Utilizing Innovative Statistical Methods and Trial Designs in Rare Disease Settings; Public Workshop


March 19, 2018


9 a.m. - 5 p.m.


DoubleTree by Hilton, 8728 Colesville Rd, Silver Spring, MD 20901


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 a number of significant challenges for clinical research, drug development, and regulatory review. Small population sizes, sometimes limited scientific understanding of the disease of interest, and misaligned policy or 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 that could form the basis of formal guidance documents.

During the course of the public workshop, speakers and participants will discuss a range of tools and methods that could 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: the use of information from sources external to a rare disease trial to better inform trial design and analysis; master protocols that incorporate innovative adaptive designs; analytical tools for trials with multiple or novel endpoints; and Bayesian borrowing in confirmatory clinical trials.


To register for this meeting, visit: https://healthpolicy.duke.edu/events/utilizing-innovative-statistical-methods-and-trial-designs-rare-disease-drug-development

Persons interested in attending this public workshop must register by 5 p.m. EDT on March 15, 2018

Federal Register Notice:

The Federal Register Notice for this meeting may be found here.

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