受COVID-19大流行影响的临床试验：拯救的适应性设计？
Clinical trials impacted by the COVID-19 pandemic: Adaptive designs to the rescue?
摘要：
最近，新的病原体严重急性呼吸道综合征冠状病毒2（SARS-CoV-2）被确定，世界卫生组织宣布2019年冠状病毒病（COVID-19）为大流行病。疫情对正在进行的非COVID-19临床试验产生了多项影响。受四个目前正在进行的各种疾病领域的临床试验的启发，我们说明了大流行所面临的挑战，并勾勒出可能的解决方案，包括适应性设计。提供了以下方面的指导：（i）盲态调整可以提供帮助的地方;（ii）如何实现第一类错误率控制（如需要）;（iii）如何处理潜在的处理效应异质性;（iv）如何利用早期读数;以及（v）如何利用贝叶斯技术。更详细地说，研究人员制定了阻止受大流行影响的试验的方法，包括考虑提前停止试验、使用组序贯设计或调整样本量。所有考虑的方法都在一个免费的R shiny应用程序中实现。此外，还讨论了监管和运营问题，包括数据监测委员会的作用。
英文摘要：
Very recently the new pathogen severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) was identified and the coronavirus disease 2019 (COVID-19) declared a pandemic by the World Health Organization. The pandemic has a number of consequences for the ongoing clinical trials in non-COVID-19 conditions. Motivated by four currently ongoing clinical trials in a variety of disease areas we illustrate the challenges faced by the pandemic and sketch out possible solutions including adaptive designs. Guidance is provided on (i) where blinded adaptations can help; (ii) how to achieve type I error rate control, if required; (iii) how to deal with potential treatment effect heterogeneity; (iv) how to utilize early readouts; and (v) how to utilize Bayesian techniques. In more detail approaches to resizing a trial affected by the pandemic are developed including considerations to stop a trial early, the use of group-sequential designs or sample size adjustment. All methods considered are implemented in a freely available R shiny app. Furthermore, regulatory and operational issues including the role of data monitoring committees are discussed.
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