论文标题
外部控制的使用：当前可以在多大程度上推荐它？
The use of external controls: To what extent can it currently be recommended?
论文作者
论文摘要
随着在临床研究之外捕获更多和更好的临床数据，并且临床研究的更多数据共享，外部控制可能成为随机临床试验的更具吸引力的替代方法。行业和监管机构都认识到，在无法进行随机研究的情况下，外部控制可以提供所需的上下文化，以便在没有随机控制的情况下更好地解释研究。还同意，外部控制将不会完全取代随机临床试验，作为在药物开发中正式证明和临床研究的标准的金标准。但是，尚不清楚在哪些情况下得出有关功效的结论以及积极的益处/风险可以可靠地基于使用外部控制。本文将概述外部控制类型，它们的应用程序以及其使用的不同偏见来源可能会产生，并讨论潜在的缓解步骤。它还将提供有关如何使用外部控制的建议。
With more and better clinical data being captured outside of clinical studies and greater data sharing of clinical studies, external controls may become a more attractive alternative to randomized clinical trials. Both industry and regulators recognize that in situations where a randomized study cannot be performed, external controls can provide the needed contextualization to allow a better interpretation of studies without a randomized control. It is also agreed that external controls will not fully replace randomized clinical trials as the gold standard for formal proof of efficacy in drug development and the yardstick of clinical research. However, it remains unclear in which situations conclusions about efficacy and a positive benefit/risk can reliably be based on the use of an external control. This paper will provide an overview on types of external control, their applications and the different sources of bias their use may incur, and discuss potential mitigation steps. It will also give recommendations on how the use of external controls can be justified.
