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

Abstract

Background: 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. Objective: 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. Design: Markov model. Data Sources: XTEND-1 study and other published sources. Target Population: Patients with severe hemophilia A. Time Horizon: Lifetime. Perspective: U.S. health system and societal. Intervention: Prophylaxis with efanesoctocog alfa versus standard-care factor VIII. Outcome Measures: Incremental cost-effectiveness ratio (ICER; cost per quality-adjusted life-year [QALY]) and threshold inequality aversion parameter (equity weight). Results of Base-Case Analysis: 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. Results of Sensitivity Analysis: 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. Limitation: Benchmark equity weights (as opposed to hemophilia-specific weights) to interpret DCEA results. Conclusion: 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.