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Emicizumab ‘lowers treatment costs and improves outcomes for patients with hemophilia A’

Treatment with emicizumab improves the health of patients with hemophilia A and inhibitors to Factor VIII, while simultaneously reducing the cost of treatment. These are the findings of the New England Comparative Effectiveness Public Advisory Council (CEPAC), one of the Institute for Clinical and Economic Review’s (ICER) three independent evidence appraisal committees.

David Rind MD, MSc, chief medical officer of ICER, described the drug as a “very rare win-win” in the treatment of the small number of patients with hemophilia A who cannot be treated with factor VIII, an essential blood-clotting protein activated in response to an injury to aid in blood coagulation and healing. Patients with hemophilia A are typically deficient in factor VIII, but in some cases, patients also have inhibitors that interfere with the replacement therapies typically used to treat the condition. 

Emicizumab is a monoclonal antibody that functions in place of a natural blood clotting factor. It goes by the brand name Hemlibra (emicizumab) and in November 2017, the Genentech drug gained FDA approval as a prophylaxis to prevent bleeding in adults and children with hemophilia A who also have coagulation factor VIII inhibitors. The application was granted Priority Review and Breakthrough Therapy, and Orphan Drug designations by the US Food and Drug Administration. It is administered via a weekly subcutaneous (under-the-skin) injection and is a first-in-class therapy that works by bridging other factors to restore blood clotting in patients.  

ICER’s assessment of the emicizumab concluded the drug offers a “high certainty of a substantial net health benefit” compared to patients without any prophylactic treatment. Long-term cost-effectiveness analyses indicated the medication also offers cost savings. These savings were as a result of direct savings to the health system and because of the broader benefits treatment with the drug offers. ICER qualified this by noting the analysis only examined the potential benefits to the currently indicated population. The assessment by ICER highlighted the benefits of emicizumab compared to other therapies, noting that many caregivers and patients observed the drug significantly improves convenience of treatment, which could boost adherence to treatment regimens. 

“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,” Rind asserted. 

Hemophilia A is more common that hemophilia B and affects around one in 5,000 male births, according to figures from the Centers for Disease Control and Prevention. There is no cure for the disease, which is estimated to affect around 20,000 people across the United States. In addition to administering factor VIII blood clotting factors, other treatment options for hemophilia A include taking desmopressin (a drug that releases stored factor VIII from the walls of the blood vessels).