临床试验中的多重性控制
Amy LaLonde1, Steven E Nissen2
1Eli Lilly and Company, Indianapolis.
NEJM evidence
|July 22, 2025
概括
多重假设测试可能会导致错误的发现. 本综述解释了传统方法和图形测试,以控制非统计学家的统计学意义错误.
科学领域:
- 统计 统计 统计 统计
- 生物统计学 生物统计学
- 临床试验 临床试验
背景情况:
- 多次统计假设测试增加了错误发现 (I型错误) 的风险.
- 非统计学家在理解和应用适当的统计方法时经常面临挑战.
- 准确地解释研究结果在科学研究中至关重要.
研究的目的:
- 为非统计学家介绍控制虚假发现率的方法.
- 解释管理I型错误率的传统方法.
- 提供可访问的图形测试程序概述.
主要方法:
- 对多重假设测试的传统统计方法的审查.
- 解释控制I型错误背后的原则.
- 介绍图形测试方法.
主要成果:
- 传统方法提供了管理虚假阳性风险增加的方法.
- 图形测试提供了一个结构化的程序,以保持统计有效性.
- 了解这些方法有助于防止从数据中得出错误的结论.
结论:
- 在测试多个假设时,实施适当的统计控制至关重要.
- 图形测试是一种有价值的,尽管看似复杂的工具,可以确保结果的有效性.
- 本次审查旨在消除这些概念的神秘性,以便在研究中更广泛地获得这些概念.
相关概念视频
Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
177
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
177
Clinical Trials
8.5K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
8.5K
Clinical Trials: Overview
3.4K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
3.4K
Blinding
2.6K
Blinding is a commonly used method of not telling participants which treatment a subject is receiving. Blinding is a critical part of a randomized control trial or RCT. It reduces the bias that affects the results. In an RCT, blinding is used in the form of a placebo. A placebo effect occurs when untreated subjects falsely believe they have received the treatment and report improved symptoms. A placebo or a dummy treatment is administered to subjects to negate the bias caused by such an effect.
2.6K
Multiple Comparison Tests
4.0K
Multiple comparison test, abbreviated as MCT, is a post hoc analysis generally performed after comparing multiple samples with one or more tests. An MCT will help identify a significantly different sample among multiple samples or a factor among multiple factors.
It would be easy to compare two samples using a significance alpha level of 0.05. In other words, there is only one sample pair to be compared. However, it would be difficult to identify a significantly different sample if the number...
It would be easy to compare two samples using a significance alpha level of 0.05. In other words, there is only one sample pair to be compared. However, it would be difficult to identify a significantly different sample if the number...
4.0K
Crossover Experiments
3.0K
Crossover experiments, also called the repeated-measurements design, is a study design in which all experimental units are exposed to all treatments in different periods. Crossover experiments are generally used in psychology, the pharmaceutical industry, agriculture, and medicine.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
Crossover designs are performed even with smaller sample sizes since the samples can act as their controls. These are better than simple randomized trials since patients are exposed to all the treatments.
3.0K


