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

The Food and Drug Administration (FDA) is announcing the following 1-day 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.