THE ECONOMIC BURDEN OF FRIEDREICH ATAXIA IN LATIN AMERICA: RESULTS OF AN ONLINE PATIENT AND CAREGIVER SURVEY
Author(s)
Sayeli Jayade, PhD1, Rishi P. Verma, PhD2, Sophie (Juihua) Tsai, MBA, MS, MD3, Michael Andreou, PhD4, Annemarie Drenth, MD4, Adrian Harrington, PhD5.
1OPEN Health, Plainsboro, NJ, USA, 2OPEN Health, Alexandria, VA, USA, 3OPEN Health, Bethesda, MD, USA, 4Biogen, Baar, Switzerland, 5Director, Biogen, Switzerland.
1OPEN Health, Plainsboro, NJ, USA, 2OPEN Health, Alexandria, VA, USA, 3OPEN Health, Bethesda, MD, USA, 4Biogen, Baar, Switzerland, 5Director, Biogen, Switzerland.
OBJECTIVES: Friedreich ataxia (FA) is a progressive rare genetic disorder significantly impacting patients and their caregivers. There is a lack of data on the multidimensional burden of FA, particularly in Latin American countries. We aimed to quantify resource utilization in FA, via a patient and caregiver survey.
METHODS: A cross-sectional online study, guided by a global steering committee (including clinicians, patient advocacy groups, and a person living with FA) was completed by FA patients and caregivers in Brazil and Argentina. Eligible respondents were individuals aged ≥12 years with a self-reported FA diagnosis and adult caregivers (≥18 years) of patients with FA. Data collection began in 2025 and was ongoing as per May 2026.
RESULTS: As of May 2026, of 124 respondents, 116 were FA patients and 8 were caregivers reporting on patients. Overall, patients averaged 33 years of age and 35% were male. About 63% of patients required assistance with walking, and 41% reported that they needed a wheelchair for mobility or were confined to bed. Approximately 70% of patients had at least one caregiver who assisted with FA-related care and needs. Patients with FA visited a range of medical professionals including general practitioners, neurologists and physiotherapists. Moreover, more than half reported that they utilized emergency or urgent care services in the past year. Furthermore, participation in education and employment was frequently affected, even for respondents with less severe disease.
CONCLUSIONS: Our study found a high burden of disease and resource utilization associated with FA in Argentina and Brazil. Distinct patterns of resource utilization were observed at different stages of disease progression. There is a need for treatments that can affect the progression of FA and alleviate the high burden of this disease for patients in Latin America.
METHODS: A cross-sectional online study, guided by a global steering committee (including clinicians, patient advocacy groups, and a person living with FA) was completed by FA patients and caregivers in Brazil and Argentina. Eligible respondents were individuals aged ≥12 years with a self-reported FA diagnosis and adult caregivers (≥18 years) of patients with FA. Data collection began in 2025 and was ongoing as per May 2026.
RESULTS: As of May 2026, of 124 respondents, 116 were FA patients and 8 were caregivers reporting on patients. Overall, patients averaged 33 years of age and 35% were male. About 63% of patients required assistance with walking, and 41% reported that they needed a wheelchair for mobility or were confined to bed. Approximately 70% of patients had at least one caregiver who assisted with FA-related care and needs. Patients with FA visited a range of medical professionals including general practitioners, neurologists and physiotherapists. Moreover, more than half reported that they utilized emergency or urgent care services in the past year. Furthermore, participation in education and employment was frequently affected, even for respondents with less severe disease.
CONCLUSIONS: Our study found a high burden of disease and resource utilization associated with FA in Argentina and Brazil. Distinct patterns of resource utilization were observed at different stages of disease progression. There is a need for treatments that can affect the progression of FA and alleviate the high burden of this disease for patients in Latin America.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE251
Topic
Economic Evaluation, Health Service Delivery & Process of Care, Patient-Centered Research
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Work & Home Productivity - Indirect Costs
Disease
Neurological Disorders, Rare & Orphan Diseases