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Pediatric Drug Development Workshop


Session 3: Leveraging Innovative Trials Design I: Product Development-Centric Registries and Platforms

Session Chair(s)

Edward  Connor, MD, MS

Edward Connor, MD, MS

  • President, Clinical Research Alliance; Chairman and President
  • I-ACT for Children, United States
Andrew E. Mulberg, MD

Andrew E. Mulberg, MD

  • Senior Vice President, Global Regulatory Affairs
  • Amicus Therapeutics , United States
This session will explore the innovative use of trial designs and registry development to address clinical issues including understanding natural history and outcome assessment for effective endpoint development in pediatric drug development. A presentation of scientific underpinnings of effective and existing registry development for the treatment of pediatric diseases will be presented.

Speaker(s)

Laura E Schanberg, MD

Speaker

Laura E Schanberg, MD

  • Associate Professor and Co-chief, Pediatric Rheumatology
  • Duke University Medical Center, United States
John-Michael  Sauer, PhD

Speaker

John-Michael Sauer, PhD

  • Biomarkers Program Officer and Executive Director
  • Critical Path Institute, United States
Abby  Bronson, MBA

Speaker

Abby Bronson, MBA

  • Vice President, Patient Advocacy and External Innovation
  • Edgewise Therapeutics, United States