How the FDA thinks about those ‘N-of-1’ studies

Among the wonders of modern medicine are truly personalized treatments, invented exclusively to help a single patient with an otherwise untreatable genetic disease. Fitting those so-called N-of-1 therapies into a decades-old regulatory framework, however, presents some challenges for the likes of the FDA.

That hasn’t escaped the notice of Dr. Janet Woodcock, director of the agency’s Center for Drug Evaluation and Research. Because these single-patient therapies don’t need to be approved for sale, the standard FDA review process doesn’t apply. But if the future brings more N-of-1 success stories, the agency will need to figure out a replicable policy.

“We’ll have to develop policies and procedures if this becomes more common — which we suspect it will be,” Woodcock said. “We need to have a standardized approach to this that’s fair to everybody.”

Read more.