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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