Abstract
OBJECTIVES: Drug-resistant epilepsy (DRE) remains a major clinical challenge, affecting approximately one-third of patients with epilepsy. Cenobamate, a novel antiseizure medication (ASM) approved in Italy in 2022, has shown promise in clinical trials. However, real-world data on its use, especially economic evaluations, remain limited. This analysis assessed the impact of cenobamate in Italian clinical practice, focusing on demographics, clinical traits, treatment patterns, health care use, and costs among patients with focal epilepsy treated or potentially eligible for cenobamate. METHODS: A retrospective observational study was conducted using administrative health care databases covering ~12 million individuals. Adult patients with focal epilepsy were identified and stratified into three groups: (1) overall focal epilepsy population, (2) DRE patients treated with cenobamate, and (3) DRE patients potentially eligible but untreated with cenobamate. Data on demographics, comorbidities, drug utilization, health care resource use, and direct costs were analyzed. Generalized linear models (GLMs) were used to identify predictors of health care and hospitalization costs. RESULTS: Among 3950 patients with focal epilepsy, 1577 (41%) had DRE. Cenobamate-treated patients were younger (mean age 42.6 vs 52.8 years) and had fewer comorbidities. Early adoption trends showed patients starting cenobamate after fewer previous treatments. Cenobamate was associated with a reduction in polypharmacy and lower hospitalization rates and specialist visits. Treatment persistence at 12 months was higher in the cenobamate group (69.6% vs 49.3%). Despite slightly higher overall health care costs (+€916, p < .05), cenobamate significantly reduced hospitalization costs (-€679, p < .05). SIGNIFICANCE: This real-world study highlights a progressively earlier adoption of cenobamate in Italy over the last few years, with patients starting after fewer prior treatments. Compared to patients potentially eligible but untreated with cenobamate, those receiving cenobamate showed greater treatment persistence, reduced polypharmacy, fewer hospitalizations, and fewer neurological visits. Despite higher drug costs, overall health care efficiency improved, supporting its potential as an effective earlier-line option.