Healthcare Resource Utilization Among Fabry Disease Patients in the U.S.: Insights From a Real-World Retrospective Study
Author(s)
Khashayar Azimpour, MD, PhD1, Lucas Oliveira, PhD, MBA2, Brooklyn Stanley, MSc2, Irene Koulinska, MD, ScD2;
1Chiesi, Toronto, ON, Canada, 2Chiesi, Boston, MA, USA
1Chiesi, Toronto, ON, Canada, 2Chiesi, Boston, MA, USA
Presentation Documents
OBJECTIVES: Fabry disease (FD) is a rare X-linked lysosomal storage disorder with systemic manifestations including cardiovascular, renal, and neurological complications. This study leverages real-world data from the Komodo Commercial Claims and Encounters (CCAE) database to evaluate healthcare resource utilization (HCRU) among FD patients in the U.S. for the year 2022, segmented by treatment categories.
METHODS: A retrospective cohort design was used to analyze claims data for FD patients, identified using ICD-10 codes. Patients were grouped into five FD treatment categories: never treated (n=1,212), untreated (n=260), sparsely treated (n=139), newly treated (n=53), and treated (n=365). Outcomes included overall inpatient and emergency room visits, physician consultations, procedures, and diagnostic assessments.
RESULTS: In 2022, the untreated group had the higher inpatient visit rates compared to the treated group (18.1% and 12.1%, respectively). Similarly, emergency room visits followed this trend, with the untreated group exhibiting a higher rate of 33.5% compared to 25.2% in the treated group. Physician office visits were dominated by cardiology in most groups, though GP/PCP visits were most frequent in the newly treated group. Across all groups, the top three procedures were ECG, echocardiography, and brain imaging (MRI, CT).
CONCLUSIONS: FD treatment was associated with numerically lower rates of inpatient and emergency room visits, compared to the untreated group. These findings underscore the potential benefits of FD treatment in reducing HCRU. Further research is needed to address population heterogeneity and enhance real-world evidence to inform clinical and economic decision-making for FD management.
METHODS: A retrospective cohort design was used to analyze claims data for FD patients, identified using ICD-10 codes. Patients were grouped into five FD treatment categories: never treated (n=1,212), untreated (n=260), sparsely treated (n=139), newly treated (n=53), and treated (n=365). Outcomes included overall inpatient and emergency room visits, physician consultations, procedures, and diagnostic assessments.
RESULTS: In 2022, the untreated group had the higher inpatient visit rates compared to the treated group (18.1% and 12.1%, respectively). Similarly, emergency room visits followed this trend, with the untreated group exhibiting a higher rate of 33.5% compared to 25.2% in the treated group. Physician office visits were dominated by cardiology in most groups, though GP/PCP visits were most frequent in the newly treated group. Across all groups, the top three procedures were ECG, echocardiography, and brain imaging (MRI, CT).
CONCLUSIONS: FD treatment was associated with numerically lower rates of inpatient and emergency room visits, compared to the untreated group. These findings underscore the potential benefits of FD treatment in reducing HCRU. Further research is needed to address population heterogeneity and enhance real-world evidence to inform clinical and economic decision-making for FD management.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE478
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Rare & Orphan Diseases