Orphan Pediatric Loophole To Be Closed By US FDA
Commissioner Gottlieb hopes accomplish through guidance a policy change that advocates failed to get included in user fee legislation passed earlier this year.
You may also be interested in...
US FDA's draft guidance closes loophole where sponsors could obtain an orphan designation for a pediatric subset and then be exempt from requirements to conduct pediatric studies.
QuintilesIMS Institute study funded by the National Organization for Rare Disorders concludes orphan drugs accounted for only 7.9% of US total drug spending in 2016, bolstering advocacy group's view that rare disease treatments are not responsible for rising healthcare costs. US FDA analysis finds most orphan drugs do not expand their labeled indications into non-rare disease settings.
User fee legislation headed for a US House vote this week would require pediatric studies of certain molecularly targeted cancer drugs; the requirement, which could apply to some orphan drugs, would not take effect for three years.