Duke-Margolis Convenes Public Workshop to Advance Biomarker Use in FDA Regulatory Review

The Duke-Margolis Center for Health Policy (search)Search term is convening a public workshop aimed at developing a preliminary policy roadmap to strengthen the use of biomarkers and surrogate endpoints in regulatory decision-making for individual product submissions. The event brings together stakeholders from across the drug development ecosystem to address persistent uncertainties surrounding biomarker evidence, sponsor engagement with the U.S. Food and Drug Administration, and the transparency of regulatory evaluations.

Biomarkers and surrogate endpoints have become increasingly important tools in regulatory science, informing decisions that can improve both the efficiency and success of drug development. While these tools have successfully supported some product submissions through the FDA's accelerated approval pathway, stakeholders continue to report significant uncertainty about how fit-for-purpose evidence should mature over time, how sponsors should engage with the FDA during development, and how regulators evaluate and communicate biomarker-related decisions.

Workshop Focus Areas

The workshop sessions are structured around two primary contexts: biomarker-supported accelerated approvals and rare disease applications. Organizers note that while these areas serve as the focal point, the discussions are designed to yield lessons that may apply across broader therapeutic areas.

Key topics on the agenda include sponsors' real-world experiences in developing biomarker evidence, the challenges associated with analytical and clinical validation, and opportunities to strengthen both FDA engagement and review transparency. By examining these issues through the lens of actual product development experiences, the workshop aims to produce actionable insights that can inform regulatory policy.

Addressing Evidence Gaps

A central theme of the workshop is the question of evidence maturation. As biomarker-driven therapies increasingly target smaller, more precisely defined patient populations, developers face growing challenges in generating the robust clinical evidence traditionally expected by regulators and payers alike. The reliance on surrogate endpoints—while enabling faster development timelines—introduces additional complexity in demonstrating clinical benefit.

The Duke-Margolis initiative seeks to clarify how biomarker evidence can be developed and presented to meet regulatory standards while acknowledging the practical constraints of studying rare diseases and biomarker-defined subpopulations. The resulting policy roadmap is expected to provide guidance on navigating these evidence challenges while maintaining scientific rigor.

Stakeholder Engagement and Transparency

Improving the interface between drug sponsors and the FDA represents another critical dimension of the workshop. Participants will explore models for more effective communication during development, including how sponsors can obtain clearer regulatory feedback on biomarker strategies and how the FDA can enhance the transparency of its decision-making processes related to biomarker acceptance.

The workshop reflects growing recognition across the pharmaceutical industry that biomarker strategy must be integrated early and comprehensively into drug development programs—not only to satisfy regulatory requirements but also to build the evidence foundation necessary for downstream market access and reimbursement decisions.