Interventions of Interest:
- Factor VIII: Factor VIII concentrate is given intravenously, whether administered on-demand or prophylactically. Prophylaxis is administered multiple times per week, which is burdensome.
- Emicizumab (Hemlibra®, Genentech): a monoclonal antibody with dual targets that allow it to bridge activated factor IX and factor X, the role normally played by activated factor VIII in the clotting cascade. Emicizumab was approved by the US Food and Drug Administration (FDA) as a prophylactic treatment for hemophilia A in patients who have inhibitors to factor VIII in 2017 and in those without inhibitors in 2018.
- Valoctocogene roxaparvovec (Roctavian ™; BioMarin): gene therapy with valoctocogene roxaparvovec results in factor VIII production in the liver, but not in the cells in the liver that normally produce factor VIII.
Hemophilia A is a condition of increased tendency to bleed due to an inherited deficiency of factor VIII, a protein that aids in blood-clotting. Hemophilia A has X-linked recessive inheritance, and so predominately affects males. It is the most common of the hemophilias with an incidence of one in 5,000 male births. Patients with hemophilia A, particularly those with severe disease, are at risk for life-threatening bleeding, including intracranial bleeding, but bleeding into a joint (hemarthrosis) or muscle is more common and can lead to substantial disability. Hemarthroses cause ongoing joint inflammation and damage and also increase the likelihood of further bleeding into the same joint.
The independent appraisal committee voted that evidence was adequate to demonstrate clinical superiority of emicizumab over factor VIII prophylaxis; emicizumab is also cost saving, but only because factor VIII prices remain so high.
Below you will find the final documents from the assessment review process:
“Emicizumab reduces burdens on patients and caregivers and may improve patient outcomes while producing overall cost savings in the US healthcare system. However, emicizumab can be cost-saving at its own very high price only because it is being compared to the unnecessarily high costs in the US of prophylaxis with factor VIII. These prices — in a space with multiple products and manufacturers — represent a market that is failing people with hemophilia, who face financial toxicity from insurance cost sharing and the erosion of access due to escalating costs. Ultimately, prices that exceed the value to patients harm everyone, and especially vulnerable patients and families, trying to keep up with the rising costs of health insurance.”