ArticleAnnals of internal medicine2025
Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis.
Article in Annals of internal medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
4 citing papers in PubMed.
- Cost Effectiveness of Efanesoctocog Alfa Versus Factor VIII Extended Half-Life in Adolescent and Adult Patients with Hemophilia A in the USA.PharmacoEconomics · 2026Article
- Evaluating Costs and Efficacy of rFVIII Prophylaxis Using Matching-Adjusted Indirect Comparisons in Hemophilia A.TH open : companion journal to thrombosis and haemostasis · 2026Article
- Anchoring the Hemophilia Joint Health Score for Conventional Value Assessment in the Care of People Living with Severe Hemophilia A.PharmacoEconomics · 2025Article
- Lifetime economic burden of hemophilia using a nationwide real-world healthcare data.PloS one · 2025Article
Corrections and comments
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Authors and funding
10 authors.
Funding
Abstract
backgroundProphylaxis 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.
objectiveTo 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.
designMarkov 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.
interventionProphylaxis 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.
conclusionProphylaxis 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. PRIMARY FUNDING SOURCE: National Institutes of Health; National Heart, Lung, and Blood Institute.
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