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:
- Data quality and data consistency
- Fit for purpose research and regulatory data sources
- Discussion on specific master protocols for rare diseases
- Application of Bayesians statistics in rare diseases.
Meeting Report (coming soon)
Presentations
Veronica Miller, Forum for Collaborative Research
Session II: Lessons from Oncology Master Protocols
Cancer Drug Development & Access to Precision Medicine for Children with Cancer
Gregory Reaman, CDER, FDA
Lessons from Oncology Master Protocols: Panel 1 - BeatAML
Nita Seibel, NCI,NIH
Beat AML Master Trial
Amy Burd, Leukemia Lymphoma Society
The LLS PedALTrial
Gwen Nichols, Leukemia Lymphoma Society
Some Industry Perspectives
Kathleen Neville, Johnson and Johnson
Gregory Reaman, CDER, FDA
Lessons from Oncology Master Protocols: Panel 1 - BeatAML
Nita Seibel, NCI,NIH
Beat AML Master Trial
Amy Burd, Leukemia Lymphoma Society
The LLS PedALTrial
Gwen Nichols, Leukemia Lymphoma Society
Some Industry Perspectives
Kathleen Neville, Johnson and Johnson
P.J. Brooks, NCATS, NIH
Bayesian Analysis in Rare Diseases
Kelley Kidwell, University of Michigan
Kelley Kidwell, University of Michigan
Session III: Specific Topics Panels
Mary Lin, CBER, FDA