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
Application of Bayesian Studies in Rare Disease Settings
Veronica Miller, The Forum for Collaborative Research
Bayesian Methods Overview
Karen Lynn Price, Eli Lilly & Company
snSMARTDesign in Rare Disease Research: Motivated by ARAMIS
Kelley M Kidwell, University of Michigan
Practical Bayesian Design for Rare Disease Drug Development
Satrajit Roychoudhury, Pfizer Inc