Efanesoctocog Alfa Not Cost-Effective for Prophylaxis in Hemophilia A

MONDAY, April 28, 2025 (HealthDay News) — 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.

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