ICER Review Finds Hemlibra Lowers Costs and Improves Outcomes in Hemophilia A

Recently, the Institute for Clinical and Economic Review (ICER) released an evidence report that investigated the comparative clinical effectiveness and value of emicizumab (marketed as Hemlibra, developed by Genentech) in the treatment of hemophilia A. The findings of the report were that emicizumab offers improvement in outcomes for patients with hemophilia A and inhibitors to factor VIII, while also lowering the cost of treatment.

The FDA approved emicizumab in November 2017 as prophylaxis for both adults and children with hemophilia A and coagulation factor VIII inhibitors. Factor VIII is a clotting protein that is deficient in patients with hemophilia A.

“Emicizumab appears to be a very rare win-win-win in treating a small population of patients who have hemophilia and cannot be treated with factor VIII. Not only does evidence suggest that the therapy improves patient health outcomes, which has a significant impact on patient quality of life, but it also offers substantial cost savings to the healthcare system, and administration is far less burdensome for patients than the previous standard of care,” said David Rind, MD, MSc, ICER's chief medical officer, in a statement.

ICER’s evaluation found that there is a high certainty of a substantial net health benefit of emicizumab compared with no prophylaxis for patients 12 years and older for whom treatment with immune tolerance induction has been unsuccessful.

In addition, a long-term cost-effectiveness analysis highlighted that it offers cost savings from both a health system perspective considering direct medical costs, as well as from a societal perspective taking into account broader benefits. However, the report noted that this finding only applies to the population studied, not necessarily to broader populations that could be included in expanded indications.

Finally, the review also considered additional benefits of medications not included in clinical trials. When comparing emicizumab with other therapies, caregivers and hemophilia patients noted that the drug offers significantly improved convenience, which researchers noted may improve adherence.

The evidence report will be discussed at an upcoming New England Comparative Effectiveness Public Advisory Council meeting in Cambridge, Massachusetts.