Economic burden associated with tuberous sclerosis complex in patients with epilepsy

Abstract

Data on the economic burden associated with tuberous sclerosis complex (TSC) among patients with epilepsy in the United States (US) are limited. This study aimed to assess all-cause and epilepsy-related healthcare resource utilization (HRU) and healthcare costs in the US among patients with epilepsy and TSC compared with patients with epilepsy but without TSC. Methods: This retrospective study was conducted using the Symphony Health Solutions claims database (April 1, 2017-June 30, 2019. Patients with ≥1 medical claim with a diagnosis code representing epilepsy or seizures were assigned to the cohort with TSC if they had ≥1 medical claim for TSC; the remaining patients were assigned to the cohort without TSC. Patients in the cohort with TSC were exactly matched 1:5 on demographics to patients in the cohort without TSC. All-cause and epilepsy-related HRU, medical charges, prescription drug costs, and the use of antiepileptic drugs (AEDs) were compared between the matched cohorts over the 1-year study period. Results: A total of 2028 patients with epilepsy and TSC were matched to 10,140 patients with epilepsy but without TSC. Patients with TSC were more likely to have a diagnosis code for refractory epilepsy (38.7% vs. 10.2%, p < 0.001) and more likely to have used an AED (89.5% vs. 71.2%, p < 0.001) than patients without TSC over the study period. On average, patients with TSC received 2.1 distinct AEDs versus 1.3 distinct AEDs among patients without TSC. Compared with patients without TSC, patients with TSC had numerically but not statistically higher incidence rates of all-cause outpatient, clinic, office, and other visits; significantly lower rates of all-cause inpatient and emergency room visits (p < 0.001); and statistically significantly higher incidence rates of epilepsy-related outpatient, inpatient, office, and other visits (p ≤ 0.001). All-cause prescription drug costs were significantly higher among patients with TSC than patients without TSC (cost difference per patient: $14,179, p < 0.001). All-cause medical service charges were numerically higher for patients with TSC, but the differences were not statistically significant (charge difference per patient: $4293 for medical services, p = 0.707). Epilepsy-related costs were significantly higher for patients with TSC; the cost difference per patient was $14,639 for prescription costs (p < 0.001), and the charge difference per patient was $16,838 for medical charges (p = 0.019). Conclusion:The results of this study underscore the high epilepsy-related HRU and costs incurred by patients with epilepsy and TSC relative to those incurred by patients with epilepsy but without TSC.

Direct and indirect costs and cost-driving factors in adults with tuberous sclerosis complex: a multicenter cohort study and a review of the literature

“…In terms of the use of healthcare resources, our results appear to be in line with other COI studies, particularly hospitalization frequency and the use of ASDs [ 9 , 11 , 15 , 16 , 37 , 42 , 44 , 45 ]. Most studies could not evaluate mTOR inhibitor use because the periods of data inclusion preceded their authorization.…”

“…However, costs in our cohort were both higher than the annual direct costs reported by studies from the UK (GBP 4227–5054 per year) [ 15 , 16 , 41 ] and lower than the costs reported by two studies from the US (USD 8543–85,397 per year) [ 37 , 38 ]. The direct costs associated with ASD were lower in our study than those reported in previous studies, despite the specific drugs that were reported being similar [ 44 ]. This finding likely reflects lower medication costs in Germany due to price negotiations between statutory health insurers and drug manufacturers in recent years [ 56 ].…”

“…This finding likely reflects lower medication costs in Germany due to price negotiations between statutory health insurers and drug manufacturers in recent years [ 56 ]. Importantly, this finding of lower costs was not true for mTOR inhibitors, which incurred higher costs than were reported by another study from the US [ 44 ]. The costs associated with hospitalizations and outpatient treatment were lower in our study than in earlier studies.…”

“…Most studies could not evaluate mTOR inhibitor use because the periods of data inclusion preceded their authorization. In our study, more patients (37%) used mTOR inhibitors than in three other recent studies (8–16.5%) [ 37 , 44 , 45 ].…”

“…To enable comparisons with other COI studies, we aimed to capture the most comprehensive set of cost items related to epilepsy and other TSC organ manifestations [ 4 , 39 ]. Previous studies have reported direct cost estimates for individuals with TSC in Europe [ 15 , 16 , 40 , 41 ], North America [ 9 , 11 , 37 , 38 , 42 – 44 ], and Asia [ 45 ], but none of these previous studies have provided indirect cost estimates for adults affected by TSC or examined the cost drivers of indirect and nursing care costs [ 4 ].…”