OBJECTIVES: Recombinant factor VIII Fc fusion protein (rFVIIIFc) was the first extended half-life (EHL) rFVIII product approved for hemophilia A in the US. Recently, another EHL (PEGylated rFVIII) and monoclonal antibody emicizumab have also been approved. This study aimed to evaluate the cost-effectiveness of rFVIIIFc, PEGylated rFVIII, and emicizumab for the prophylactic treatment of severe hemophilia A patients without inhibitors in the US.

METHODS: A Markov model was constructed to assess the lifetime costs and outcomes associated with rFVIIIFc, PEGylated rFVIII, and emicizumab from a third-party US payer perspective. Efficacy data were sourced from published pivotal trial documentation. Dosing information were identified via product labels and literature searches. The impact of treatment on joint health was assessed via the rates of target joint development and resolution, as well as joint function outcomes via the modified Hemophilia Joint Health Score (mHJHS). Health-related quality of life (HRQoL) data were sourced from published literature. Costs and outcomes were discounted at 3% per annum.

RESULTS: Based on the literature review, rFVIIIFc was associated with improved joint health over time measured by mHJHS; no data regarding improved mHJHS were identified for PEGylated rFVIII or emicizumab, hence no effect was assumed in the base-case. The model included the impact of improved mHJHS on HRQoL and showed that compared with PEGylated rFVIII, rFVIIIFc provided more quality-adjusted life-years (QALYs) (26.15 vs. 25.82) at a reduced cost ($15.64m vs. $17.07m) over a lifetime horizon. Compared with emicizumab, rFVIIIFc provided more QALYs (26.15 vs. 25.83) at a reduced cost ($15.64m vs. $16.10m). Sensitivity analyses showed consistent results.

CONCLUSIONS: rFVIIIFc is the only EHL rFVIII treatment with published evidence demonstrating improved mHJHS. This cost-effectiveness analysis, which includes the impact of treatment on joint health, indicates that rFVIIIFc is associated with lower costs and more QALYs compared to PEGylated rFVIII and emicizumab.