Long-Term Impact of Emicizumab in Hemophilia A (HA): Analysis by Inhibitor Status in Three Latin American (LATAM) Countries
Author(s)
Josue Hidalgo, PharmD1, Adriana Bustinza, MD2, Jaime Garcia, MD3, Nancy Loayza, MD4, Juan Cristobal Morales, MD5, Andrés Valenzuela, MD6, Laura Villarreal, MD7, Melissa Diaz, MD8, Denisse Cañete, MD9, Paula Soledad Luque, Sr., MD10, Ana Maria Ramos, MD11, Emilio Muciño, MSc12, Jose Restrepo, MD13.
1Hemophilia Access & Value LATAM Lead, Roche LATAM, San José, Costa Rica, 2Sociedad Peruana de Hematología, Lima, Peru, 3Instituto Mexicano de Seguridad Social, Ciudad de México, Mexico, 4Hospital Nacional Dos de Mayo, Lima, Peru, 5Hospital Dr. Sótero del Río, Santiago, Chile, 6Pontificia Universidad Católica de Chile, Santiago, Chile, 7Hospital Universitario Dr. José Eleuterio González, Ciudad de México, Mexico, 8Roche, Bogotá, Colombia, 9Roche, Santiago, Chile, 10Roche, José León Suarez, Argentina, 11Productos Roche S.A, Bogotá, Colombia, 12Productos Roche S.A. de C.V., DF, Mexico, 13Roche Peru, Lima, Peru.
1Hemophilia Access & Value LATAM Lead, Roche LATAM, San José, Costa Rica, 2Sociedad Peruana de Hematología, Lima, Peru, 3Instituto Mexicano de Seguridad Social, Ciudad de México, Mexico, 4Hospital Nacional Dos de Mayo, Lima, Peru, 5Hospital Dr. Sótero del Río, Santiago, Chile, 6Pontificia Universidad Católica de Chile, Santiago, Chile, 7Hospital Universitario Dr. José Eleuterio González, Ciudad de México, Mexico, 8Roche, Bogotá, Colombia, 9Roche, Santiago, Chile, 10Roche, José León Suarez, Argentina, 11Productos Roche S.A, Bogotá, Colombia, 12Productos Roche S.A. de C.V., DF, Mexico, 13Roche Peru, Lima, Peru.
OBJECTIVES: To evaluate the clinical, economic, and social impact of emicizumab for moderate-to-severe HA patients with and without inhibitors in Peru(PE), Mexico(MX), Chile’s public(CL-PUB) and private(CL-PRIV) health systems. Modeling short-term outcomes under current adoption rates and long-term impacts over 25 years, assuming widespread use.
METHODS: A treatment impact model estimated quality-adjusted life years(QALYs), bleeding events, hospitalization days, arthroplasties, direct medical costs, and productivity losses (indirect costs) in patients treated with emicizumab versus no emicizumab. Clinical efficacy inputs from HAVEN trials, and country-specific epidemiology, treatment patterns, and costs were used.
RESULTS: Up to 2024, for both populations, emicizumab reduced bleeding events in PE:-3,924(-14.2%), CL-PUB:-742(−13%), CL-PRIV:-607(−12.2%), MX:-2,105(−13.6%), with total direct costs averted of: PE:USD-1,587,275,617, CL-PUB:USD-31,557,433, CL-PRIV:USD-39,782,543, MX:USD-86,318,012. Over 61% of these benefits were driven by the inhibitor population, where adoption is already high. Over 25-years, bleeding events in patients with inhibitors will be reduced: PE:-2,450(-80%), CL-PUB:-589(−71.4%), CL-PRIV:-347(-70.6%), MX:-1,323(-68.5%), and decrease in hospitalization days: PE:-803(-65.4%), CL-PUB:-829(−74.4%), CL-PRIV:-452(-76.8%), MX:-540(-60.5%). Cost reductions in inhibitors patients across all countries will reach USD-1,671,114,168 with avoided productivity losses ranging from CL-PRIV:USD-25,885 to MX:USD-143,744 (average:-72.3%). QALYs become non-calculable when complete emicizumab uptake is achieved due to reduction in population with inhibitors. Peak QALYs gains will be reached between 2025 to 2038 with PE:42, CL-PUB:27, CL-PRIV:11, MX:18. Assuming higher adoption, patients without inhibitors will experience bleeding events reductions of PE:-10,797(-65.3%), CL-PUB:-2,192(−58.8%), CL-PRIV:-2,605(-62.8%), MX:-7,153(-63.3%), with avoided hospitalization days of CL-PUB:-181, CL-PRIV:-19, MX:-294, and QALYs gained: PE: 104, CL-PUB: 35, CL-PRIV: 28, MX:72. Cost reductions across countries in non-inhibitors will reach USD-1,162,408,433 and avoided productivity losses between CL-PRIV:USD-29,734 to MX:USD-400,524 (average:-60.75%).
CONCLUSIONS: While high adoption of emicizumab has already benefited patients with inhibitors, findings support national strategies to expand access to emicizumab to those without inhibitors. Broader adoption can further reduce disease burden, close outcome gaps, and support more equitable and sustainable hemophilia care across LATAM.
METHODS: A treatment impact model estimated quality-adjusted life years(QALYs), bleeding events, hospitalization days, arthroplasties, direct medical costs, and productivity losses (indirect costs) in patients treated with emicizumab versus no emicizumab. Clinical efficacy inputs from HAVEN trials, and country-specific epidemiology, treatment patterns, and costs were used.
RESULTS: Up to 2024, for both populations, emicizumab reduced bleeding events in PE:-3,924(-14.2%), CL-PUB:-742(−13%), CL-PRIV:-607(−12.2%), MX:-2,105(−13.6%), with total direct costs averted of: PE:USD-1,587,275,617, CL-PUB:USD-31,557,433, CL-PRIV:USD-39,782,543, MX:USD-86,318,012. Over 61% of these benefits were driven by the inhibitor population, where adoption is already high. Over 25-years, bleeding events in patients with inhibitors will be reduced: PE:-2,450(-80%), CL-PUB:-589(−71.4%), CL-PRIV:-347(-70.6%), MX:-1,323(-68.5%), and decrease in hospitalization days: PE:-803(-65.4%), CL-PUB:-829(−74.4%), CL-PRIV:-452(-76.8%), MX:-540(-60.5%). Cost reductions in inhibitors patients across all countries will reach USD-1,671,114,168 with avoided productivity losses ranging from CL-PRIV:USD-25,885 to MX:USD-143,744 (average:-72.3%). QALYs become non-calculable when complete emicizumab uptake is achieved due to reduction in population with inhibitors. Peak QALYs gains will be reached between 2025 to 2038 with PE:42, CL-PUB:27, CL-PRIV:11, MX:18. Assuming higher adoption, patients without inhibitors will experience bleeding events reductions of PE:-10,797(-65.3%), CL-PUB:-2,192(−58.8%), CL-PRIV:-2,605(-62.8%), MX:-7,153(-63.3%), with avoided hospitalization days of CL-PUB:-181, CL-PRIV:-19, MX:-294, and QALYs gained: PE: 104, CL-PUB: 35, CL-PRIV: 28, MX:72. Cost reductions across countries in non-inhibitors will reach USD-1,162,408,433 and avoided productivity losses between CL-PRIV:USD-29,734 to MX:USD-400,524 (average:-60.75%).
CONCLUSIONS: While high adoption of emicizumab has already benefited patients with inhibitors, findings support national strategies to expand access to emicizumab to those without inhibitors. Broader adoption can further reduce disease burden, close outcome gaps, and support more equitable and sustainable hemophilia care across LATAM.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE569
Topic
Economic Evaluation, Epidemiology & Public Health
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Value of Information
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases