Modelling future usage and cost of factor and emicizumab to treat haemophilia A for the US Western States Region IX haemophilia treatment centres

Introduction: Increased usage of emicizumab in the United States will affect standard half-life (SHL) and extended half-life (EHL) products usage and cost. Aim: To model the usage and cost of SHL and EHL products, and emicizumab to treat haemophilia A (HA) in the 13 Western States Region IX haemophilia treatment centres (HTCs.) (California, Nevada, Hawaii and Guam). Methods: We modelled product usage and cost using decision analysis methods. Variables: epidemiology/demographics, treatment and product cost. Data were from the US Western States Region IX, US Centers for Disease Control and Prevention, American Thrombosis and Hemostasis Network and the literature. Results: Prior to EHL products and emicizumab, the usage of SHL products was similar to 300 million international units (IUs) or 6.8 IUs/capita and a cost of $430 million. With the uptake of EHL and emicizumab, the 2025 estimated usage of factor (SHL and EHL) was 270 million IUs (5.4 IU per capita) and 1,993 grams (40 micrograms/capita) for emicizumab and a cost of $532 million. As the number of HA patients in the region increases by 59%, factor usage increases by 20%, emicizumab usage increases by 26%, and cost increases to $650 million. Conclusion: The entrance of emicizumab into the market may radically change the use of SHL and EHL products. Our model suggests that emicizumab use will likely increase total product costs. While our estimates are most useful for the United States, the effect of emicizumab on factor use will likely be similar in other parts of the world.

Automated summary

The increased use of emicizumab in the United States is projected to have a significant impact on the usage and cost of standard half-life and extended half-life products for treating hemophilia A. By 2025, with a 59% increase in the number of HA patients, factor and emicizumab usage is expected to rise, leading to a total cost of $650 million.