Clinical Trials: Discerning Hype From Substance
Clinical Trials: Discerning Hype From Substance
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DOI：
10.7326/0003-4819-153-6-201009210-00008
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发表时间：
2010-09-21
影响因子：
39.2
通讯作者：
Fleming, Thomas R.
中科院分区：
文献类型：
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作者：
Fleming, Thomas R.
能够解释和报告临床试验结果的兴趣是有利的，这在整个卫生保健研究中是普遍存在的。需要认识到这一重要的偏倚来源，并采取措施有效地解决这一问题。在新闻稿和期刊出版物中传播结果时，应明确规定临床试验的主要终点和次要终点的预先指定的主要分析。在解释结果时，应重点关注这些分析。通过进行探索性分析产生偏倚结论的重大风险，目的是确定实验干预的获益-风险特征是有利的，而不是确定是否有利。在探索性分析中，当没有提供实际的抽样背景以进行适当的解释时，P值将具有误导性，并且由于“随机高”偏倚，具有特别有利估计值的结局的效应量可能被高估。进行探索性分析应被视为产生假设，这些假设通常需要在前瞻性验证性试验中重新评估。对这些问题的认识将有意义地提高我们在做出基于证据的医疗保健决策时受到实质指导的能力，而不是炒作。
The interest in being able to interpret and report results in clinical trials as being favorable is pervasive throughout health care research. This important source of bias needs to be recognized, and approaches need to be implemented to effectively address it. The prespecified primary analyses of the primary and secondary end points of a clinical trial should be clearly specified when disseminating results in press releases and journal publications. There should be a focus on these analyses when interpreting the results. A substantial risk for biased conclusions is produced by conducting exploratory analyses with an intention to establish that the benefit-to-risk profile of the experimental intervention is favorable, rather than to determine whether it is. In exploratory analyses, P values will be misleading when the actual sampling context is not presented to allow for proper interpretation, and the effect sizes of outcomes having particularly favorable estimates are probably overestimated because of "random high" bias. Performing exploratory analyses should be viewed as generating hypotheses that usually require reassessment in prospectively conducted confirmatory trials. Awareness of these issues will meaningfully improve our ability to be guided by substance, not hype, in making evidence-based decisions about medical care.