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Push To Create Ultra-Rare Orphan Drug Designation Finds Resistance At US FDA

 
• By Derrick Gingery

Agency officials don’t think a new definition or incentives will fix the issues related to development in rare diseases with only a handful of patients, as advocates continue pushing the idea.

roadblock
Without FDA support, passing legislation to create ultra-orphan incentives will become more difficult. • Source: Shutterstock

Efforts to create an ultra-rare disease designation and related incentives seem to have hit a roadblock at the US Food and Drug Administration.

Two prominent FDA officials, who oversee many of its rare disease programs, are not interested in creating a new designation within the Orphan Drug Act

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