title: Postmarketing trials and pediatric device approvals.
abstract: BACKGROUND
  Medical devices can be useful in a variety of diseases, but few devices have been specifically approved for use in children. The 2007 Pediatric Medical Device Safety and Improvement Act was passed to stimulate pediatric device development. The current state of trial evidence underpinning the approval of pediatric devices remains poorly described.
  
  
  METHODS
  We identified all high-risk (ie, class III) devices approved through the premarket approval or humanitarian device exemption pathways for therapeutic use in children between 2008 and 2011. We collected key information on clinical trial design (randomization, blinding, controls, and types of end points) as well as age distribution of trial participants. We also identified US Food and Drug Administration (FDA)-mandated postmarketing trials.
  
  
  RESULTS
  Twenty-two devices were approved for use in children via the premarket approval pathway and 3 via the humanitarian device exemption pathway. Twenty-two (88%) qualified as pediatric despite minimum approval ages of ≥18 years (the FDA Center for Devices and Radiologic Health considers patients 18-21 years old as pediatric). Most devices were approved on the basis of nonrandomized (59%), open-label (68%) studies with surrogate effectiveness end points (86%). Overall, 21 (84%) devices were not studied in any patients <18 years of age. Postmarketing studies were mandated by the FDA for 19 (76%) devices, although only 3 (18%) required enrollment of pediatric patients.
  
  
  CONCLUSIONS
  Most high-risk pediatric devices are approved on the basis of trials in patients ≥18 years old, with few pediatric patients exposed to the devices before market availability. Few postmarketing studies require additional study in pediatric patients.
authors.affiliation: Faculty of Arts and Sciences, Harvard University, Cambridge, Massachusetts; Program on Regulation, Therapeutics, and Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts;
authors.forename: Thomas J
authors.initials: TJ
authors.lastname: Hwang
authors.affiliation: Program on Regulation, Therapeutics, and Law, Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts;
authors.forename: Aaron S
authors.initials: AS
authors.lastname: Kesselheim
authors.affiliation: Division of Emergency Medicine, Boston Children's Hospital, Boston, Massachusetts; and Department of Pediatrics, Harvard Medical School, Boston, Massachusetts florence.bourgeois@childrens.harvard.edu.
authors.forename: Florence T
authors.initials: FT
authors.lastname: Bourgeois
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D015331:Cohort Studies; D057176:Compassionate Use Trials / Q000331:legislation & jurisprudence; D018795:Device Approval / Q000331:legislation & jurisprudence*; D004869:Equipment Safety; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011358:Product Surveillance, Postmarketing; D016032:Randomized Controlled Trials as Topic; D014481:United States; D014486:United States Food and Drug Administration; D055815:Young Adult
keywords: children; clinical trial; device safety; medical device; pediatric; postmarketing
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