FDA Revises Guidance To Spur Efficient Rare Disease Drug Development

By David Roza / January 17, 2019 at 1:55 PM
Three years after publishing a draft guidance on rare disease drug development, FDA on Wednesday (Jan. 16) released a revised version of the guidance that agency commissioner Scott Gottlieb said will “provide additional advice to innovators on how they can pursue more efficient, effective development programs.” A leading rare disease patient advocacy organization praised FDA’s work on the guide, saying the agency’s clarification on the types of studies that can be used is crucial for the rare disease space. Gottlieb...


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