OBJECTIVES: The objective was to develop an understanding of healthcare resource utilisation associated with the current management of Dravet syndrome (DS) in a large pan-European survey cohort of patients and their caregivers (DISCUSS) conducted in 2016.

METHODS: Quality of life (family, career, leisure, childcare), disease severity (diagnosis journey, epilepsy management, comorbidities) and support (therapy, social and financial support, out of pocket expenses, healthcare system use) data were summarised statistically by age groups. Healthcare costs were based on participant only information and literature reported health service costs, personal costs on input data from participants only.

RESULTS: The survey cohort consists of 584 caregivers of paediatric (93%) and adult (17%) patients with DS (<1 year – 48 years old, mean age 10 years, median 9 years), of which over 90% reside in Europe. The mean EQ5D-5L index value for patients two years or older (completed in proxy by the caregiver) was very low (0.42 ±0.29, range -0.35 – 1). Most (80%) caregivers reported that caring for a child with DS had influenced their career choices. Nearly a third (30%) were unemployed. Of those in work (65%), 28% percent had missed over three working days in the past four weeks to care for the needs of their son or daughter (e.g. emergency care, routine visit to doctor). Half of patients required at least one emergency admission and 46% at least one ambulance call in the past 12 months. Although emergencies decreased with age, these remained notably high in adult patients, of which 28% reported at least one emergency admission in the last 12 months. Annual healthcare utilization costs (not including drugs) are on average $1467 per patient in the EU5 countries.

CONCLUSIONS: Families caring for a member with DS must manage multiple lifelong impairments in addition to refractory epilepsy symptoms alongside considerable social and financial impacts.