Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis.

Abstract

Prophylaxis goals for patients with severe hemophilia A encompass advancement toward the hemophilia-free mind (freedom from bleeding, pain, arthropathy, and treatment burden). Efanesoctocog alfa, the first ultra-long half-life factor VIII agent that enables once-weekly prophylaxis, shows a 77% improvement in annualized bleeding rate compared with standard-care factor VIII prophylaxis. To evaluate the conventional and distributional cost-effectiveness of prophylaxis with efanesoctocog alfa versus standard-care factor VIII (standard half-life and extended half-life) in the United States. Markov model. XTEND-1 study and other published sources. Patients with severe hemophilia A. Lifetime. U.S. health system and societal. Prophylaxis with efanesoctocog alfa versus standard-care factor VIII. Incremental cost-effectiveness ratio (ICER; cost per quality-adjusted life-year [QALY]) and threshold inequality aversion parameter (equity weight). Efanesoctocog alfa and standard-care factor VIII accrued 19.7 and 14.6 discounted lifetime QALYs at costs of $22.1 million and $11.2 million, respectively, with an ICER of $2.13 million per QALY. The distributional cost-effectiveness analysis (DCEA)-derived threshold equity weight was 6.9, exceeding commonly used U.S. equity standards. Results were consistent across all scenario analyses. Only the price of efanesoctocog alfa could change the conclusion. Efanesoctocog alfa needs to be priced at less than 53% of its current price for conventional cost-effectiveness and less than 59% for distributional cost-effectiveness. Standard-care factor VIII was favored in 100% of 10 000 probabilistic iterations. Benchmark equity weights (as opposed to hemophilia-specific weights) to interpret DCEA results. Prophylaxis with efanesoctocog alfa is not conventionally or distributionally cost-effective for severe hemophilia A in the United States at current pricing and equity weight thresholds. National Institutes of Health; National Heart, Lung, and Blood Institute.