Efanesoctocog Alfa: Not a Cost-Effective Option for Hemophilia A Prevention

Recent research indicates that using efanesoctocog alfa for prophylactic treatment in patients with severe hemophilia A is neither cost-effective nor sustainable from an economic standpoint, particularly in the United States. The study, published online on April 22 in the Annals of Internal Medicine, assessed the long-term value of this therapy compared to standard care.

Researchers led by Dr. Satoko Ito from Yale School of Medicine employed a Markov model to compare conventional factor VIII treatments—including both standard and extended half-life products—with efanesoctocog alfa. The analysis showed that over a lifetime, patients receiving efanesoctocog alfa gained 19.7 quality-adjusted life years (QALYs), at a total cost of approximately $22.1 million. In contrast, those on standard care accrued 14.6 QALYs at a significantly lower cost of $11.2 million. Consequently, the incremental cost-effectiveness ratio (ICER) stood at a staggering $2.13 million per QALY, far above the typical thresholds used for healthcare decision-making.

Further sensitivity analyses revealed that to make efanesoctocog alfa a cost-effective option, its price would need to be reduced substantially—by at least 47% for standard cost-effectiveness and nearly 41% for distributional cost-effectiveness. This indicates that current pricing renders the therapy financially unviable under typical healthcare budgets. Moreover, probabilistic simulations confirmed that standard-care factor VIII remains the preferred option in 100% of scenarios.

The authors emphasize that ongoing research, longer-term clinical data, and reevaluation of pricing models are essential to understand the potential role of efanesoctocog alfa. Until then, current evidence suggests that its high cost outweighs the clinical benefits, making it an unlikely choice for widespread prophylactic use in hemophilia A patients in the U.S.

For more details, see the original publication: Satoko Ito et al., 'Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A,' Annals of Internal Medicine, 2025.

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