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

Join us as we provide an overview of the clinical development process for rare diseases. The EveryLife Foundation’s Community Congress will host a panel of experts to provide insight into the world of drug development. This webinar is free to attend but registration is required to obtain login information.  


  • 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, Unraveling 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

Throughout the year, the Foundation holds quarterly Community Congress meetings in addition to our in-person meeting, scheduled for November 16 in Washington D.C.  For more information on the EveryLife Foundation’s Community Congress, visit the program’s homepage