FDA's Peter Marks lays out plans to support gene therapies for ultra-rare diseases
It’s rare to hear an FDA leader say that the use of randomized controlled trials “borders on the absurd.”
But that’s what CBER director Peter Marks told listeners at a Duke-Margolis event last week, with his presentation on developing gene therapies for ultra-rare conditions where there might be just 20 patients worldwide, which is why an RCT may not be a good fit.
So how then could a biotech or pharma company address such small populations?
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