FDA’s Peter Stein On Hitting The ‘Persuasive Evidence’ Bar For Rare Disease Drug Repurposing

The US Food and Drug Administration’s evidence expectations for sponsors looking to repurpose an available therapy for a rare disease may be easier to hit than many assume, increasing the importance of early conversations with agency officials on moving the product forward, particularly if there are concerns about the financial case for the indication, agency leadership said.

The sentiment from Peter Stein, director of the Office of New Drugs in the Center for Drug Evaluation and Research, emerged after he was asked about repurposing challenges, in particular that the financials don’t always add up