Abstract
Abstract
The US Food and Drug Administration (FDA) has historically exercised substantial regulatory flexibility to adjust the type, amount, and quality of evidence necessary to support drug approval for areas of unmet need. Our objective was to characterize how FDA officials described and justified regulatory flexibility in recent public statements. We conducted a thematic analysis of quotations related to regulatory flexibility from 31 FDA officials published in Pink Sheet and STAT, two leading news sources covering FDA, from January 2021 to June 2024. In these statements, FDA officials typically justified prioritizing patient access to promising treatments over certainty regarding efficacy at the time of approval, even outside the accelerated approval pathway. Regulators also highlighted confirmatory studies conducted after accelerated approval as important for establishing drug efficacy. However, they recognized challenges to their completion, including insufficient industry incentives and FDA enforcement, and were reticent to interpret failed studies as evidence of failed drugs. Overall, in public statements from 2021–2024, FDA officials acknowledged challenges posed by regulatory flexibility but publicly justified its broad use to improve patient access for serious diseases despite even extended uncertainty. Under the Trump Administration, many officials have changed but efforts to advance regulatory flexibility continue.