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Sustainability of Rare Disease Drug Development
Session Chair(s)
Darcy Frear, PHD
Associate Director, BridgeBio, United States
Panelists will reflect on the past forty years of the orphan drug act including advancements and impediments. The panel will discuss what is needed in the future to continue to advance rare disease drug development to treat as many patients as possible.
Learning Objective : Discuss the outcomes, challenges, and opportunities in the next forty years of the Orphan Drug Act; Identify policy advancements that need to be made to continue innovative rare disease drug development.
Speaker(s)
Katherine Donigan, PHD
Executive Director, Science and Regulatory Policy, Sarepta, United States
Panelist
Patroula Smpokou, MD
Division Director , FDA, United States
Panelist
Karin Hoelzer, DVM, PHD
Senior Director, Policy and Regulatory Affairs, NORD, United States
Panelist
Julia Tierney, JD
Principal, Leavitt Partners, United States
Panelist
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