Pivotal clinical trials of novel ophthalmic drugs and medical devices: retrospective observational study, 2002-2012.
Pivotal clinical trials of novel ophthalmic drugs and medical devices: retrospective observational study, 2002-2012.
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新型眼科药物和医疗器械的关键临床试验：回顾性观察研究，2002-2012 年。
DOI：
10.1136/bmjopen-2015-007987
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发表时间：
2015
期刊：
影响因子：
2.9
通讯作者：
Ciolino,JosephB
中科院分区：
文献类型：
--
作者：
Hwang,Jenny;Hwang,ThomasJ;Ciolino,JosephB
目的新的治疗方法是眼科医生的重要组成部分，必须仔细评估这些治疗方法的风险和益处。我们试图量化支持新眼科药物和医疗器械监管批准的关键临床试验的特征。设计回顾性观察性研究。设置和数据来源美国食品药品监督管理局（FDA）公开发布的新眼科药物和高风险器械批准的医学审查档案。主要结局指标随机化、设盲、活性或安慰剂对照和亚组分析;试验入组的总数和中位数;以及批准的药物和设备的数量，要求批准后study.ResultsFrom 2002年至2012年，FDA批准了11种眼科药物和25种设备。在我们的研究队列中，支持眼科药物批准的关键性试验入组了中位数为809例患者。几乎所有的药物试验都是随机和设盲的（91%），其中7项（70%）使用安慰剂对照。眼科器械的临床试验平均招募了324名患者，随机（16% vs 91%; p<0.001）或设盲（12% vs 91%; p<0.001）的眼科器械与药物试验显著较少。8（32%）眼科设备和6（55%）眼科药物批准所需的批准后studies.ConclusionsOphthalmic治疗批准的基础上不同程度的证据。批准后研究可用于确认或反驳这些治疗药物的安全性和有效性的早期迹象，研究结果可供需要做出知情治疗决定的患者和临床医生使用。
ObjectivesNovel 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.DesignRetrospective observational study.Setting and data sourceMedical review dossiers for new ophthalmic drug and high-risk device approvals released publicly by the US Food and Drug Administration (FDA).Main outcome measuresProportion 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.ResultsFrom 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.ConclusionsOphthalmic 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.