Objective: The study aimed to investigate healthcare utilization, costs, and the distribution of costs across ambulation levels among patients with Friedreich’s ataxia.

Background: Friedreich’s ataxia (FA) is a rare neurodegenerative disease with multisystemic symptoms which require complex management of treatment and care. Due to the progressive character, patients have an increasing demand for formal and informal care. Healthcare resource utilization allows the investigation of patients’ needs as well as the economic and societal burden of FA.

Method: Data from 61 FA patients from the international, prospective observational PROFA study was used. Healthcare utilization was assessed via an app-based self-assessment, including physician visits, in-hospital stays, medication and medical aids use, therapies and formal and informal care. Utilization and costs were presented descriptively across FARS disability stages combined into ambulation levels (ambulant 1–3, assisted ambulation 4–5, non-ambulant 6) and by country (Germany and Austria, France) from payer and family perspectives. Generalized multilinear models identified cost-associated factors.

Results: Total societal cost was € 54,342 (± 50,542). 32% of costs were covered by payers, while near 70% represented informal care and productivity loss of caregiver and patient. The main cost driver in payer costs was medications, and for family costs productivity loss of the patient. The highest costs were reported for non-ambulant patients. Different patterns of healthcare utilization in FA patients at each ambulation level indicated varying needs and disease management. The costs of FA increased with the disease progression for family costs and total cost, however this pattern was not followed by the payers cost, where the assisted-ambulation group reported the lowest cost. The costs of FA were significantly associated with the ambulation levels and, for the family and total costs, also with the female sex.

Conclusion: FA is a significant financial burden on both healthcare systems and families. Transitions between ambulation levels are particularly critical economically. Identifying patient needs and cost distribution is key to provide effective support for patients and caregivers.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/healthcare-utilization-and-economic-burden-in-patients-with-friedreichs-ataxia-baseline-results-of-the-profa-study/