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.
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Commissioner Gottlieb tells Congress that concept could be used for some clinical endpoints when a large benefit is seen in a small trial, which could benefit rare disease drug development.
Frank Sasinowski updates his 2012 landmark analysis and finds that FDA maintained its rate of flexibility in the evidentiary standards for approval of orphan products.
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.