ArticlePharmacoEconomics2026
Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII Extended Half-Life in Adolescent and Adult Patients with Hemophilia A in the USA.
Article in PharmacoEconomics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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4 authors.
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Abstract
BACKGROUND AND
objectiveEfanesoctocog alfa is a first-in-class high-sustained factor VIII therapy approved for prophylaxis, on-demand treatment, and peri-operative management of bleeding in hemophilia A. This analysis aimed to compare the cost effectiveness of efanesoctocog alfa prophylaxis with factor VIII extended half-life prophylaxis.
methodsA lifetime Markov model was developed from a US payer perspective, using clinical data from an indirect treatment comparison of phase III studies and inputs derived from the literature. A cohort of patients aged ≥ 12 years with severe hemophilia A without inhibitors, who received prophylaxis using any regimen or on-demand treatment, entered the model. Outcomes included joint and non-joint bleeds, quality-adjusted life-years, total direct costs, and the incremental cost-effectiveness ratio. Costs were expressed in US dollars and inflated to January 2023 prices. Discount rates of 3% were used. One-way probabilistic and scenario analyses were conducted. The willingness-to-pay threshold was assumed at $150,000 per quality-adjusted life-year.
resultsEfanesoctocog alfa was more effective and less costly (also referred to as 'dominant') versus factor VIII extended half-life therapies, with a lower lifetime number of joint (undiscounted 34.00 vs 140.65) and non-joint (undiscounted 13.33 vs 55.99) bleeds, higher quality-adjusted life-years (24.00 vs 22.92), and lower total costs ($30,716,640 vs $32,953,485). A broad range of scenario analyses and probabilistic sensitivity analyses resulted in 100% of simulations being cost effective. Dosing level and drug costs had the largest impact on results in the deterministic sensitivity analyses.
conclusionsOur analysis suggests that efanesoctocog alfa was dominant versus prophylaxis with standard and elevated factor VIII extended half-life dosing regimens. Efanesoctocog alfa was associated with better joint health and, hence, contributed to fewer bleeds, lower costs, and higher quality-adjusted life-years.
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