Point estimation for adaptive trial designs II: practical considerations and guidance
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自适应试验设计的点估计（二）：实践考量与指南
- 研究问题
- 自适应临床试验完成后，应如何选择和报告处理效应点估计，并处理不同估计量在单次试验中可能明显分歧的问题？
- 方法与思路
- 作者讨论偏差影响及当前报告实践，以真实自适应试验演示不同估计量和代码，并基于该案例进行模拟研究。
- 材料陈述的贡献
- 摘要称不同估计量平均值虽可能接近，单次试验结果却可明显不同；作者建议预先规定估计策略并优先考虑无偏或减偏估计。
- 阅读关注点
- 核对真实试验的自适应规则、候选估计量及模拟设定，评估选择建议如何平衡偏差、变异性和具体试验的估计目标。
- 阅读边界
- 这是 arXiv 预印本，不能据此认定已同行评审或获期刊接收；摘要未给估计差异数值，实践建议的适用范围仍需全文核对。
原文摘要
In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is desirable to report estimates of treatment effects that reduce or remove this bias. However, it may be unclear which of the available estimators are preferable, and their use remains rare in practice. This paper is the second in a two-part series that studies the issue of bias in point estimation for adaptive trials. Part I provided a methodological review of approaches to remove or reduce the potential bias in point estimation for adaptive designs. In part II, we discuss how bias can affect standard estimators and assess the negative impact this can have. We review current practice for reporting point estimates and illustrate the computation of different estimators using a real adaptive trial example (including code), which we use as a basis for a simulation study. We show that while on average the values of these estimators can be similar, for a particular trial realisation they can give noticeably different values for the estimated treatment effect. Finally, we propose guidelines for researchers around the choice of estimators and the reporting of estimates following an adaptive design. The issue of bias should be considered throughout the whole lifecycle of an adaptive design, with the estimation strategy pre-specified in the statistical analysis plan. When available, unbiased or bias-reduced estimates are to be preferred.