Abstracts

Rationale: Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are rare childhood developmental and epileptic encephalopathies that are often treated with multiple antiseizure medications. Current insight into treatment preferences of patients or their caregivers is limited. This study evaluated the treatment selection preferences of DS or LGS caregivers across the USA, UK, and Germany.


Methods: This mixed-methods study collected qualitative and quantitative data. Primary caregivers of patients with DS (2–21 years old) or LGS (2–55 years old) completed an online stated-preference survey to elicit benefit-risk tradeoffs among 3 unlabeled hypothetical treatments A (soticlestat-like), B (cannabidiol-like), and C (fenfluramine-like) defined by attributes with varying levels (Table 1). After the survey, they attended focus groups (conducted January–March 2024) to explore drivers of their choices and elicit additional benefit-risk tradeoffs. Treatment attributes included seizure frequency reduction, risk of decreased appetite or somnolence, heart/liver monitoring requirements, and potential drug-to-drug interactions. Attributes were developed from package inserts, peer-reviewed publications, expert clinical opinion, and caregiver input. Caregivers were not told which medicines the profiles reflected. The survey evaluated caregivers’ tolerance of changes in one treatment attribute at a time. Focus groups evaluated tradeoffs they would consider between efficacy, adverse events, and other factors. A follow-up question elicited their propensity to switch choices if their preferred medicine was only available as an oral pill.


Results: Eighteen DS and 17 LGS caregivers completed the initial online survey for medicine choice (Table 2). Most (30/35) preferred treatment A (soticlestat-like), 1 LGS and 3 DS caregivers preferred treatment C (fenfluramine-like), and 1 LGS caregiver preferred treatment B (cannabidiol-like). Of those who attended focus groups, 15/17 DS and 16/16 LGS caregivers selected treatment A. Primary decision drivers included the avoidance of hospital visits and check-ups associated with organ monitoring, and fewer medication interactions. Caregivers also preferred easily administered medications, such as oral solutions or pills that could be crushed, particularly for younger children. Most DS caregivers (15/18) would accept a medicine offering a ≤ 50% reduction in convulsive seizure frequency and most LGS caregivers (15/17) a medicine offering a ≤ 15% reduction in drop seizure frequency if invasive safety monitoring could be avoided (Table 1).

Conclusions: Findings from the stated-preference exercise and focus groups suggested DS or LGS caregivers may prefer a treatment with fewer medication interactions and no additional monitoring requirements. Caregivers made tradeoff decisions that consistently tried to maximize benefits while minimizing risks. The study highlights the need for new treatment options that address the complexities of care for individuals with DS or LGS while improving quality of life and treatment burden.

Funding: Takeda Development Center Americas, Inc.