OBJECTIVES

Fabry disease (FD) is an inherited, X-linked, lysosomal storage disorder that can affect multiple organs and may progress to chronic kidney disease (CKD), including end-stage renal disease (ESRD). While healthcare resource utilization (HCRU) and related costs are expected to be high in FD, there are limited data on this topic in the US, particularly from the perspective of CKD involvement. Therefore, HCRU and related costs were evaluated to better understand the economic impact of CKD in patients with FD.

METHODS

This retrospective, observational analysis identified patients with a diagnosis code for FD or an FD-related drug code in the IQVIA PharMetrics Plus™ database between October 1, 2014, and September 30, 2019. Patient demographics, comorbid diagnoses of CKD or ESRD, and per-patient HCRU and related costs were analyzed.

RESULTS

A total of 1705 patients with FD (mean age: 36.03 years; 48.97% female) were identified in the database. Mean ± standard deviation (SD) annual per-patient HCRU was greater in FD patients with CKD (n=341) vs without CKD (n=1,549) for emergency department visits (0.77±3.53 vs 0.54±2.03; P<0.01), physician office visits (14.92±13.51 vs 9.75±12.8; P<0.0001), outpatient hospital visits (15.16±31.94 vs 3.41±7.78; P<0.0001), and prescription medications (58.47±70.72 vs 22.81±30.23; P< 0.0001). Patients with CKD incurred mean annual medical costs that were 3.5 times higher than those incurred by patients without CKD ($52,281 vs $14,950; P<0.01). Mean annual costs for patients with ESRD were 2.5 times higher than those for patients with earlier stages of kidney disease ($98,461 vs $34,521; P<0.0001), suggesting that increasing costs positively correlate with deteriorating kidney function.

CONCLUSIONS

Patients with FD and CKD incurred more HCRU-related costs compared with patients without CKD. Therefore, therapies that reduce the risk of CKD involvement in patients with FD are needed to reduce the economic impact of FD.