Disease Burden in Patients with Friedreich’s Ataxia in the US – Real World Evidence (RWE) Retrospective Claims Analysis



Poster Number: 189


Christene Song, Reata Pharmaceuticals, Daniel Brinza, MD, Reata Pharmaceuticals, Nandini Hadker, Trinity Life Sciences Conrad Tenenbaum, PhD, Trinity Life Sciences, Adrienne Lovink, Trinity Life Sciences, Seemi Khan, MD, Reata Pharmaceuticals, Oliver Jack, Reata Pharmaceuticals

Background and Objective Friedreich’s ataxia (FA) is a rare, inherited, degenerative, neuromuscular disease. Prevalence estimates in the US indicate ~4000 patients are living with FA. Most patients have disease onset before 15 years of age, with a mean duration of 11 to 14 years after disease onset to wheelchair use. This analysis of RWE characterizes comorbidities and healthcare resource utilization associated with FA. Methods Retrospective, longitudinal, anonymous health insurance claims data from a commercially available database were analyzed. Patients diagnosed with FA who were enrolled ≥12 continuous months between 1/2016 and 2/2022 were selected. An age, gender and geographic region-matched non-FA cohort was included 5:1 (non-FA:FA ratio) as a control. Results A total of 2,348 and 11,740 patients were included in FA and control cohorts, respectively. Patients with FA under age 65 had the most significant differences in disease burden as compared to control; data from this age-group are summarized. More patients with FA than control were non-ambulatory (32% vs. 4%). Patients with FA, compared to control, had higher rates of cardiac disease (69% vs. 5%) and type II diabetes (29% vs. 12%). The proportion of patients in the FA cohort requiring 1 or more inpatient hospitalizations was 2X and mean length of stay was 2X that of the control. The proportion of patients using the emergency department (ED) was 2X and mean number of ED visits was 2.5X in the FA cohort as compared to control. The proportion of patients utilizing home health visits was 6X in the FA cohort as compared to control. Conclusions This RWE analysis is the first large-scale, systematic assessment of healthcare claims of patients with FA in comparison to a matched control cohort, which demonstrates greater disease burden for patients with FA.