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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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.
Attorneys Sasinowski and Varond write that in some cases weak clinical evidence on the surrogate endpoint was not a barrier, but strong findings on unmet need and rarity usually are necessary for accelerated approval.