PDUFA V: Accelerated Approval Expansion May Outshine Rare Disease Improvements

Rare disease advocate says the focus should be on ensuring the accelerated approval expansion and surrogate endpoint guidances become FDA priorities as the rare disease drug program is set to grow under PDUFA V.

Congress mandated specific upgrades to FDA’s rare disease program in the new user fee legislation, but the largest impact may come through the expected changes to the accelerated approval pathway.

Rare disease-related improvements and mandates were sprinkled throughout the prescription drug user fee reauthorization and agency policy changes in the

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