Biomarkers For Rare Diseases Get Scaled-Back Plea In New White Paper
EveryLife Foundation’s finalized white paper on using accelerated approval in rare diseases indicates that biomarkers don’t have to be discussed with FDA at the pre-IND stage, but should be done early enough to allow development process predictability.
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If they reach a public disclosure agreement, a handful of sponsors will be admitted to a new pilot program that allows four meetings with the FDA on rare disease endpoint issues.
Success of enhanced communications in particular made one stakeholder wonder whether FDA could use it with non-breakthrough applications.
CDER’s Woodcock says accelerated approval requirements in 21st Century Cures legislation could create a process akin to how FDA handles breakthrough designations.