Caregiver impact and health service use in high and low severity Dravet syndrome: A multinational cohort study

Abstract

A R T I C L E I N F O Keywords:Dravet syndrome Health service use Rare disease Developmental encephalopathy Caregiver burden A B S T R A C TPurpose: To estimate costs associated with the current management of Dravet syndrome (DS), explore psychosocial aspects of the disease in caregivers and siblings, and identify patient characteristics associated with higher costs in a large, predominantly European survey cohort of patients and their caregivers conducted in 2016. Methods: Health and social care resource use, productivity and quality of life (QoL) data were summarised. Costs for European five (EU5) countries (France, Germany, Italy, Spain and UK) were calculated and patients with high and low current seizure burden compared. Direct healthcare costs and out-of-pocket costs were calculated using literature reported health service costs and participant reported costs, respectively. Results: Direct annual costs of management of non-seizure-related symptoms ($7929) contributed to approximately 50% of all costs (including medication). Excluding medication, non-seizure-related costs dominated costs of care. Cost for patients with high seizure burden were higher for seizure-related healthcare use and physiotherapy, but lower for other therapies. Most (80%) caregivers reported an influence on their career choices and 28% of those in work had missed over three working days in the past four weeks for emergency or routine needs of their child. Caregivers had little free time, relied on family members for support and respite, and experienced emotional stress and uncertainty about their child's future healthcare needs. Conclusion: Families caring for a DS patient manage considerable social and financial impacts. Total direct costs of DS patients (excluding drugs) are driven by non-seizure-related healthcare use and high seizure burden is associated with higher healthcare costs. Germany comparing direct costs before and after treatment with https://doi.

A multicenter, matched case‐control analysis comparing burden‐of‐illness in Dravet syndrome to refractory epilepsy and seizure remission in patients and caregivers in Germany

“…This analysis revealed that DS patients had the greatest use of ancillary treatments while physiotherapist visits accounted for the highest proportion of visits to all ancillary specialists across the three groups. This aligns with the DISCUSS survey, which reported that physiotherapy accounted for the highest non–seizure‐related mean cost per patient in Germany …”

“…Although more patients in the DS cohort experienced seizures at a shorter frequency, whether and to what extent this accounts for the higher socioeconomic impact of the disease is beyond the scope of this comparative analysis (matching of patients by seizure frequency would not be possible because of the small sample sizes). Nevertheless, a recent study conducted by Lagae et al on a large cohort of 584 DS patients showed that excluding medication, non–seizure‐related costs dominated costs of care, suggesting that the higher costs incurred by patients with DS compared with DRE in this study could be explained in part by the higher burden of their additional symptoms …”

“…This suggests that the impact of disease in the DS population is more severe than in the other two populations and confirms observations that DS is more than a drug-resistant epilepsy syndrome. Indeed, the impact of comorbidities has been shown by Lagae et al 11 and Strzelczyk et al 12 to be a driver of economic cost.…”

“…Studies evaluating the direct and indirect economic burden of epilepsy have reported a high‐resource consumption for drug‐resistant epilepsy (DRE) patients, and low costs once patients are in seizure remission (SR) . The socioeconomic impact for DS patients and their caregivers has been shown to be substantial in several studies . However, DS patients have not previously been compared with patients experiencing DRE or in SR.…”

“…STRZELCZYK ET aL. shown to be substantial in several studies. [10][11][12][13][14] However, DS patients have not previously been compared with patients experiencing DRE or in SR.…”