Cost-effectiveness study of prophylaxis with emicizumab versus bypassing agents in patients with severe hemophilia A in Peru

Abstract

Setting Hemophilia is a coagulation disorder that occurs in one in 5000 male births. Patients with un-treated severe hemophilia A have hemorrhagic complications, including joint bleeds and de-creased survival. Emicizumab is a monoclonal antibody approved by the United States for routine prophylaxis of pediatric and adult patients with severe hemophilia A with factor VIII inhibitors. Objectives To perform a cost-effectiveness study of emicizumab prophylaxis for children and adults with severe hemophilia A compared with the current disease management in the Peruvian Ministry of Health and the Social Security Health Insurance. Methods The patient transition between medical states was modeled with the Markov methodology, and the lifetime costs and incremental effects of emicizumab compared to current management were estimated. The budget impact of emicizumab was estimated by projecting annual net costs and its five-year present value. Results In the Ministry of Health, emicizumab would generate savings between 14.6 and 16.0 per child and 11.8 per adult, in current US$ million. Social Security Health Insurance savings would be 12.8 to 14.9 per child and 40.1 per adult. In addition, this strategy would generate effectiveness gains, measured in quality-adjusted life-years, of 0.36 per child and 0.56 per adult and 0.25 per child, and 0.36 per adult in those respective institutions. The budget impact would be a net annual saving of 12.8 and 15.0 US$ million in those entities. Conclusion The current management of hemophilia A is very costly and has health outcomes inferior to those possible with emicizumab. This drug would produce significant savings and better patient health. The Ministry of Health and the Social Health Insurance should implement hemophilia prophylaxis and treatment protocols and finance this drug.