Turmoil at FDA puts rare disease review capacity at risk

A relentless six-week assault on the civil service, with more upheaval planned, has set off a “five-alarm fire for rare disease,” an FDA reviewer told BioCentury this weekend. The reviewer warned that an exodus of experienced staff that leads to lower quality, slower product reviews, rather than increased efficiency, is a “likely” outcome of orders from the White House and presidential adviser Elon Musk encouraging staff to quit, combined with an imminent end to remote work.

If the fire isn’t extinguished quickly, whatever remains of the agency’s orphan drug review teams will be ineffective, unqualified and politically compromised, said the reviewer, who does not want to be identified because they are not authorized to speak with the media. “What’s left won’t be what anyone hoped for, and time is not on our patients’ side.”...