The search retrieved 57 studies, of which 5 fulfilled the eligibility criteria. Among these, 2 reported resource utilization, and 5 reported cost data. No economic evaluation was identified. Neurologists and cardiologists were the most frequently visited physicians, seen by 61-86% and 57-86% of FA patients, respectively. About 23-46% of FA patients were hospitalized for an average of 5-9 days per year. Mean annual direct medical and non-medical cost per patient ranged from £8.893 in the UK to...
Wednesday, July 13, 2022
EE146 Cost and Resource Utilization in Friedreich Ataxia: A Systematic Literature Review
R Zhang, K Buesch; Value in Health, Volume 25, Issue 7, Supplement, 2022, Page S363, doi:10.1016/j.jval.2022.04.396.
CO49 Clinical Efficacy and Safety of Therapeutic Interventions Used in Friedreich Ataxia: A Systematic Review
P Jain, L Badgujar, JA Spoorendonk, K Buesch; Value in Health, Volume 25, Issue 7, Supplement, 2022, Pages S312-S313, doi:10.1016/j.jval.2022.04.147.
In total, 32 relevant publications were identified, of which 24 were randomized controlled trials. These publications investigated idebenone (n=11), recombinant erythropoietin (n=6), omaveloxolone (n=3), amantadine hydrochloride (n=2), and A0001, CoQ10, creatine, deferiprone, interferon-γ-1b, L-cartinine levorotatory form of 5-hydroxytryptophan, luvadaxistat, resveratrol, RT001, vatiquinone (all n=1). Age of study participants ranged from 8 to 73 years and disease duration ranged from 4 to 19.
Natural History of Friedreich's Ataxia: Heterogeneity of Neurological Progression and Consequences for Clinical Trial Design
Christian Rummey, Louise A Corben, Martin Delatycki, George Wilmot, Sub H Subramony, Manuela Corti, Khalaf Bushara, Antoine Duquette, Christopher Gomez, J Chad Hoyle, Richard Roxburgh, Lauren Seeberger, Grace Yoon, Katherine Mathews, Theresa Zesiewicz, Susan Perlman, David R Lynch; Neurology Jul 2022, 10.1212/WNL.0000000000200913; DOI: 10.1212/WNL.0000000000200913
Understanding of the diversity within Friedreich's ataxia populations and their patterns of functional decline provides an essential foundation for future clinical trial design including patient selection and facilitates the interpretation of the clinical relevance of progression detected in Friedreich's ataxia.
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