Physicians’ Knowledge About FDA Approval Standards and Perceptions of the “Breakthrough Therapy” Designation

A survey of 675 board-certified internists and specialists found widespread misunderstanding of FDA approval standards. 73% incorrectly believed approval means a drug is as effective as others for the same condition, and only 6% correctly identified that FDA approval requires neither a statistically significant nor a clinically important result specifically. When presented with 2 identical hypothetical drugs differing only in whether one carried the “breakthrough” label, physicians significantly preferred prescribing the designated “breakthrough” drug, demonstrating that the term influences prescribing behavior independent of actual clinical evidence.