PRO100 EVALUATION OF TREATMENT PATTERNS OF FABRY DISEASE UTILIZING MEDICAL CLAIMS ANALYSES OF A GERMAN SICKNESS FUND DATABASE

Abstract

Fabry disease (FD) is a rare disease due to absent/deficient alpha-galactosidase which has a debilitating impact on affected untreated patients. Analyses were conducted to evaluate the treatment patterns of FD in Germany on a representative database covered by the German statutory health insurance system consisting of 5.5 million individuals during the study period from 2010 – 2017. FD confirmed cases had ICD-10-GM codes (E75.2) for sphingolipidosis and ATCC (Anatomic-Therapeutic-Chemical Classification) code for approved FD therapies. Analyses were conducted on ICD-10-GM codes, EBM-Codes (Einheitlicher Bewertungsmaßstab) for outpatient care, OPS (Operation and Procedure Classification system) codes for inpatient care, and sick leaves documented in the database for four quarters pre-and post-index date (i.e., quarter of first diagnosis). Forty-six FD patients were treated with an approved therapy (agalsidase alfa, n=37; agalsidase beta, n=12; migalastat, n=8) yielding an estimated treated FD prevalence of 0.85 per 100,000 insured patients from 2010-2017. The cohort included 41% (n=19) females; mean age was 42 years (SD=15); mean follow-up was > 5 years (SD=3). Post-index the top three associated comorbidities were essential hypertension (44%), chronic kidney disease (37%), and back pain (33%). Most patients (52%) had at least one sick leave post-index, seven patients (15%) had at least one sick leave pre-index. Overall mean duration of sick leaves was 17 days (median=2) post- and 21 days (median=0) pre-index. Most patients received their first diagnosis in outpatient care (76%, n=35). Specialists consulted in outpatient care post-index were nephrologists (46%), internal medicine (44%) and ophthalmologists (28%). Thirty-three patients (71%) had ≥1 hospital stay post-index, and nine patients (20%) had ≥1 hospital stay pre-index. Overall mean hospital stays pre- and post-index was 0.6 (SD=1.4) and 1.7 (SD=1.7), respectively. The study quantified the burden of FD on patient productivity and healthcare utilization from insurance claims analysis in Germany.