Historical controls (HCs) can be used for model parameter estimation at the study design phase, adaptation within a study, or supplementation or replacement of a control arm. Currently on the latter, there is no practical roadmap from design to analysis of a clinical trial to address selection and inclusion of HCs, while maintaining scientific validity. This paper provides a comprehensive roadmap for planning, conducting, analyzing and reporting of studies using HCs, mainly when a randomized clinical trial is not possible. We review recent applications of HC in clinical trials, in which either predominantly a large treatment effect overcame concerns about bias, or the trial targeted a life-threatening disease with no treatment options. In contrast, we address how the evidentiary standard of a trial can be strengthened with optimized study designs and analysis strategies, emphasizing rare and pediatric indications. We highlight the importance of simulation and sensitivity analyses for estimating the range of uncertainties in the estimation of treatment effect when traditional randomization is not possible. Overall, the paper provides a roadmap for using HCs.

中文翻译：

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在临床试验中使用历史对照的路线图 - 由药物信息协会自适应设计科学工作组 (DIA-ADSWG) 制定。

历史对照 (HC) 可用于研究设计阶段的模型参数估计、研究内的调整或控制臂的补充或更换。目前，对于后者，还没有从临床试验的设计到分析的实际路线图来解决 HC 的选择和纳入问题，同时保持科学有效性。本文提供了一个全面的路线图，用于规划、实施、分析和报告使用 HC 的研究，主要是在无法进行随机临床试验的情况下。我们回顾了 HC 在临床试验中的最新应用，其中要么主要是巨大的治疗效果克服了对偏倚的担忧，要么试验针对的是危及生命的疾病，没有治疗选择。相比之下，我们讨论如何通过优化的研究设计和分析策略来加强试验的证据标准，强调罕见和儿科适应症。我们强调当传统随机化不可能时，模拟和敏感性分析对于估计治疗效果估计中的不确定性范围的重要性。总体而言，本文提供了使用 HC 的路线图。