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Disease 'Subsetting' Not Driving Jump In Orphan Designations, US FDA Says

 
• By Derrick Gingery

Preliminary study results suggest policy tweaks may not be necessary, even as an oncology orphan product rush appears to be coming.

Targeted therapies do not appear to be driving the growing popularity of orphan drug designations, according to preliminary data from an FDA study.

"We're still in the final analysis, but it doesn't really look like the subsetting is really what is driving the numbers going up," Gayatri Rao, director of the US FDA’s Office of Orphan Products Development, told the National Organization for

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