Population-Level Impact of Emicizumab for Hemophilia A Patients in Six Latin American (LATAM) Countries: A 25-Year Model
Author(s)
Josue Hidalgo, PharmD1, Alejandra Baques, MD2, Armando R. Estrada, MD3, Javier M Cortes, MD4, Kimberly Rojas, MD5, Krismely Moya, MD6, Silvana Carolina Oliva Lara, MD7, Daniel Samaca, Psych8, Bruno R. Boietti, MD9, Melissa Diaz, MD8, Laura C. Prieto, MD8, Allan Rimola, MD1;
1Roche, San José, Costa Rica, 2Hospital Cesar Milstein, Ciudad Autónoma de Buenos Aires, Argentina, 3Hospital de Niños Benjamin Bloom, San Salvador, El Salvador, 4Integral Solutions SD SAS, Bogotá, Colombia, 5Caja Costarricense del Seguro Social, San José, Costa Rica, 6Hospital Pediatrico Robert Reid Cabral, Santo Domingo, Dominican Republic, 7Instituto Guatemalteco de Seguridad Social, Ciudad de Guatemala, Guatemala, 8Roche, Bogotá, Colombia, 9Roche, Buenos Aires, Argentina
1Roche, San José, Costa Rica, 2Hospital Cesar Milstein, Ciudad Autónoma de Buenos Aires, Argentina, 3Hospital de Niños Benjamin Bloom, San Salvador, El Salvador, 4Integral Solutions SD SAS, Bogotá, Colombia, 5Caja Costarricense del Seguro Social, San José, Costa Rica, 6Hospital Pediatrico Robert Reid Cabral, Santo Domingo, Dominican Republic, 7Instituto Guatemalteco de Seguridad Social, Ciudad de Guatemala, Guatemala, 8Roche, Bogotá, Colombia, 9Roche, Buenos Aires, Argentina
Presentation Documents
OBJECTIVES: Hemophilia A has a significant burden in LATAM, where healthcare systems are often under financial strain. This research quantifies the impact of emicizumab on disease burden and treatment costs among Hemophilia A patients across six LATAM countries over 25 years.
METHODS: Were analyzed quality-adjusted life years (QALYs), events (bleeds, arthroplasties, deaths), costs (USD), and productivity losses associated with emicizumab in patients with moderate-severe hemophilia A. Sources of information include clinical evidence from HEAVEN trials, local epidemiology, and cost inputs.
RESULTS: As of 2024, 3051, 1670, 607, 3179, 1749, and 257 bleeds have been avoided with the use of emicizumab in Argentina (ARG), Colombia (COL), Costa Rica (CRI), Dominican Republic (DR), El Salvador (SV) and Guatemala (GT), respectively. This represents a reduction between 7-36%. After 25 years, avoided bleeds will range from 1417(CRI) to 24060(ARG). GT and CRI had the smallest populations (n=142, 150), while DR, SV, and GT showed the highest on-demand therapy rates before emicizumab´s introduction. In patients with inhibitors, the additional QALYs gained by 2024 ranged from 1(GT) to 43(ARG). QALYs become non-calculable at 25 years due to complete emicizumab uptake and its impact on population reduction with inhibitors after 10 years. At 25 years, QALYs gained will range from 16(CRI) to 207(ARG) in the non-inhibitor population. As of 2024, the costs saved for treating bleeds, arthroplasties, and hospitalizations were ARG: $178,254,731, COL: $30,302,310, CRI: $22,353,691, DR: $18,023,973, SV: $17,835,969, and GT: $6,615,614. Over 25 years, cost reduction will reach $513,344,425 in all six countries. Total avoided productivity-loss costs will be ARG: $1,016,623, COL: $122,995, CRI: $431,603, DR: $599,068, SV: $530,118, and GT: $585,924, averaging a 65% reduction.
CONCLUSIONS: Emicizumab in hemophilia A is associated with reducing disease burden and increasing the patient's quality of life, resulting in a significant short- and long-term cost reduction for healthcare systems in LATAM countries.
METHODS: Were analyzed quality-adjusted life years (QALYs), events (bleeds, arthroplasties, deaths), costs (USD), and productivity losses associated with emicizumab in patients with moderate-severe hemophilia A. Sources of information include clinical evidence from HEAVEN trials, local epidemiology, and cost inputs.
RESULTS: As of 2024, 3051, 1670, 607, 3179, 1749, and 257 bleeds have been avoided with the use of emicizumab in Argentina (ARG), Colombia (COL), Costa Rica (CRI), Dominican Republic (DR), El Salvador (SV) and Guatemala (GT), respectively. This represents a reduction between 7-36%. After 25 years, avoided bleeds will range from 1417(CRI) to 24060(ARG). GT and CRI had the smallest populations (n=142, 150), while DR, SV, and GT showed the highest on-demand therapy rates before emicizumab´s introduction. In patients with inhibitors, the additional QALYs gained by 2024 ranged from 1(GT) to 43(ARG). QALYs become non-calculable at 25 years due to complete emicizumab uptake and its impact on population reduction with inhibitors after 10 years. At 25 years, QALYs gained will range from 16(CRI) to 207(ARG) in the non-inhibitor population. As of 2024, the costs saved for treating bleeds, arthroplasties, and hospitalizations were ARG: $178,254,731, COL: $30,302,310, CRI: $22,353,691, DR: $18,023,973, SV: $17,835,969, and GT: $6,615,614. Over 25 years, cost reduction will reach $513,344,425 in all six countries. Total avoided productivity-loss costs will be ARG: $1,016,623, COL: $122,995, CRI: $431,603, DR: $599,068, SV: $530,118, and GT: $585,924, averaging a 65% reduction.
CONCLUSIONS: Emicizumab in hemophilia A is associated with reducing disease burden and increasing the patient's quality of life, resulting in a significant short- and long-term cost reduction for healthcare systems in LATAM countries.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE263
Topic
Economic Evaluation
Topic Subcategory
Value of Information
Disease
SDC: Rare & Orphan Diseases, STA: Biologics & Biosimilars