Real-World Clinical Burden of Friedreich Ataxia in the United States

Speaker(s)

Lynch D1, Setyawan J2, Lawson R3, Yang H4, Zhang S4, Jiang A4, Hua Q4, Khan S2, Bajaj A5, Jaramillo R2, Perlman S6
1University of Pennsylvania, Philadelphia, PA, USA, 2Employee of Reata Pharmaceutical at the time of this study. Reata was acquired by Biogen (Cambridge, MA) in 2023, Plano, TX, USA, 3Biogen, Gaithersburg, MD, USA, 4Analysis Group, Inc., Boston, MA, USA, 5Biogen, Cambridge, MA, USA, 6Ronald Reagan UCLA Medical Center, Los Angeles, CA, USA

Presentation Documents

OBJECTIVES: Friedreich ataxia (FA) is a rare, inherited neurodegenerative, progressive disorder that leads to loss of ambulation, multisystem involvement, poor quality of life, and ultimately, premature death. This study assessed real-world clinical burden of FA in the United States.

METHODS: This is a matched case-control study using Komodo Claims Data (2016-2023). Patients with FA were selected as cases; index date was defined as a random date with FA diagnosis; cases were required to have at least 12-month continuous enrollment before and after index. Individuals without early-onset cerebellar ataxia were eligible for controls. Controls were selected by matching to cases at a 5:1 ratio on age, gender, insurance, region, and continuous enrollment; the same index date as cases was assigned to matched controls. Patient characteristics during the 12-month pre-index period were summarized. For selected clinical outcomes, proportions of patients who had an event post-index were summarized and compared between cases and matched controls using generalized equation estimation models.

RESULTS: 652 FA cases (median follow-up: 26.2 months) and 3,260 matched controls (median follow-up: 28.3 months) were included. In both cases and matched controls, the mean age at index date was 33.2 years and 51.4% of patients were female. The incremental clinical burden in FA cases vs. matched controls was high. Post-index, more FA cases had loss of ambulation (62.6% vs. 1.3%; odds ratio [OR]:158.0, p<0.001), cardiomyopathy (41.9% vs. 1.4%; OR:59.2 p<0.001), scoliosis (41.4% vs. 1.6%; OR:49.0, p<0.001), falls (31.4% vs. 6.4%; OR: 7.4; p<0.001), diabetes (21.3% vs. 10.3%; OR:2.5; p<0.001), head injury (17.8% vs. 8.9%; OR:2.4; p<0.001), and fracture (17.6% vs. 6.5%; OR: 3.3; p<0.001) than matched controls.

CONCLUSIONS: Real-world data suggested patients with FA were associated with substantial and incremental clinical burden compared to matched controls and will benefit in advances in treatment for FA.

Code

RWD46

Topic

Clinical Outcomes, Study Approaches

Topic Subcategory

Clinical Outcomes Assessment

Disease

Neurological Disorders, Rare & Orphan Diseases