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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)
Panelist
Katherine Donigan, PhD
Sarepta, United States
Senior Director, Science and Regulatory Policy
Panelist
Patroula Smpokou, MD
FDA, United States
Lead Physician, Division of Rare Diseases/Medical Genetics, OND, CDER
Panelist
Karin Hoelzer, DVM, PhD
NORD, United States
Senior Director, Policy and Regulatory Affairs
Panelist
Julia Tierney, JD
FDA, United States
Deputy Center Director for Strategy, Policy, and Legislation, CBER
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