THE ECONOMIC BURDEN OF FRIEDREICH ATAXIA IN EUROPE: 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 that significantly impacts patients and their caregivers. There is a lack of knowledge of the multidimensional burden of FA. This research aimed to quantify healthcare 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 France, Germany, Ireland, Italy, Spain and the UK. 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 366 respondents, 330 were FA patients and 36 were caregivers reporting on patients. Overall, patients averaged 41 years of age and 58% were male. About 60% of patients required assistance with walking, and 14% reported that they needed a wheelchair for mobility or were confined to bed. Almost all patients (96%) 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. Additionally, 9% reported that they utilized emergency or urgent care services in the past year. In addition, 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 healthcare utilization associated with FA in the six European countries represented. 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 FA in Europe.
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 France, Germany, Ireland, Italy, Spain and the UK. 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 366 respondents, 330 were FA patients and 36 were caregivers reporting on patients. Overall, patients averaged 41 years of age and 58% were male. About 60% of patients required assistance with walking, and 14% reported that they needed a wheelchair for mobility or were confined to bed. Almost all patients (96%) 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. Additionally, 9% reported that they utilized emergency or urgent care services in the past year. In addition, 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 healthcare utilization associated with FA in the six European countries represented. 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 FA in Europe.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE108
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
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Neurological Disorders, Rare & Orphan Diseases