Webinar: Bayesian Studies in Rare Diseases

E-mail Print

Original Webinar Date: January 22, 2020

The Rare Diseases Forum held its first webinar for the Bayesian Studies in Rare Diseases Working Group, under the Innovation in Trial Designs Workstream. 175 people registered for the session which was the launch event for the working group. Registrants represented patients, advocacy organizations, academia, federal agencies, industry, professional societies, and other relevant entities. This workstream was initiated to address a challenge by Dr. Janet Woodcock at the Rare Diseases Forum 1 meeting to transform the clinical trial paradigm for rare diseases through application of novel approaches.

 The project seeks to:

  • Enhance data quality and analysis
  • Advance innovation in clinical trial design
  • Improve and expand research and regulatory data sources
  • Encourage discussion on specific master protocols
  • Explore application of Bayesian statistics in rare diseases

Presentations

pdf Application of Bayesian Studies in Rare Disease Settings
       Veronica Miller, The Forum for Collaborative Research

pdf Bayesian Methods Overview
       Karen Lynn Price, Eli Lilly & Company

pdf snSMARTDesign in Rare Disease Research: Motivated by ARAMIS
       Kelley M Kidwell, University of Michigan

pdf Practical Bayesian Design for Rare Disease Drug Development
       Satrajit Roychoudhury, Pfizer Inc