EE166 Cost-Effectiveness of Treatment Strategies for Severe Hemophilia a Patients in the United States

Abstract

Hemophilia A is a rare genetic bleeding disorder associated with high morbidity and significant financial burden. For bleed prevention, about 80% of patients use prophylaxis, 20% on-demand infusions of either standard half-life factor VIII products (SHL-FVIII) or the newer extended half-life products (EHL–FVIII). Adding heterogeneity to the treatment paradigm, gene therapy is a new promising treatment being evaluated for hemophilia and multiple other genetic pathologies. Our study examined the cost-effectiveness of eight treatment strategies: on-demand and prophylaxis using either SHL-FVIII or EHL-FVIII, with and without gene therapy.