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:

  • The large number of different rare diseases (more than 7,000)
  • The small number of patients with the individual disorders which can make clinical trial design and interpretation challenging
  • The lack of drug development precedent for most rare diseases
  • The lack of established endpoints, including biomarkers and clinical outcome measures, for some rare disease clinical trials

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

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.

Food and Drug Administration NIH