探索对人口的分层策略 - 与基于随机化的推理对比
Marco Novelli1, William F Rosenberger2
1Department of Statistics Bologna, University of Bologna, Bologna, Italy.
Pharmaceutical statistics
|July 10, 2024
概括
在临床试验中,预分层提供了最小的好处,并且可以降低推理精度,特别是基于随机化的方法. 这种策略甚至可能是有害的,影响实验结果的可信度.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 实验设计 实验设计
背景情况:
- 分层是常见的临床试验实践,以确保基线共变量平衡.
- 在科学文献中,分层对推理精度的实际好处仍在争论中.
- 之前的研究表明,分层的效率增长有限或可以忽略不计,特别是在大样本中.
研究的目的:
- 在临床试验中研究子组分析策略.
- 将预分层的推断精度与分层后和 hoc后回归调整进行比较.
- 讨论每个方法的基于人口的与基于随机化的推断.
主要方法:
- 对前分层,后分层和回归调整进行比较分析.
- 对临床试验策略的评估,考虑治疗对共变体的相互作用.
- 推断方法的评估,包括基于人口的和基于随机化的方法.
主要成果:
- 预分层一般在临床试验中没有提供实质性的推断益处.
- 预分层可能是有害的,特别是基于随机化的推断,具有时间偏差.
- 即使有治疗因共变量相互作用,预分层可能会降低推断精度.
结论:
- 通常不建议在临床试验中常规使用预分层.
- 其他方法,如后分层或回归调整,可能会提供更好的推断精度.
- 在选择子组分析策略时,仔细考虑潜在的偏见和相互作用至关重要.
相关概念视频
Stratified Sampling Method
11.9K
Sampling is a technique to select a portion (or subset) of the larger population and study that portion (the sample) to gain information about the population. The sampling method ensures that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest.
To choose a stratified sample, divide the population into groups called strata and then take a...
To choose a stratified sample, divide the population into groups called strata and then take a...
11.9K
Randomized Experiments
6.9K
The randomization process involves assigning study participants randomly to experimental or control groups based on their probability of being equally assigned. Randomization is meant to eliminate selection bias and balance known and unknown confounding factors so that the control group is similar to the treatment group as much as possible. A computer program and a random number generator can be used to assign participants to groups in a way that minimizes bias.
Simple randomization
Simple...
Simple randomization
Simple...
6.9K
Sampling Plans
180
Sampling is a crucial step in analytical chemistry, allowing researchers to collect representative data from a large population. Common sampling methods include random, judgmental, systematic, stratified, and cluster sampling.
Random sampling is a method where each member of the population has an equal chance of being selected for the sample. It involves selecting individuals randomly, often using random number generators or lottery-type methods. For example, when analyzing the properties of a...
Random sampling is a method where each member of the population has an equal chance of being selected for the sample. It involves selecting individuals randomly, often using random number generators or lottery-type methods. For example, when analyzing the properties of a...
180
Strategies for Assessing and Addressing Confounding
92
Confounding is a critical issue in epidemiological studies, often leading to misleading conclusions about associations between exposures and outcomes. It occurs when the relationship between the exposure and the outcome is mixed with the effects of other factors that influence the outcome. Given that, addressing confounding is of high importance for drawing accurate inferences in research.
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
Confounding can be addressed at both the design phase of a study and through analytical methods after data...
92
Cluster Sampling Method
11.9K
Appropriate sampling methods ensure that samples are drawn without bias and accurately represent the population. Because measuring the entire population in a study is not practical, researchers use samples to represent the population of interest.
To choose a cluster sample, divide the population into clusters (groups) and then randomly select some of the clusters. All the members from these clusters are in the cluster sample. For example, if you randomly sample four departments from your...
To choose a cluster sample, divide the population into clusters (groups) and then randomly select some of the clusters. All the members from these clusters are in the cluster sample. For example, if you randomly sample four departments from your...
11.9K
Group Design
8.9K
The most basic experimental design involves two groups: the experimental group and the control group. The two groups are designed to be the same except for one difference— experimental manipulation. The experimental group gets the experimental manipulation—that is, the treatment or variable being tested—and the control group does not. Since experimental manipulation is the only difference between the experimental and control groups, we can be sure that any differences between...
8.9K


