Cost‐effectiveness of fenfluramine as add‐on treatment in the management of Dravet Syndrome: A real‐world multicenter study

作者：Paolo Angelo Cortesi, Carla Fornari, Alessandra Boncristiano, Rita Facchetti, Simona Balestrini, Viola Doccini, Nicola Specchio, Nicola Pietrafusa, Marina Trivisano, Francesca Darra, Alberto Cossu, Domenica Battaglia, Michela Quintiliani, Maria Luigia Gambardella, LG Mantovani, Renzo Guerrini · 发表于：Epilepsia Open · 年份：2025 · DOI：10.1002/epi4.70200 · 被引用次数：2 · 研究领域：Epilepsy research and treatment、Pharmacological Effects and Toxicity Studies、Anesthesia and Sedative Agents

OBJECTIVE: Dravet syndrome (DS) is a rare disease with a high clinical and socioeconomic impact on patients, society, and the healthcare system. The recent approval of therapies such as fenfluramine (FFA) has transformed the treatment landscape; however, data on their cost-effectiveness are still scarce. This study evaluates the real-world cost-effectiveness of adding FFA to existing therapies for DS patients from the Italian National Healthcare System perspective. METHODS: This retrospective multicenter observational study included 124 Italian DS patients initiating FFA as add-on treatment between February 2017 and August 2022 and followed until December 2022 or disenrollment (post-FFA period). Data on drug treatments, healthcare resource utilization, and main outcomes-rescue medication, hospital admission, and status epilepticus (SE) episodes-were collected both in the post-FFA period and in a pre-FFA period matching each patient's follow-up duration (median 2.9 years). Annual per-patient rates and costs, with 95% confidence intervals (CI), were determined for each outcome in both periods. Incremental cost-effectiveness ratios (ICERs) with 95% CIs and cost-effectiveness acceptability curves (CEACs) were also calculated. RESULTS: The median age at FFA initiation was 8.5 years. Mean annual healthcare cost of patients with DS was €5740 (95% CI 2896-9930) in the pre-FFA period. FFA introduction added an annual per-patient drug acquisition cost of €16 476, but it significantly r...