Overview of the Research System Cost-effectiveness of Emicizumab in Preventing Bleeding for People with Hemophilia A

Abstract

Systematic review research helps provide more scientific evidence for doctors in drug use as well as policymakers for safe, rational, and effective drug use. Emicizumab was first approved by the FDA in 2017 for patients with congenital hemophilia A and was later approved for prophylaxis. However, the high cost of Emicizumab treatment makes it difficult for patients to access the drug as well as pay for this by the insurance fund. A systematic review of studies on Emicizumab in the world that meets the conditions inclusion and exclusion criteria shows that 7 out of 8 studies (accounting for 87.5%) showed results that helped reduce total costs and increase treatment effectiveness compared to the placebo or alternatives. One study (accounting for 12.5%) resulted in increased total costs and reduced effectiveness compared to the intervention substance. Of these seven studies, one study achieved cost-effectiveness, three studies did not achieve cost-effectiveness (because ICER was lower than the willingness-to-pay threshold), and three studies could not be concluded as to whether they achieved cost-effectiveness. Keywords: Systematic review, cost, effectiveness, Emicizumab, Hemophilia A.