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Biomarkers For Rare Diseases Get Scaled-Back Plea In New White Paper

 
• By Derrick Gingery

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.

A biomarker qualification meeting for a rare disease drug attempting to use accelerated approval need not be held in the pre-IND stage, a rare disease group decided, but early enough to deliver some certainty to the development process.

Conducting disease surveys early in product development also could better inform FDA about the flexibility needed to push the potential new treatment through the pathway, the EveryLife Foundation for Rare Diseases said in the final version of a

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