Design of platform trials with a change in the control treatment arm.

IF 1.7 4区 数学 Q3 BIOLOGY
Biometrics Pub Date : 2025-04-02 DOI:10.1093/biomtc/ujaf073
Peter Greenstreet, Thomas Jaki, Alun Bedding, Pavel Mozgunov
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引用次数: 0

Abstract

Platform trials are an efficient way of testing multiple treatments. We consider platform trials where, if a treatment is found to be superior to the control, it will become the new standard of care. The remaining treatments are then tested against this new control. In this setting, one can either keep the information on both the new standard of care and the other active treatments before the control is changed or discard this information when testing for benefit of the remaining treatments. We show analytically and numerically, retaining the information collected before the change in control can be detrimental to the power in a frequentist multi-arm multi-stage trial. Specifically, we consider the overall power, the probability that the active treatment with the greatest treatment effect is found during the trial, and the conditional power, the probability a given treatment is found superior against the current control. Also studied is the conditional type I error, the probability a given treatment is incorrectly found superior against the current control. We prove when retaining the information decreases both the overall and conditional power but also decreases the conditional type I error. A motivating example is then studied. Based on these observations, we discuss different aspects to consider when deciding whether to run a continuous platform trial or run an inherently new trial using the same trial infrastructure.

改变控制治疗臂的平台试验设计。
平台试验是测试多种治疗方法的有效方法。我们考虑平台试验,如果发现一种治疗优于对照组,它将成为新的护理标准。然后对剩余的处理方法进行新的对照试验。在这种情况下,人们可以在改变对照之前保留关于新的护理标准和其他积极治疗的信息,或者在测试剩余治疗的益处时丢弃这些信息。我们通过分析和数值表明,保留在控制变化之前收集的信息可能对频率学家多臂多阶段试验中的功率有害。具体来说,我们考虑总体功率,即在试验期间发现治疗效果最大的积极治疗的概率,以及条件功率,即发现给定治疗优于当前控制的概率。还研究了条件I型误差,即给定处理被错误地发现优于当前控制的概率。我们证明,保留信息不仅降低了总功率和条件功率,而且还降低了条件I型误差。然后研究了一个激励的例子。基于这些观察,我们讨论了在决定是运行连续平台试验还是使用相同的试验基础设施运行固有的新试验时需要考虑的不同方面。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Biometrics
Biometrics 生物-生物学
CiteScore
2.70
自引率
5.30%
发文量
178
审稿时长
4-8 weeks
期刊介绍: The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.
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