Co-creation of digital outcome measures for Dravet syndrome: a patient and public involvement approach (Preprint)

BACKGROUND Dravet syndrome is a complex developmental and epileptic encephalopathy characterized by treatment-resistant seizures and multiple comorbidities that significantly affect quality of life. Traditional clinic-based assessments often fail to capture real-world functional abilities and behavioral changes. OBJECTIVE This project aimed to explore the feasibility of co-creating digital outcome measures with caregivers to inform future clinical research. METHODS A multi-stage Patient and Public Involvement activity was conducted in collaboration with a patient advocacy organization and a digital health company. The process included a literature review, a caregiver survey to identify meaningful aspects of health, a design workshop to refine priorities and technology preferences, and usability testing of a prototype app. The app incorporated questionnaires, seizure diaries, and video-based tasks designed to reflect daily functional abilities. Feedback was collected through structured surveys and a follow-up workshop. No statistical hypothesis testing was performed; descriptive insights guided iterative design. RESULTS Fifty caregivers completed the survey, highlighting neuropsychiatric symptoms, independence, and motor limitations as key priorities. Eight caregivers participated in the design workshop, emphasizing flexibility, age-appropriate tasks, and reduced reporting burden. Usability testing with five caregivers demonstrated high acceptance of digital tools and willingness to engage with app features. Participants valued customizable options, such as open-text fields and adaptable task lists, but noted challenges with video recording and repetitive questionnaires. Feedback underscored the need for simplified workflows and individualized approaches to maintain engagement. CONCLUSIONS Co-creation with caregivers is feasible and essential for developing meaningful digital outcome measures in Dravet syndrome. Video-based tasks and remote reporting tools show promise for capturing motor, cognitive, and behavioral domains beyond seizure frequency. Future work should focus on iterative refinement and formal validation of these measures as endpoints in clinical trials, ensuring they reflect outcomes that matter most to patients and families.