The Regulatory Landscape: Pathways, End Points, and Clinical Trials – 2023 RARE Drug Development Symposium

May 10, 2023

In May 2023, Global Genes and the Orphan Disease Center of the University of Pennsylvania hosted the RARE Drug Development Symposium. In this session, you’ll learn about the different pathways open for rare disease therapeutics, why the involvement of patients is essential to establishing meaningful outcome measures in these pathways, and what patient advocates can do to influence clinical trial design.

Key Takeaways:

  • Regulatory is not as scary as you think!
  • Focus on early, frequent and regular communication and engagement, getting feedback on proof of concept, design, and strategy as you progress.
  • Take advantage of the many different pathways that FDA provides to include the voice of the patient.

Katherine Maynard, Communications and Alliance Development, PWR

Lea Anne Browning-McNee, MS
Director of Communication and Stakeholder Engagement, Reagan-Udall Foundation

Neena Nizar
Founder and President, The Jansen’s Foundation

Elizabeth Ottinger, PhD
Acting Director, Therapeutic Development Branch, National Center for Advancing Translational Sciences

Issac Rodriguez-Chavez, PhD, MHS, MS

Dan Lavery
Chief Scientific Officer, Loulou Foundation


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