Advancing the Regulatory Framework to Increase Biomarker Utilization in Application Review

Abstract biological signals pass through four translucent layers, becoming increasingly structured before resolving into an organized evidence dossier and decision point.

Overview

The Duke-Margolis Institute for Health Policy is leading a policy research project on improving how biomarkers and surrogate endpoints support FDA decision-making in individual product submissions. This research focuses especially on biomarker-supported accelerated approval and rare disease contexts; it does not focus on FDA’s formal Biomarker Qualification Program.

Aims

This policy research project aims to develop a practical policy roadmap for improving how biomarker and surrogate endpoint evidence supports individual product development and FDA decision-making. The roadmap will identify policy actions to:

Better calibrate evidence expectations to intended use and uncertainty
Enable responsible transfer and comparison of evidence across contexts
Support earlier and more decision-focused FDA engagement
Improve learning from prior regulatory decisions
Strengthen reusable evidence infrastructure
Account explicitly for the feasibility constraints of rare-disease development and patient participation

Event - 7/15/2026

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

Washington, DC

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.