hemophilia-related treatment accounted for 82.5% to 97% of total costs. In Europe, the mean adjusted direct medical costs were 4.7 times higher for severe versus mild hemophilia A or B without inhibitors (€3,949 vs. €844), and mean societal cost was €11,115 higher for patients with severe versus mild disease (2020 cost year). In the United States, patients with severe hemophilia B had 84-fold higher total healthcare costs relative to controls without hemophilia, whereas those with mild disease had 7-fold higher costs. Cost and resource use also were impacted by inhibitor status. Data from Portugal showed that patients with severe hemophilia A or B with inhibitors had 3.3- to 3.5-times higher costs than patients without inhibitors. Patients without inhibitors were less likely to be hospitalized than patients with inhibitors. Significant differences in costs were observed between countries, likely due to variations in sample sizes, disease management, and health system strategies.
CONCLUSIONS: Hemophilia is associated with substantial economic burden due to hemophilia-related treatment. Higher costs were associated with severe disease and presence of inhibitors in several countries.
