Drug Offers Cost-Effective Protection for Newborns with Genetic Bleeding Disorder

Severe hemophilia A is a genetic disorder that prevents blood from clotting properly, posing a constant risk of dangerous bleeding episodes. Infants with severe hemophilia A (HA) are at risk of brain bleeds, which can lead to serious long-term brain damage. Emicizumab (Hemlibra) was approved by the FDA in 2018 to prevent and reduce bleeding for patients who suffer from hemophilia A. The HAVEN 7 trial showed that emicizumab is both safe and effective in protecting infants from brain bleeds, although concerns were raised about the affordability of this drug.

A new study led by researchers at the Goshua Lab, based at Yale School of Medicine, found that emicizumab prophylaxis is a cost-effective strategy to give infants more quality years of life compared to not treating them until after they had a brain bleed. According to research, emicizumab cost about $9.8 million over a patient’s life, while the other approach cost about $9.7 million. When comparing cost versus quality of life, standard dose emicizumab provided a good value, while a low-dose approach increased this value.

“We know from natural history data that there is a subset of children with severe hemophilia A who will suffer head bleeds without prophylaxis, some of which will lead to neurologic disability,” said senior author Dr. George Goshua, an assistant professor of medicine (hematology) at Yale School of Medicine and member of Yale Cancer Center. “Protecting this subset of children against head bleed risk with emicizumab prophylaxis in the age 0-2 period appears to be cost-effective in the United States despite the expense of emicizumab”

Samira Glaeser-Khan, a first-year medical student in the Goshua Lab, will present the findings during an oral presentation at the American Society of Hematology (ASH) Annual Meeting in San Diego, Calif. on December 7. Other Yale authors include Rhys Richmond, Satoko Ito, Robert Bona, and Harlan Krumholz. Adam Cuker from the University of Pennsylvania also contributed to this study.