The Food and Drug Administration (FDA) is announcing a public workshop entitled “Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings.” This workshop is convened by the Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University and supported by a cooperative agreement with FDA. The purpose of the public workshop is to bring rare disease stakeholders together to discuss the challenges associated with the development and regulatory decision-making for rare disease treatments and to also discuss promising study designs and analytical methods that can help overcome these challenges.
Rare disease settings pose several significant challenges for clinical research, drug development, and regulatory review. Small population sizes, possible limited scientific understanding of the disease of interest, and a lack of market incentives often preclude more traditional clinical trial or analytical approaches from being pursued. To help collaboratively address these barriers, FDA is working with stakeholders to solicit feedback on promising designs and methodologies for use in the development of rare disease treatments that can form the basis of formal guidance documents.
During the public workshop, speakers and participants will discuss a range of tools and methods that can be used in the development of treatments for rare diseases and small patient populations. The meeting will include both presentations by panelists and dedicated time for questions and comments from attendees. Topics will include: Master protocols, use of external controls in single-arm trials, analytical tools for trials with multiple or novel endpoints, and best practices for leveraging Bayesian statistics and adaptive study designs.
Members of the public may attend in person or via webcast. More information is available via the Federal Register notice.
SciPol Editors' Note: This event is co-sponsored by Duke University's Duke-Robert J. Margolis, MD, Center for Health Policy. The Margolis Center is not directly affiliated with SciPol.