TOPLINE:
Prophylaxis with efanesoctocog alfa, while reducing annualized bleeding rate by 77% compared with standard-care factor VIII in severe hemophilia A, is not cost-effective at current US pricing, requiring a > 47% price reduction to meet cost-effectiveness thresholds.
METHODOLOGY:
- A Markov cohort model was constructed using data from the XTEND-1 study, which enrolled previously treated patients aged ≥ 12 years without inhibitors, with a mean age of 34 years.
- Analysis included weekly prophylaxis with efanesoctocog alfa (50 IU/kg) versus standard-care factor VIII prophylaxis (octocog alfa 25 IU/kg three times weekly) over a lifetime horizon.
- Researchers evaluated both conventional and distributional cost-effectiveness from United States health system and societal perspectives, using a willingness-to-pay threshold of $100,000 per quality-adjusted life year.
- Model validation included comparing projected annualized bleeding rates with XTEND-1 study findings and real-world bleeding outcome data from a global cohort of 225 persons with severe hemophilia A.
TAKEAWAY:
- Efanesoctocog alfa prophylaxis accrued 5.11 additional lifetime discounted quality-adjusted life years and $10.9 million in additional lifetime discounted costs compared with standard-care factor VIII, resulting in an incremental cost-effectiveness ratio of $2.13 million per quality-adjusted life year (95% CI, $1.2-3.2 million).
- The price of efanesoctocog alfa would need to decrease to less than 53% of its current price to meet the cost-effectiveness threshold of $100,000 per quality-adjusted life year.
- Standard-care factor VIII was favored in 100% of the 10,000 Monte Carlo simulations in probabilistic sensitivity analysis.
- According to the authors, the distributional cost-effectiveness analysis showed that standard-care factor VIII was favored when using an inequality aversion parameter of 3.0, the higher end of commonly used values in the United States.
IN PRACTICE:
“Although efanesoctocog alfa is a promising new prophylactic therapy for patients with severe hemophilia A, its substantial cost undermines its value from a societal perspective relative to standard-care factor VIII replacement therapy,” wrote the authors of the study.
SOURCE:
The study was led by Satoko Ito, MD, PhD, and Kunal Potnis, MD, Yale School of Medicine in New Haven. It was published online on April 22 in Annals of Internal Medicine.
LIMITATIONS:
The researchers noted that the study relied on benchmark equity weights rather than hemophilia-specific weights for interpreting distributional cost-effectiveness analysis results. Additionally, the long-term efficacy and safety of efanesoctocog alfa have yet to be established, and the cost-effectiveness analysis will need to be refined with longer-term follow-up data. The investigators could not compare efanesoctocog alfa with emicizumab due to the lack of direct or indirect comparative studies.
DISCLOSURES:
The study was supported by the NOMIS Foundation, the Frederick A. DeLuca Foundation, Yale Cancer Center, the Yale Bunker Endowment, and grants from the National Heart, Lung, and Blood Institute and National Cancer Institute of the National Institutes of Health.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.