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We sought to quantify the characteristics of the pivotal clinical trials supporting the regulatory approval of new ophthalmic drugs and medical devices.<\/jats:p><\/jats:sec><jats:sec><jats:title>Design<\/jats:title><jats:p>Retrospective observational study.<\/jats:p><\/jats:sec><jats:sec><jats:title>Setting and data source<\/jats:title><jats:p>Medical review dossiers for new ophthalmic drug and high-risk device approvals released publicly by the US Food and Drug Administration (FDA).<\/jats:p><\/jats:sec><jats:sec><jats:title>Main outcome measures<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>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&lt;0.001) or masked (12% vs 91%; p&lt;0.001). 8 (32%) ophthalmic devices and 6 (55%) ophthalmic drugs were approved with required postapproval studies.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>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.<\/jats:p><\/jats:sec>","DOI":"10.1136\/bmjopen-2015-007987","type":"journal-article","created":{"date-parts":[[2015,6,5]],"date-time":"2015-06-05T01:57:21Z","timestamp":1433469441000},"page":"e007987","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":13,"title":["Pivotal clinical trials of novel ophthalmic drugs and medical devices: retrospective observational study, 2002\u20132012"],"prefix":"10.1136","volume":"5","author":[{"given":"Jenny","family":"Hwang","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0717-1497","authenticated-orcid":false,"given":"Thomas J","family":"Hwang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph B","family":"Ciolino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2015,6,4]]},"reference":[{"key":"2024051513581398000_5.6.e007987.1","doi-asserted-by":"publisher","DOI":"10.1136\/bjophthalmol-2011-300539"},{"key":"2024051513581398000_5.6.e007987.2","doi-asserted-by":"publisher","DOI":"10.1136\/bmjopen-2013-003471"},{"key":"2024051513581398000_5.6.e007987.3","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1001\/jamaophthalmol.2015.54","article-title":"Retinal implants and medicare reimbursement policies for breakthrough treatments in ophthalmology","volume":"133","author":"Hwang","year":"2015","journal-title":"JAMA Ophthalmol"},{"key":"2024051513581398000_5.6.e007987.4","doi-asserted-by":"publisher","DOI":"10.1038\/nm1010-1107"},{"key":"2024051513581398000_5.6.e007987.5","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.316.7126.201"},{"key":"2024051513581398000_5.6.e007987.6","unstructured":"21 Code of Federal Regulations \u00a7 314.126."},{"key":"2024051513581398000_5.6.e007987.7","unstructured":"21 Code of Federal Regulations \u00a7 814.100."},{"key":"2024051513581398000_5.6.e007987.8","doi-asserted-by":"publisher","DOI":"10.1001\/archinternmed.2011.396"},{"key":"2024051513581398000_5.6.e007987.9","doi-asserted-by":"publisher","DOI":"10.1002\/pds.1825"},{"key":"2024051513581398000_5.6.e007987.10","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1110823"},{"key":"2024051513581398000_5.6.e007987.11","doi-asserted-by":"crossref","first-page":"pii: e002088","DOI":"10.1136\/bmjopen-2012-002088","article-title":"Decline in new drug launches: myth or reality? 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