Emicizumab (Hemlibra, Roche Holding AG), a costly new drug to treat the bleeding disorder hemophilia A, could significantly reduce health care expenses for certain patients, according to a draft report from an independent U.S. nonprofit organization that evaluates clinical and cost effectiveness of new medicines.
Emicizumab was approved by the FDA in November as a once-weekly injection for adults and pediatric patients with hemophilia A who have developed inhibitors, or resistance, to other treatments. Roche’s medicine is required to carry a boxed warning, the most serious, about the risk of blood clots.
According to a Reuters report, the Institute for Clinical and Economic Review (ICER) found that, for such patients ages 12 years and older, emicizumab at current wholesale prices would reduce spending by around $1.85 million per patient annually. In patients under 12 years of age, emicizumab would reduce costs by about $720,000 per patient annually.
Hemophilia is a rare bleeding disorder in which a clotting protein is missing or does not function normally. Roche plans to charge about $482,000 for the first year of treatment and $448,000 a year after that.
To avoid joint damage and other complications, patients with severe hemophilia need regular infusions of very expensive clotting factors. About 25% of people with severe hemophilia A develop factor antibodies, called inhibitors, at some point, making it difficult to control bleeding without very high doses of clotting factors or other expensive treatments known as bypassing agents (BPAs), such as activated prothrombin complex concentrate or recombinant activated factor VII.
Hemlibra is an antibody designed to activate the natural coagulation cascade and restore the blood clotting process for hemophilia A.
ICER noted in its analysis that hemophilia is a lifelong disease that creates substantial burdens for patients.
“Our analysis indicates that that emicizumab prophylaxis compared to no prophylaxis and BPA prophylaxis in hemophilia A patients with inhibitors would be cost-saving. Emicizumab was estimated to be more effective and to generate more QALYs [quality-adjusted life years] at lower total cost, both from a health system and societal perspective, compared to no prophylaxis and to BPA prophylaxis,” ICER wrote.
“This finding remained robust over a wide range of sensitivity and scenario analyses. These included analyses of patient age at model entry, reduced mortality, higher bleed rates in patients with target joints, proportion of patients able to use aPCC on demand when treated with emicizumab, and assuming persistence of childhood bleeding reduction into adulthood,” ICER wrote.