FDA Regulatory Innovation: Plausible Mechanism

For many rare diseases, especially those with strong biological rationale but limited clinical data, regulatory approaches may rely on alternative forms of evidence. This session will provide context on how concepts such as plausible mechanism and platform-based development are considered within the regulatory framework for therapies targeting small populations.

Moderator: Julia Vitarello, Founder & CEO, Mila’s Miracle Foundation 

Panelists:

• Teresa Buracchio, MD, Director, Office of Neuroscience, CDER, FDA
• Michelle Mellion, MD, Chief Medical Officer, Clinical Development and Strategy Advisor; former CMO, EveryONE Medicines
• Lowell Schiller, JD, Nonresident Senior Scholar, USC Schaeffer Center, a NORD Rare Disease Center of Excellence; former Principal Associate Commissioner for Policy, FDA
• Judy Stecker, Founder, Wheeler’s Warriors; Patient Advocate; Former Deputy Chief of Staff, HHS
• Timothy Yu, MD, PhD, Associate Professor of Pediatrics, Harvard Medical School, Boston Children’s Hospital, a NORD Rare Disease Center of Excellence

View Slideshow Presentation from this Session