title: Pivotal clinical trials of novel ophthalmic drugs and medical devices: retrospective observational study, 2002-2012.
abstract: OBJECTIVE
  Novel therapeutics are an important part of ophthalmologists' armamentarium, and the risks and benefits of these therapies must be carefully evaluated. We sought to quantify the characteristics of the pivotal clinical trials supporting the regulatory approval of new ophthalmic drugs and medical devices.
  
  
  METHODS
  Retrospective observational study.
  Medical review dossiers for new ophthalmic drug and high-risk device approvals released publicly by the US Food and Drug Administration (FDA).
  
  
  METHODS
  Proportion of pivotal trials with randomisation, masking, active or placebo controls and subgroup analyses; total and median number of trial enrollees; and the number of drugs and devices approved with required postapproval studies.
  
  
  RESULTS
  From 2002 to 2012, the FDA approved 11 ophthalmic drugs and 25 devices. The pivotal trials underlying the approvals of ophthalmic drugs in our study cohort enrolled a median of 809 patients. Virtually all drug trials were randomised and masked (91%), of which 7 (70%) used a placebo control. Pivotal trials for ophthalmic devices enrolled 324 patients on average, and significantly fewer trials for ophthalmic devices versus drugs were randomised (16% vs 91%; p<0.001) or masked (12% vs 91%; p<0.001). 8 (32%) ophthalmic devices and 6 (55%) ophthalmic drugs were approved with required postapproval studies.
  
  
  CONCLUSIONS
  Ophthalmic therapeutics were approved based on varying levels of evidence. Postapproval studies could be used to confirm or refute early indications of safety and effectiveness of these therapeutics, with the study results accessible to patients and clinicians who need to make informed treatment decisions.
authors.affiliation: St John's Riverside Hospital, Yonkers, New York, USA.
authors.forename: Jenny
authors.initials: J
authors.lastname: Hwang
authors.affiliation: Faculty of Arts and Sciences, Harvard University, Cambridge, Massachusetts, USA The Blackstone Group, London, UK.
authors.forename: Thomas J
authors.identifier: http://orcid.org/0000-0003-0717-1497
authors.initials: TJ
authors.lastname: Hwang
authors.affiliation: Department of Ophthalmology, Massachusetts Eye and Ear Inﬁrmary, Harvard Medical School, Boston, Massachusetts, USA.
authors.forename: Joseph B
authors.initials: JB
authors.lastname: Ciolino
mesh_terms: D060433:Administration, Ophthalmic; D003234:Conjunctivitis, Bacterial / Q000188:drug therapy*; D003657:Decision Making; D017277:Drug Approval; D005260:Female; D006801:Humans; D007910:Lenses, Intraocular; D008268:Macular Degeneration / Q000188:drug therapy*; D008297:Male; D009883:Ophthalmic Solutions; D004364:Pharmaceutical Preparations; D016032:Randomized Controlled Trials as Topic; D012189:Retrospective Studies; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D009883:Ophthalmic Solutions; D004364:Pharmaceutical Preparations
keywords: THERAPEUTICS