Orphan Drug Development: Could We Be Seeing The End Of P Values?
Executive Summary
Panel of experts stresses need to move toward innovative approaches for rare disease drug development such as Bayesian methods, but this would require a paradigm shift in US FDA's regulatory structure.
You may also be interested in...
Woodcock Hopes I-SPY 2 Trial Is 'Blazing The Trail' For Future Adaptive Design Uptake
CDER director renews US FDA's support for complex trials, but acknowledges that sponsors are still hesitant to adopt them.
Orphan Drug Policy Changes To Be Considered Under US FDA's FY 2019 Budget Proposal
Proposed $20m funding boost for rare disease development includes review of orphan incentives, including how to potentially stimulate development in ultra-orphan diseases.
Moscicki's Move From FDA To PhRMA About 'Best' Use Of Leadership Skills
At inconveniently timed NORD conference, the former CDER deputy center director finds himself unable to speak for either FDA or the Pharmaceutical Research and Manufacturers of America.