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Unvalidated PROs In Rare Diseases: US FDA May Have To ‘Work Through’ Them, CBER’s Marks Says

 
• By Sue Sutter

Given the challenges of validating patient-reported outcome instruments specific to very rare diseases, agency is probably going to have to accept the use of unvalidated measures in some cases, Peter Marks tells American Society of Gene and Cell Therapy’s annual meeting.

Check marks
FDA wants sponsors to use validated PROs but may have to accept unvalidated measures in the case of very rare diseases. • Source: Shutterstock

The US Food and Drug Administration may need to consider accepting unvalidated patient-reported outcome measures in the context of very rare diseases, Center for Biologics Evaluation and Research Director Peter Marks said.

Speaking during a wide-ranging discussion on emerging regulatory trends at the American Society of Gene and Cell Therapy’s annual meeting on 18 May, Marks acknowledged the challenges that rare disease drug sponsors face in trying to demonstrate improvement in outcomes that matter to patients when the number of individuals

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