Defending the Rationale for the Two-Tailed Test in Clinical Research
Defending the Rationale for the Two-Tailed Test in Clinical Research
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捍卫临床研究中双尾测试的基本原理
DOI：
10.1161/01.cir.0000018283.15527.97
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发表时间：
2002
期刊：
影响因子：
--
通讯作者：
A. Tita
中科院分区：
文献类型：
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作者：
L. Moye;A. Tita
临床试验分析中的单边和双侧假设检验问题一直是医学和统计文献中争论的主题。1-4⇓⇓⇓这种设计考虑代表着研究界的严重分歧，将研究人员对治疗有效性的根深蒂固的信念与他们对患者福利的强制性主要关注分开。在最近的一篇评论中，Knottnerus和Bouter5提出了一个理论，即对于评估以前没有研究过的新干预措施的个人试验，从伦理和成本效益的角度来看，片面的假设检验似乎都是合理的。当然，对于许多医疗研究人员来说，单尾假设检验的诱惑可能难以抗拒。在单尾假设检验中，第一类错误的位置与研究人员对研究结果的预期直觉(基于可用但有时具有误导性的信息)完全一致。对于指定的I类错误的实验概率，与单尾检验相关的较小样本量的吸引力进一步增强了它对一些研究人员的吸引力。然而，我们在这里认为，出于伦理和成本效益的原因，在医疗保健研究中应该避免单尾试验，特别是在研究人员控制干预的随机试验中。第一类错误不应反映研究人员的先验直觉，而应反映研究工作未来结论的不确定性。在一个医疗从业者和医疗研究人员可能无意中伤害患者的领域，这一点至关重要。
在临床干预试验中，我们作为研究者希望证明被测试的干预是有益的。作为临床研究人员，我们不喜欢怀有这样的想法，即我们为患者的利益而开发的干预措施可能会造成伤害。然而，危害往往是结果，正如过去医生认为他们理解的出血和有效的疾病通便药的使用所表明的那样。这些信息现在揭穿了…
The issue of one-sided versus two-sided hypothesis testing in clinical trial analyses has been the subject of debate in the medical and statistical literature.1–4⇓⇓⇓ This design consideration represents a sharp divide in the research community, separating the investigators’ deep-seated beliefs in therapy effectiveness from their obligatory primary concern for patient welfare. In a recent commentary, Knottnerus and Bouter5 advanced the theory that, for individual trials evaluating new interventions not previously studied, a one-sided hypothesis test seems sensible from both an ethical and cost-efficiency perspective. Certainly, to many healthcare researchers, the temptation of one-tailed hypothesis testing, in which the location of type I errors coincides exactly with the investigator’s prospective intuition (based on available but sometimes misleading information) about the research result, can be difficult to resist. For a specified experimental probability of type I error, the allure of the smaller sample size associated with one-tailed testing further strengthens its attraction for some researchers. We argue here, however, that one-tailed testing should be avoided in healthcare research for ethical and cost-efficiency reasons, especially in randomized trials in which the investigator controls the intervention. Rather than reflecting the investigators’ a priori intuition, the type I error should reflect the uncertainty of the research effort’s future conclusions. This is critical in a field in which healthcare practitioners and healthcare researchers can inadvertently do harm to their patients.
In clinical intervention trials, we as investigators wish to demonstrate that the tested intervention is beneficial. As clinical researchers, we do not like to harbor the notion that the interventions we have developed for the benefit of our patients can cause harm. Nevertheless, harm is often the result, as demonstrated by the use in the past of bleedings and potent purgatives for diseases that practitioners believed they understood. These now debunked …