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US FDA Open To Innovative Statistical Approaches For Rare Disease

 
• By Sarah Karlin-Smith

Move over big data, a biostatistician calls for more people to think small for rare diseases and FDA officials indicate openness to the trial approaches laid out.

woodenblocks that say "think" and a block that has big on one face and small on the other face
While big data is all the rage, more statisticians need to be working on being efficient with small data, FDA-Duke meeting speaker says • Source: Shutterstock

The US Food and Drug Administration is open to “innovative” statistical approaches to address challenges in rare disease drug development, FDA supervisory mathematical statistician Rebecca Chiu said at a recent meeting.

Her remarks in particular referenced a presentation from Kelley Kidwell, of the University of Michigan School of Public Health at a December FDA-Duke Margolis Center for Health Policy meeting on advancing the development of therapeutics through rare disease patient

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