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Orphan Drug Act: Congressional, FDA, NORD Reviews Come Amid Pricing Debate

 
• By Sue Sutter

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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Congress and the US FDA are carrying out separate but seemingly parallel evaluations of whether the Orphan Drug Act is working to spur rare disease research and whether its incentives have been abused.

Three Republican senators have requested the Government Accountability Office (GAO) quantify the impact that the law – which provides tax credits, marketing exclusivity and other incentives – has had on drug development and opine as to

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