FDA Leadership Changes Prompt Concerns Among Rare Disease Drug Developers

Drug industry executives and an academic expert said Wednesday that recent departures of key officials at the Food and Drug Administration have created uncertainty for companies developing treatments for rare diseases. They also discussed how much flexibility the agency should allow when companies design clinical trials for these medicines.

Weiss said she receives frequent questions from investors about how the personnel changes will affect her company. "There isn’t a day where I don’t find an email in my inbox where my investors are questioning the recent news they read, and how it will affect our future as a company, our certainty of getting our drugs approved," Weiss said.

"It’s a roller coaster," she added. The discussion took place at the STAT Breakthrough Summit West in San Francisco.