Clinical Evidence Supporting US Food and Drug Administration Premarket Approval of High-Risk Otolaryngologic Devices, 2000-2014

Otolaryngol Head Neck Surg. 2017 Feb;156(2):285-288. doi: 10.1177/0194599816684094. Epub 2017 Jan 17.


The US Food and Drug Administration (FDA) approves high-risk medical devices based on premarket pivotal clinical studies demonstrating reasonable assurance of safety and effectiveness and may require postapproval studies (PAS) to further inform benefit-risk assessment. We conducted a cross-sectional analysis using publicly available FDA documents to characterize industry-sponsored pivotal studies and PAS of high-risk devices used in the treatment of otolaryngologic diseases. Between 2000 and 2014, the FDA approved 23 high-risk otolaryngologic devices based on 28 pivotal studies. Median enrollment was 118 patients (interquartile range, 67-181), and median duration of longest primary effectiveness end point follow-up was 26 weeks (interquartile range, 16-96). Fewer than half were randomized (n = 13, 46%), blinded (n = 12, 43%), or controlled (n = 10, 36%). The FDA required 23 PASs for 16 devices (70%): almost two-thirds (n = 15, 65%) monitored long-term performance, and roughly one-third (n = 8, 35%) focused on subgroups. Otolaryngologists should be aware of limitations in the strength of premarket evidence when considering the use of newly approved devices.

Keywords: FDA; medical device; otolaryngology; premarket approval.

MeSH terms

  • Clinical Trials as Topic
  • Cross-Sectional Studies
  • Device Approval*
  • Equipment Safety
  • Humans
  • Otolaryngology / instrumentation*
  • Risk Assessment
  • United States
  • United States Food and Drug Administration*