Obtaining accurate outcome assessment data is a challenge in children with heterogeneous neurodegenerative disorders and may significantly vary with age, causing great challenges when setting clinical trial endpoints. Qualitative data in the form of patient and caregiver input is increasingly used in the process of therapy development1 and is essential to understand how well existing tools are measuring efficacy outcomes important to patients and families that are associated with neurodegenerative disorders
• FDA has provided a series of guidance documents intended to facilitate the collection of robust, meaningful input2
• In this international study, qualitative data were gathered in order to better understand functional challenges faced by children impacted by CLN3 Batten disease and compared items included in two commonly used clinical rating instruments, the UBDRS3 and Vineland III4 to evaluate their validity