Strategy for Generating Blinded Evidence for Single-Arm Trials with External Controls Using Expert Review of Home Video

The study introduces a novel, blinded strategy for single-arm trials using expert panel reviews of home videos to assess developmental milestones in children with neurodegenerative diseases. By conducting interviews with parents/caregivers and clinical experts, the researchers identified meaningful milestones and standardized video recording procedures. A pilot study with 5 parents/caregivers and 3 grading experts demonstrated the feasibility and effectiveness of this approach, with participants finding the process clear and reflective of children's abilities. The strategy allows for objective, blinded grading of developmental achievements, offering a scalable and flexible method for data collection applicable to various disease contexts.