It is important to understand the burden of F A, including its extent and the most salient elements. The objective of this study is therefore to systematically review the literature regarding the aspects of prevalence, health-related quality of life (HRQoL), and economic outcomes that are associated with F A, and to subsequently identify relevant knowledge gaps. Three systematic literature reviews were conducted to assess publications regarding F A prevalence, HRQoL, and economic outcomes. Search strategies were implemented in MEDLINE (Ovid) and EMBASE databases; study selection and quality assessment were conducted using current best practices. For each review, study characteristics and findings were summarized. A total of 36 studies were included.
Review of prevalence studies (n = 22) indicated variation in the number of cases by region, and many regions not represented at all. Regarding HRQoL (n = 12 studies), physical domains were consistently impacted, although findings regarding other domains and overall HRQoL were less clear. Cost studies (n = 2) encompassed 4 regions and revealed that costs related to the provision of care, including non-medical direct costs and indirect costs, accounted for the majority of F A-related costs. Findings from this systematic review revealed several knowledge gaps that would preclude the conduct of a robust assessment of the benefits and outcomes associated with a disease-modifying F A therapy. Additional understanding regarding patient and caregiver HRQoL and costs is required.