Description of Collaborative Activity: |
On May 16 and 17, 2022, the FDA Center for Drug Evaluation and Research (CDER) and NCATS hosted a jointly sponsored virtual workshop on regulatory fitness in rare disease clinical trials. Rare disease drug development represents an area of high unmet medical need. This workshop focused on academic investigators and those looking to learn how to bridge the gap between academic investigation and the regulatory aspects of drug development. The workshop provided lessons on the fundamentals of drug development and included discussion of rare disease case studies shared by academic investigators; shared experiences and expertise from multi-disciplinary stakeholders; discussed regulatory considerations for rare disease clinical trials; and discussed 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 CDER, and case studies. |
