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) are hosting a jointly sponsored virtual workshop to explore Regulatory Fitness in Rare Disease Clinical Trials.

The virtual workshop will take place over two days on May 16th – 17th.

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:

  • Provide lessons on the fundamentals of drug development and discuss rare disease case studies shared by academic investigators
  • Share experiences and expertise from multi-disciplinary stakeholders
  • Discuss regulatory considerations for rare disease clinical trials
  • Explore topics such as:
    • Adequate and well-controlled trials
    • Core principles for clinical trials
    • The nuts and bolts of Investigational New Drug (IND) applications and additional considerations
    • Additional pathways to interact with the FDA
    • Case studies

Registration is mandatory to attend the workshop.

More information and registration here: https://events-support.com/events/CDER_NCATS_Regulatory_Rare_Disease_Workshop