title: Clinical Evidence Supporting US Food and Drug Administration Premarket Approval of High-Risk Otolaryngologic Devices, 2000-2014.
abstract: 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.
authors.affiliation: 1 Department of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, USA.|2 Department of Otology and Laryngology, Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Vinay K
authors.initials: VK
authors.lastname: Rathi
authors.affiliation: 3 Department of Medicine, Stanford University School of Medicine, Stanford, California, USA.
authors.forename: Bo
authors.initials: B
authors.lastname: Wang
authors.affiliation: 4 Center for Outcomes Research and Evaluation, Yale-New Haven Hospital, New Haven, Connecticut, USA.|5 Section of Health Policy and Administration, Department of Epidemiology and Public Health, Yale University School of Medicine, New Haven, Connecticut, USA.|6 Section of General Internal Medicine, Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut, USA.
authors.forename: Joseph S
authors.initials: JS
authors.lastname: Ross
authors.affiliation: 7 Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
authors.forename: Nicholas S
authors.initials: NS
authors.lastname: Downing
authors.affiliation: 8 Program on Regulation, Therapeutics, and Law, Division of Pharmacoepi- demiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
authors.forename: Aaron S
authors.initials: AS
authors.lastname: Kesselheim
authors.affiliation: 1 Department of Otolaryngology-Head and Neck Surgery, Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, USA.|2 Department of Otology and Laryngology, Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Stacey T
authors.initials: ST
authors.lastname: Gray
mesh_terms: D002986:Clinical Trials as Topic; D003430:Cross-Sectional Studies; D018795:Device Approval; D004869:Equipment Safety; D006801:Humans; D010036:Otolaryngology / Q000295:instrumentation*; D018570:Risk Assessment; D014481:United States; D014486:United States Food and Drug Administration
keywords: FDA; medical device; otolaryngology; premarket approval