POPULATION-LEVEL IMPACT OF EMICIZUMAB IN MODERATE-SEVERE HEMOPHILIA A: A TREATMENT IMPACT MODEL
Author(s)
Josue Hidalgo, PharmD1, Katiuska Moreno, MD2, Lorena Sánchez, MD3, Melissa Diaz Puentes, MSc, MD4, Jose Restrepo, MD MSc5;
1Roche Central America, Alajuela, Costa Rica, 2Hospital Especialidades Portoviejo, Portoviejo, Ecuador, 3Clínica Rendon, Guayaquil, Ecuador, 4Roche, Bogotá, Colombia, 5Roche, Lima, Peru
1Roche Central America, Alajuela, Costa Rica, 2Hospital Especialidades Portoviejo, Portoviejo, Ecuador, 3Clínica Rendon, Guayaquil, Ecuador, 4Roche, Bogotá, Colombia, 5Roche, Lima, Peru
Presentation Documents
OBJECTIVES: Hemophilia A generates a significant clinical burden with considerable direct and indirect costs, including productivity losses. This study assessed the clinical, economic, and societal impact of emicizumab for moderate-to-severe hemophilia A in Ecuador.
METHODS: A population-level model was developed to estimate short- and long-term outcomes over a 25-year horizon, comparing scenarios with and without emicizumab. The model starts in 2020 (local market approval) and projects outcomes for 2025, under current adoption, and until 2045 with increased uptake. Epidemiologic and cost inputs were obtained from public sources. Outcomes included bleeding events, direct and indirect costs avoided, and gained quality-adjusted life years (QALYs).
RESULTS: By 2025, emicizumab reduced bleedings by 7.4% versus no emicizumab (-1,162 bleedings avoided). Patients with inhibitors (INH) experienced a 54.2% reduction (-533), while patients without inhibitors (non-INH) showed a 4.3% reduction (-629). Approximately 73% were joint bleeds. By 2045, assuming increased uptake, bleedings are projected to decrease by 65.2% (-14,219): a 72.8% reduction in INH-patients (-981) and a 64.7% reduction in non-INH-patients (-13,239). Avoided direct medical costs reached USD25.8M (2025) and USD49.3M (2045) in INH-patients, and USD3.0M (2025) and USD99.0M (2045) in non-INH-patients. The model projected a 21.7% decrease in the number of INH-patients by 2045 with increased adoption of emicizumab. By 2045, emicizumab resulted in 119 QALYs, reduced hospitalization days by 51.6%, and avoided 97,271 work-inability days (USD1.57M), with 9,495 avoided by 2025.
CONCLUSIONS: Emicizumab demonstrates reductions in the clinical and socio-economic burden of hemophilia A in Ecuador. Short-term effects reflect high uptake in INH patients, with larger long-term reductions as adoption expands in non-INH patients. The projected decrease in inhibitor prevalence suggests lower risk of future complications and related resource use. Overall reductions in bleeding events and healthcare utilization support a sustained population-level impact of expanded emicizumab adoption in the Ecuadorian context.
METHODS: A population-level model was developed to estimate short- and long-term outcomes over a 25-year horizon, comparing scenarios with and without emicizumab. The model starts in 2020 (local market approval) and projects outcomes for 2025, under current adoption, and until 2045 with increased uptake. Epidemiologic and cost inputs were obtained from public sources. Outcomes included bleeding events, direct and indirect costs avoided, and gained quality-adjusted life years (QALYs).
RESULTS: By 2025, emicizumab reduced bleedings by 7.4% versus no emicizumab (-1,162 bleedings avoided). Patients with inhibitors (INH) experienced a 54.2% reduction (-533), while patients without inhibitors (non-INH) showed a 4.3% reduction (-629). Approximately 73% were joint bleeds. By 2045, assuming increased uptake, bleedings are projected to decrease by 65.2% (-14,219): a 72.8% reduction in INH-patients (-981) and a 64.7% reduction in non-INH-patients (-13,239). Avoided direct medical costs reached USD25.8M (2025) and USD49.3M (2045) in INH-patients, and USD3.0M (2025) and USD99.0M (2045) in non-INH-patients. The model projected a 21.7% decrease in the number of INH-patients by 2045 with increased adoption of emicizumab. By 2045, emicizumab resulted in 119 QALYs, reduced hospitalization days by 51.6%, and avoided 97,271 work-inability days (USD1.57M), with 9,495 avoided by 2025.
CONCLUSIONS: Emicizumab demonstrates reductions in the clinical and socio-economic burden of hemophilia A in Ecuador. Short-term effects reflect high uptake in INH patients, with larger long-term reductions as adoption expands in non-INH patients. The projected decrease in inhibitor prevalence suggests lower risk of future complications and related resource use. Overall reductions in bleeding events and healthcare utilization support a sustained population-level impact of expanded emicizumab adoption in the Ecuadorian context.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE187
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Novel & Social Elements of Value, Work & Home Productivity - Indirect Costs
Disease
SDC: Pediatrics, SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain), STA: Biologics & Biosimilars