US FDA Open To Innovative Statistical Approaches For Rare Disease

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...