Postmarketing Trials and Pediatric Device Approvals
Postmarketing Trials and Pediatric Device Approvals
复制标题
DOI：
10.1542/peds.2013-3348
复制
发表时间：
2014-05-01
期刊：
影响因子：
8
通讯作者：
Bourgeois, Florence T.
中科院分区：
文献类型：
--
作者：
Hwang, Thomas J.;Kesselheim, Aaron S.;Bourgeois, Florence T.
背景：医疗器械可用于各种疾病，但很少有器械专门批准用于儿童。2007年通过了《儿科医疗器械安全和改进法案》，以刺激儿科器械的开发。支持儿科器械批准的试验证据的当前状态仍然描述不清。方法：我们确定了2008年至2011年期间通过上市前批准或人道主义器械豁免途径批准用于儿童治疗的所有高风险（即III类）器械。我们收集了关于临床试验设计（随机化、盲法、对照和终点类型）以及试验参与者年龄分布的关键信息。我们还确定了美国食品和药物管理局（FDA）授权的上市后试验。结果：22个设备被批准用于儿童通过上市前批准途径和3通过人道主义设备豁免途径。尽管最低批准年龄为18岁（FDA器械和放射健康中心将18-21岁的患者视为儿科患者），但仍有22例（88%）符合儿科资格。大多数器械是基于非随机（59%）、开放标签（68%）研究和替代有效性终点（86%）获得批准的。总体而言，21件（84%）器械未在任何患者中进行研究
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