Innovative Design and Statistical Methods for Issues in Neuroscience Clinical Trials

Drug development in neuroscience is marked by profound complexity, substantial costs, and significant uncertainty, due to the intricate challenges posed by neurodegenerative and neuropsychiatric disorders. Overcoming these barriers demands innovative statistical methodologies and collaborative expertise throughout the industry. Supported by the ASA Biopharmaceutical Section's Neuroscience Scientific Working Group, this session brings together statisticians from leading pharmaceutical organizations to share advances and practical solutions in neuroscience clinical trials. Speakers will highlight real-world implementations of adaptive endpoints and analysis strategies using creative study designs; discuss the use of digital therapeutics to address specific challenges in a pivotal Phase 3 schizophrenia study; demonstrate the benefits of Bayesian borrowing paired with propensity score methods for pediatric major depressive disorder; as well as present a novel simulation framework for generating synthetic progression data to model pre-symptomatic frontotemporal dementia. The session will close with a discussant who will integrate the presented ideas and stimulate dialogue, aiming to identify persistent challenges and emerging opportunities in neuroscience drug development, and to spark further innovation in statistical methods to improve clinical trial success rates.