Abstract
INTRODUCTION: Emicizumab is a first-in-class monoclonal antibody, recently authorized for the treatment of hemophilia A with inhibitors. This study aims to estimate the direct and indirect costs of the management of hemophilia A with inhibitors, in adult and pediatric patients, including the prophylaxis with emicizumab. METHODS: We calculated the costs of the on-demand and prophylactic treatments with bypassing agents (activated prothrombin complex concentrate and recombinant activated factor VII) and the emicizumab prophylaxis, from the societal perspective, over 1 year. The study considered direct healthcare costs (drugs, visits, tests, and hospitalizations), direct non-healthcare costs (informal caregivers), and indirect costs (productivity loss). Data were obtained from a literature review and were validated by an expert group. Costs were expressed in 2019 euros. RESULTS: Our results showed that the annual costs of the prophylactic treatment per patient varied between €543,062.99 and €821,415.77 for adults, and €182,764.43 and €319,826.59 for children, while on-demand treatment was €532,706.84 and €789,341.91 in adults, and €167,523.05 and €238,304.71 in pediatric patients. In relation to other prophylactic therapies, emicizumab showed the lowest costs, with up to a 34% and 43% reduction in the management cost of adult and pediatric patients, respectively. It reduced the bleeding events and administration costs, as this drug is less frequently administered by subcutaneous route. Emicizumab prophylaxis also decreased the cost of other healthcare resources such as visits, tests, and hospitalizations, as well as indirect costs. CONCLUSION: In comparison to prophylaxis with bypassing agents, emicizumab reduced direct and indirect costs, resulting in cost savings for the National Health System and society.