Wed, Mar 29, 2017
1:00 PM - 2:00 PM

3/29/17 Community Congress Webinar


Regulatory Working Group Webinar

Clinical Development for Rare Diseases: A Primer for Rare Disease Patients and Advocates

Wednesday, March 29th

1:00 pm ET/10:00 am PT



  • Introduction to Community Congress and Regulatory Science Working Group
    • Max Bronstein, Chief Science Policy and Advocacy Officer, EveryLife Foundation for Rare Diseases
    • Lynne McGrath, Vice-President of Regulatory Affairs, RegenXBio
    • Isabelle Lousada, CEO and President, Amyloidosis Research Consortium
  • Importance of Natural History Studies and Patient Registries
  • Overview of Rare Disease Clinical Development Process - Phases and Timelines of Drug Development, Unravelling Industry Jargon, Importance of Endpoints
  • Special Approval Pathways and Incentives - Accelerated Approval, Fast Track, Priority Review, Breakthrough, Orphan Drug Act Incentives
  • Early Access and Right to Try
    • David Farber, Partner, King & Spalding

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