As technology becomes grows increasingly sophisticated, scientists are able to expand their targets to diseases and disorders that were formerly untreatable. With the opportunity to develop treatments for diseases with unmet medical needs comes the challenges of designing clinical trials. This panel of researchers, clinicians and members of the advocacy community will describe their experiences designing clinical trials in an area with no historical experience, share lessons learned and their recommendations for moving forward.

• Describe design and implementation of clinical trials for diseases with unmet medical need
• Increase understanding of challenges inherent in designing "first in category" treatments
• Gain knowledge about best practices and recommendations for future activities