A Systematic Review Of The Food And Drug Administration's 'Exception From Informed Consent' Pathway
A Systematic Review Of The Food And Drug Administration's 'Exception From Informed Consent' Pathway
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DOI：
10.1377/hlthaff.2018.0501
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发表时间：
2018-10-01
期刊：
影响因子：
9.7
通讯作者：
Kesselheim, Aaron S.
中科院分区：
文献类型：
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作者：
Feldman, William B.;Hey, Spencer Phillips;Kesselheim, Aaron S.
1996年，美国食品和药物管理局（FDA）创建了“知情同意例外”（EFIC）途径，用于对因危及生命的疾病而丧失行为能力的人进行试验，治疗时间窗口太窄，无法获得可靠的替代同意。其目标是促进治疗创新，鼓励对这一弱势群体进行严格但符合伦理的实验。为了评估这一途径的结果，我们回顾了过去二十年来FDA EFIC试验的完整摘要，包括41项试验。在本综述纳入的46,964名患者中，96%的患者未经同意入组，不到1%的患者在主要终点前退出。在24项优势试验中，有2项（8%）证明了实验干预的益处。许多干预措施与不良反应相关，包括死亡率增加、神经功能缺损和心肌梗死。EFIC试验中近三分之一的美国患者是非裔美国人。虽然EFIC试验已经取得了医学上的进步，但在未来的试验中，研究人员必须更加注意对退出的管理和确保公平的人口代表性。
In 1996 the Food and Drug Administration (FDA) created an "exception from informed consent" (EFIC) pathway for trials conducted on people incapacitated by life-threatening conditions with a therapeutic time window too narrow for reliable surrogate consent. The goals were to promote therapeutic innovation and encourage rigorous but ethical experimentation on this vulnerable population. To evaluate outcomes of this pathway, we reviewed the complete FDA docket of EFIC trials from the past two decades, encompassing forty-one trials. Among the 46,964 patients included in this review, ninety-six percent were enrolled without consent, and fewer than 1 percent withdrew before the primary endpoint. Two (8 percent) of the twenty-four superiority trials demonstrated a benefit from the experimental interventions. Many interventions were associated with adverse effects, including increased mortality, neurological deficits, and myocardial infarctions. Nearly one-third of US patients in EFIC trials were African American. While EFIC trials have yielded medical advances, investigators in future trials must pay better attention to managing withdrawals and ensuring fair demographic representation.