US FDA's Orphan Drug Designation Reviews Often Don't Include Regulatory Histories, GAO Says

Lack of information may mean FDA cannot conduct a thorough review, but HHS argues that products usually are very early in development and therefore don't have a regulatory history to document.

FDA entrance sign 2016

Staff of the US FDA often did not record background information as required when reviewing orphan designation requests, the Government Accountability Office found, which GAO suggested called into question the extent of the agency's evaluations.

The findings may not be too dire however, as HHS argued the background information GAO said was missing is actually rarely available when a sponsor requests an orphan

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