Application of Model-Informed Approaches in Neuroscience Drug Development and Regulatory Decision-Making

Model-informed drug development (MIDD) approaches are being increasingly used in neuroscience drug development programs and regulatory decision-making. In this work, the authors summarize a select set of cases in neurology, psychiatry, addiction, anesthesia, and analgesia, where MIDD approaches were utilized to address critical drug development questions or fill knowledge gaps. These examples include efficacy considerations such as biomarker-clinical endpoint relationships, surrogate endpoints for accelerated approval, extrapolation of efficacy, and safety aspects such as informing driving studies. Additionally, the examples also inform optimal dosing regimen selection, dose adjustments in drug interactions, life cycle management decisions such as indication expansion, formulation switching, and informing patient population selection. Together, these examples offer insights into the utility of MIDD approaches to streamline drug development and opportunities to save costs and resources.