Abstract
BACKGROUND: A range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough bleeding rates). While clinically effective, the cost effectiveness of these treatments has not been established. OBJECTIVE: This study presents a cost-effectiveness analysis of contemporary rFVIII treatments for severe HA patients without inhibitors. METHODS: A published semi-Markov model was used to compare three different prophylaxis regimens: (1) extended half-life (EHL) recombinant Factor VIII (rFVIII) Fc-fusion protein (rFVIIIFc, Eloctate(®), Sanofi), (2) EHL PEGylated rFVIII (PEG-rFVIII, Adynovate(®), Takeda), and (3) standard half-life (SHL) rFVIII (antihemophilic factor [recombinant], Advate(®), Takeda), used as a proxy for all SHL rFVIII treatments. Acquisition costs were included based on published dosing and weight data. Benefits were incorporated through published annualized bleeding rates, rates of target joint development/resolution, and improvements in the modified hemophilia joint health score. Results were presented as total, discounted costs, and quality-adjusted life-years (QALYs). RESULTS: rFVIIIFc was shown to provide the most QALYs (27.922) compared with both PEG-rFVIII (27.454) and SHL rFVIII (27.071), at lower costs. Discounted lifetime costs were estimated at US$18.235m (rFVIIIFc), US$20.198m (PEG-rFVIII), and US$18.285m (SHL rFVIII), and were predominantly affected by model settings related to acquisition costs, patient weight, and dosing. CONCLUSIONS: rFVIIIFc may offer a cost-effective option for severe HA patients. Uncertainties owing to the limited evidence base is the main limitation of the study.