Hemlibra is an Effective and Cost-saving Hemophila A Therapy, Report Finds

Hemlibra (emicizumab) is an effective and less costly therapy for hemophilia A patients who have substances in their blood that prevent clotting factor therapies from working as well as they should, according to a therapy rating organization.

The Institute for Clinical and Economic Review (ICER) updated an initial report on Hemlibra after a four-week public discussion period that attracted comments and suggestions from patient groups, doctors, drug manufacturers, and others. A group of outside experts will review the revised report on March 29.

U.S. regulators approved the Genentech therapy in 2017 as a preventive treatment for adults and children with hemophilia A and coagulation factor VIII inhibitors.

ICER concluded in a press release that Hemlibra is far better than no preventive treatment for hemophilia A patients aged 12 years or older who fail to respond to immune tolerance induction therapy.

It also offers small to substantial benefits to children younger than 12 years of age, compared with no preventive therapy, the report said. And it is better for both children and adults than preventive treatment with bypassing agents.

In addition, Hemlibra offers cost savings to the three groups, the report said. The savings “would not necessarily apply to broader populations,” it said.

Another finding was that taking Hemlibra, which is injected under the skin, is more convenient for patients and caregivers than similar IV-administered therapies. This may lead to patients doing a better job of sticking with the treatment, the report said.

The updated report will be discussed again March 29 at a meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) in Cambridge,  Massachusetts. The group is one of three evidence appraisal committees that ICER put together to obtain comments on its draft reports. The groups are composed of medical evidence experts, doctors, patients, and others.

“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,”  David Rind, ICER’s chief medical officer, said in the press release. “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 health care system, and administration is far less burdensome for patients than the previous standard of care.

“During our public meeting later this month, we will work with patients and other stakeholders to identify ways in which the evidence can be used to improve patient care. We will also explore ways to increase affordability for individuals and the health care system given the extremely high costs of treating hemophilia A,” Rind added.