On May 16 and 17, 2022, the National Institutes of Health (NIH), National Center for Advancing Translational Sciences (NCATS) and the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER) will host a jointly sponsored virtual workshop to explore Regulatory Fitness in Rare Disease Clinical Trials.
Advances in rare diseases, particularly in the field of genetics, result in an active, highly innovative, and rapidly evolving area of science and drug development. Nonetheless, rare disease drug development represents an area of high unmet medical need. There are many challenges in rare disease drug development that are broadly recognized by the rare disease drug development community, such as:
Academic investigators, patient groups, and small or emerging pharmaceutical and biotechnology companies play a critical role in rare disease drug development and are often the sponsors for rare disease drug development, but they may lack regulatory experience. This workshop focuses on academic investigators and those looking to learn how to bridge the gap between academic investigation and the regulatory aspects of drug development.
This workshop will:
Participation in this workshop will allow attendees to improve their familiarity and understanding of Federal regulations, guidelines, and approaches for good quality rare disease clinical trials. While specific questions about applications will not be addressed, topics in general will be discussed and case studies will be presented as learning examples.
For more information about the event, please click here.