Efanesoctocog Alfa Not Cost-Effective for Prophylaxis in Hemophilia A
By Elana Gotkine HealthDay Reporter
MONDAY, April 28, 2025 -- For U.S. patients with severe hemophilia A, prophylaxis with efanesoctocog alfa is neither conventionally nor distributionally cost-effective, according to a study published online April 22 in the Annals of Internal Medicine.
Satoko Ito, M.D., Ph.D., from the Yale School of Medicine in New Haven, Connecticut, and colleagues examined the conventional and distributional cost-effectiveness of prophylaxis with efanesoctocog alfa versus standard-care factor VIII (standard and extended half-life) in the United States using a Markov model.
The researchers found that efanesoctocog alfa and standard-care factor VIII accrued 19.7 and 14.6 discounted lifetime quality-adjusted life years (QALYs) at costs of $22.1 and $11.2 million, respectively; the incremental cost-effectiveness ratio per QALY was $2.13 million. The distributional cost-effectiveness analysis-derived threshold equity weight was 6.9, which exceeded U.S. equity standards that are commonly used. Across all scenario analyses, the results were consistent. In a sensitivity analysis, the conclusion could only be changed by the price of efanesoctocog alfa. For conventional and distributional cost-effectiveness, efanesoctocog alfa needs to be priced at less than 53 and less than 59 percent of its current price, respectively. In 100 percent of 10,000 probabilistic iterations, standard-care factor VIII was favored.
"Alternative pricing mechanisms, longer-term clinical follow-up data, and studies across additional jurisdictions where efanesoctocog alfa is approved will be critical to understanding the value of this therapy in the context of existing treatment options in the hemophilia A arena," the authors write.
Several authors disclosed ties to relevant organizations.
Abstract/Full Text (subscription or payment may be required)
Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.
© 2025 HealthDay. All rights reserved.
Posted April 2025
Read this next
FDA Approves Hympavzi for Hemophilia
TUESDAY, Oct. 15, 2024 -- The U.S. Food and Drug Administration (FDA) approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding...
Efanesoctocog Alfa Prophylaxis Beneficial for Children With Hemophilia A
FRIDAY, July 19, 2024 -- For children with severe hemophilia A, once-weekly prophylaxis with efanesoctocog alfa is associated with high sustained factor VIII activity, resulting...
ISTH Issues Clinical Practice Guideline for the Treatment of Hemophilia
MONDAY, July 1, 2024 -- In a clinical practice guideline issued by the International Society on Thrombosis and Haemostasis and published online June 13 in the Journal of...
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.