Rare Diseases Workshop 1

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The Rare Diseases Forum Workshop 1 "Innovation in Trial Designs: Bringing Lessons from the Oncology Experience to Rare Diseases" was held on Monday, September 23, 2019 at the National Press Club, in Washington, DC. 106 people attended the event. Of those, 80 attended in person, and 26 registered for the webcast. This workshop was the launching event for the "Innovation in Trial Designs" work stream. The event convened a stellar group of experts in oncology, rare diseases and statistics.

Discussions to be undertaken by the Rare Diseases Forum as outcomes of this one-day workshop included:

  1. Data quality and data consistency
  2. Fit for purpose research and regulatory data sources
  3. Discussion on specific master protocols for rare diseases
  4. Application of Bayesians statistics in rare diseases.
pdf Agenda

       Meeting Report (coming soon)

pdf Participants

 
Presentations
 
Session I: Introduction & Project Overview
 
pdf The Rare Diseases Forum - Innovation in Trial Design Workshop
       Veronica Miller, Forum for Collaborative Research
 
Session II: Lessons from Oncology Master Protocols
 
pdf Cancer Drug Development & Access to Precision Medicine for Children with Cancer
       Gregory Reaman, CDER, FDA

pdf Lessons from Oncology Master Protocols: Panel 1 - BeatAML
       Nita Seibel, NCI,NIH

pdf Beat AML Master Trial
       Amy Burd, Leukemia Lymphoma Society

pdf The LLS PedALTrial
       Gwen Nichols, Leukemia Lymphoma Society

pdf Some Industry Perspectives
       Kathleen Neville, Johnson and Johnson
 
pdf Thousands of Rare Diseases, Far Fewer Etiologies
       P.J. Brooks, NCATS, NIH
 
pdf Bayesian Analysis in Rare Diseases
       Kelley Kidwell, University of Michigan
 
Session III: Specific Topics Panels
 
pdf Data Quality in Outcome Assessments
       Mary Lin, CBER, FDA