Advancing Biomarker Use in Regulatory Review: Evidence, Engagement, and Ecosystem Opportunities

Biomarkers and surrogate endpoints inform regulatory decision-making and can improve the efficiency and success of drug development. Although biomarkers have successfully supported some product submissions through the U.S. Food and Drug Administration’s accelerated approval pathway, stakeholders continue to report uncertainty about how fit-for-purpose evidence should mature, how sponsors should engage FDA, and how regulators evaluate and communicate biomarker-related decisions. 

Duke-Margolis is convening this public workshop to inform a preliminary policy roadmap to improve the use of biomarkers and surrogate endpoints in regulatory decision-making for individual product submissions. The workshop sessions focus on biomarker-supported accelerated approvals and rare disease contexts, while considering lessons that may apply to broader therapeutic areas. Discussions will highlight sponsors’ real-world experiences developing biomarker evidence, challenges related to analytical and clinical validation, and opportunities to strengthen FDA engagement and review transparency.