Orphan Drug Act: Congressional, FDA, NORD Reviews Come Amid Pricing Debate
Republican Senators request US Government Accountability Office quantify law’s impact and assess whether regulatory or legislative changes are needed. FDA plans ‘unbiased’ look at outcomes from law’s incentives, while NORD is developing data on total percentage of drug spending represented by orphan indications.
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Next Step After 'Right To Try'? Senate Hearing Includes Call To Reopen Orphan Drug Act
Trump Administration’s successful campaign to pass “Right to Try” legislation may be emboldening advocates to push for other policies that will loosen FDA regulatory requirements on rare disease drug development.
Orphan Designations For Pediatric Subgroups Of Common Diseases To End
US FDA's draft guidance closes loophole where sponsors could obtain an orphan designation for a pediatric subset and then be exempt from requirements to conduct pediatric studies.