FDA CDER & NIH NCATS Regulatory Fitness in Rare Disease Clinical Trials Workshop
Are you an academic investigator looking to learn how to bridge the gap between academic investigation and the regulatory aspects of rare disease drug development?
On May 16-17, 2022, the FDA’s Center for Drug Evaluation and Research (CDER) and NIH’s National Center for Advancing Translational Sciences (NCATS) 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. The FDA recognizes the serious nature of rare diseases, the impact rare diseases have on patients and their families, the unmet medical need for therapies, and the urgency to make safe and effective treatments available.
The workshop will be an opportunity for attendees to improve their familiarity and understanding of federal regulations, guidelines, and approaches for good quality rare disease clinical trials. This workshop will:
- Provide lessons on the fundamentals of drug development and discuss rare disease case studies
- Share experiences and expertise from multi-disciplinary stakeholders
- Discuss regulatory considerations for 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.
The workshop is free and open to the public. Please visit FDA CDER & NIH NCATS Regulatory Fitness in Rare Disease Clinical Trials Workshop for more information, the draft agenda, and to register for the upcoming workshop.
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