群组顺序的两阶段偏好设计
Ruyi Liu1,2, Fan Li1,3, Denise Esserman1,3
1Department of Biostatistics, Yale School of Public Health, New Haven, Connecticut, USA.
Statistics in medicine
|November 27, 2023
概括
本研究引入了一种群体顺序两阶段偏好设计 (GS-TSPD),以减少在临床试验中估计治疗效应,选择效应和偏好效应的样本大小要求.
科学领域:
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
- 医疗保健服务研究 医疗服务研究
背景情况:
- 两阶段偏好设计 (TSPD) 允许对治疗疗效,患者选择效应和偏好效应进行公正的估计.
- 一个重要的TSPD限制是它对大样本大小的要求,这影响了实际实施.
- 患者的偏好显著影响治疗结果,需要考虑这一因素的设计.
研究的目的:
- 提出一个组顺序两阶段偏好设计 (GS-TSPD),以提高TSPD的效率.
- 为了在发现治疗,选择或偏好效应的充分证据时,能够提前停止临床试验.
- 通过结合顺序监测来减少临床试验中预期的资源分配.
主要方法:
- 拟议的GS-TSPD将顺序监测与传统的TSPD框架相结合.
- 重复的假设测试在中间分析期间对累积的数据进行,以评估研究终止标准.
- 集团顺序停止边界用于保持I型错误率,使用近似的顺序密度函数.
主要成果:
- 模拟表明,与传统的TSPD相比,GS-TSPD可以在较小的样本大小下实现所需的统计能力.
- 拟议的设计有效地控制了I型错误率,同时允许提前终止.
- 通过一项涉及C型肝炎治疗方式的案例研究,说明了GS-TSPD的适用性.
结论:
- 在涉及患者偏好的临床试验中,GS-TSPD为传统TSPD提供了一个统计严格和资源高效的替代方案.
- 该设计通过在积累证据的基础上提前结束试验,从而促进及时决策.
- 对于旨在了解受患者选择影响的复杂治疗效应的研究来说,GS-TSPD是一种有价值的方法进步.
更多相关视频
相关概念视频
02:01Group 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
02:01Factorial Design
13.0K
Factorial Analysis is an experimental design that applies Analysis of Variance (ANOVA) statistical procedures to examine a change in a dependent variable due to more than one independent variable, also known as factors. Changes in worker productivity can be reasoned, for example, to be influenced by salary and other conditions, such as skill level. One way to test this hypothesis is by categorizing salary into three levels (low, moderate, and high) and skills sets into two levels (entry level...
13.0K
01:16Crossover Experiments
2.9K
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.
2.9K
01:16Experimental Designs
11.4K
An experimental design is a systematic process that allows researchers to evaluate the relationship between dependent and independent variables. There are three widely used types of experimental design - pre-experimental design, true experimental design, and quasi-experimental design. In pre-experimental design, the researcher compares the data before and after some interventions or treatments. The true-experimental design has more than one purposefully created group, a commonly measured...
11.4K
01:13Randomized Experiments
7.0K
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...
7.0K
01:50Cross-Sectional Research
11.3K
In cross-sectional research, a researcher compares multiple segments of the population at the same time. If they were interested in people's dietary habits, the researcher might directly compare different groups of people by age. Instead of following a group of people for 20 years to see how their dietary habits changed from decade to decade, the researcher would study a group of 20-year-old individuals and compare them to a group of 30-year-old individuals and a group of 40-year-old...
11.3K