外部试点试验的三种结果设计,包括进度标准
Duncan T Wilson1, Eleanor Hudson2, Sarah Brown2
1Clinical Trials Research Unit, Leeds Institute of Clinical Trials Research, University of Leeds, Leeds, LS2 9JT, UK. D.T.Wilson@leeds.ac.uk.
BMC medical research methodology
|October 2, 2024
概括
三种结果的试验设计为试点研究提供了灵活的框架,优化了进展标准和样本大小. 然而,这些设计可能需要比较简单的两种结果替代方案更大的样本大小.
科学领域:
- 生物统计学 生物统计学
- 临床试验设计 临床试验设计
- 药学研究 药学研究
背景情况:
- 试点研究对于决定是否进入最终试验至关重要.
- 进度标准和试点样本大小的确定缺乏全面的研究.
- 现有的方法通常依赖于预先规定的定量进展标准,有三种可能的结果.
研究的目的:
- 将三种结果试验设计扩展到外部试点研究.
- 提出一个统一的框架,以优化试点试验的进展标准和样本大小.
- 将三种结果设计的效率与两种结果替代方案进行比较.
主要方法:
- 审查二期瘤学的三个结果设计.
- 开发基于单变量假设测试和频率错误率控制的统一框架.
- 将框架应用于一个例子并与两种结果设计进行比较.
主要成果:
- 三种结果设计适用于试点研究,但并不总是比两种结果设计更有效.
- 它们允许结合边界结果的额外信息,尽管试验样本大小较大.
- 如果效应可预测,这些设计可以促进前最终试验调整.
结论:
- 拟议的三种结果框架优化了试点试验进展标准和样本大小,以满足所需的操作特征.
- 它适用于有或没有预期的试点后调整.
- 一般来说,三种结果设计需要比两种结果选择更大的样本大小.
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