Healthcare and Economic Impact of Friedreich's Ataxia in Italy: A Stratified Analysis Based On Patients’ Ambulatory Status
Author(s)
Ippazio Cosimo Antonazzo, PhD1, Paolo Angelo Cortesi, PhD2, Lorenzo G Mantovani, ScD2, Richard Lawson, MSc3, Leila Khader, MD4, Claudia Oliviero, M.Sc4, Laura Santoni, Sr., MSc, PharmD4, Antonio Cittadini, MD5, Gabriella Paparella, MD6, Francesco Saccà, MD7.
1PhD, Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy, 2Research Centre on Public Health (CESP), University of Milan-Bicocca, Monza, Italy, 3Biogen, Gaithersburg, MD, USA, 4Biogen Italia, Milano, Italy, 5Department of Translational Medical Sciences, Federico II University, Naples, Italy, 6Scientific Institute IRCCS E. Medea, Conegliano, Italy, 7Department of Neurosciences, Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
1PhD, Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy, 2Research Centre on Public Health (CESP), University of Milan-Bicocca, Monza, Italy, 3Biogen, Gaithersburg, MD, USA, 4Biogen Italia, Milano, Italy, 5Department of Translational Medical Sciences, Federico II University, Naples, Italy, 6Scientific Institute IRCCS E. Medea, Conegliano, Italy, 7Department of Neurosciences, Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
OBJECTIVES: Friedreich ataxia (FA) isa rare, progressive, neurodegenerative disorder. This study aimed to assess FA resource utilization and relative costs from the perspective of the Italian National Healthcare Service (NHS) and society.
METHODS: A multidisciplinary group experienced in treating FA (neurologist, physiatrist, and cardiologist) was surveyed. Patients were classified as ambulatory or non-ambulatory based on their score in the walking/falling questions in the “FA-Activities-of-Daily-Living” (FA-ADL) scale: scores of 0-2 indicated ambulatory, scores of 3 or 4 indicated non-ambulatory status. Resource use was estimated across five FA-ADL composites (walking/falling, dressing/hygiene/feeding, swallowing/speech, bladder function, sitting position) and the prevalence of cardiomyopathy and scoliosis. Annual (e.g., frequency of specialist and emergency room visits, formal/informal caregiver time, physiotherapy) and one-time (i.e., Intensive Care Unit hospitalization, pacemaker implantation, wheelchair, Hoyer lift, speech devices, home/car modifications) resource use was estimated from the experts. Costs, based on NHS tariffs and societal data, were expressed in €/patient/year by multiplying user proportion, frequency, and unit cost by group.
RESULTS: Among non-ambulatory patients, 56% were estimated to have difficulties (score 3-4 in FA-ADL) with dressing/hygiene/feeding, 29% with swallowing/speech, 25.5% with bladder function, and 37.3% with sitting posture. Additionally, 45% had cardiomyopathy. Non-ambulatory patients had an estimated 25.8 specialist visits and 273 days of home health nursing, along with continuous caregiving per annum compared to 8.1 specialist visits and 30 days of home health nursing for ambulatory patients. From the NHS perspective, the estimated annual cost for non-ambulatory patients was €39,843 with also additional one-time costs of €23,428 due to loss of ambulation compared to €8,545 annually for ambulatory patients. When considering the societal perspective, the estimated annual costs per non-ambulatory patient was €99,354 plus €26,917 one-time costs vs €24,507 annually per ambulatory patient.
CONCLUSIONS: Loss of ambulation increases FA patient management costs from both NHS and societal perspectives.
METHODS: A multidisciplinary group experienced in treating FA (neurologist, physiatrist, and cardiologist) was surveyed. Patients were classified as ambulatory or non-ambulatory based on their score in the walking/falling questions in the “FA-Activities-of-Daily-Living” (FA-ADL) scale: scores of 0-2 indicated ambulatory, scores of 3 or 4 indicated non-ambulatory status. Resource use was estimated across five FA-ADL composites (walking/falling, dressing/hygiene/feeding, swallowing/speech, bladder function, sitting position) and the prevalence of cardiomyopathy and scoliosis. Annual (e.g., frequency of specialist and emergency room visits, formal/informal caregiver time, physiotherapy) and one-time (i.e., Intensive Care Unit hospitalization, pacemaker implantation, wheelchair, Hoyer lift, speech devices, home/car modifications) resource use was estimated from the experts. Costs, based on NHS tariffs and societal data, were expressed in €/patient/year by multiplying user proportion, frequency, and unit cost by group.
RESULTS: Among non-ambulatory patients, 56% were estimated to have difficulties (score 3-4 in FA-ADL) with dressing/hygiene/feeding, 29% with swallowing/speech, 25.5% with bladder function, and 37.3% with sitting posture. Additionally, 45% had cardiomyopathy. Non-ambulatory patients had an estimated 25.8 specialist visits and 273 days of home health nursing, along with continuous caregiving per annum compared to 8.1 specialist visits and 30 days of home health nursing for ambulatory patients. From the NHS perspective, the estimated annual cost for non-ambulatory patients was €39,843 with also additional one-time costs of €23,428 due to loss of ambulation compared to €8,545 annually for ambulatory patients. When considering the societal perspective, the estimated annual costs per non-ambulatory patient was €99,354 plus €26,917 one-time costs vs €24,507 annually per ambulatory patient.
CONCLUSIONS: Loss of ambulation increases FA patient management costs from both NHS and societal perspectives.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE497
Topic
Economic Evaluation, Epidemiology & Public Health, Health Service Delivery & Process of Care
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Neurological Disorders, Rare & Orphan Diseases