Biomarker Utilization for Regulatory Decision Making: A Landscape Analysis of Neurological Drug Products Approved by...

Over the past 15 years, biomarkers have evolved into a significant component of neurological drug development and regulatory evaluation, with expanded roles and increasing impact. This landscape analysis covers new molecular entity (NME) products approved by Food and Drug Administration from 2008 to 2024 for neurological diseases, which demonstrates the growing utilization of biomarkers in regulatory decision making. This includes their use as surrogate endpoints, confirmatory evidence, and basis for dose selection. Our analysis suggested prominent roles of biomarkers in various therapeutic modalities, including small molecules, oligonucleotides, and monoclonal antibodies, as well as in the context of rare or slowly progressive diseases. However, several challenges hinder the effective utilization of biomarker data, including lack of established clinical relevance, variability in data quality, and bioanalytical issues. Addressing these challenges will require cross-sector collaboration, rigorous analytical validation, and a clear demonstration of the linkage between biomarker changes and meaningful clinical benefits. By strengthening the evidentiary foundation of biomarker data, stakeholders can accelerate innovative drug development for neurological diseases and other therapeutic areas.