Importance There is a recognized unmet need for clinical trials to provide evidence-informed care for infants, children and adolescents. This Special Communication outlines the capacity of 3 distinct trial design strategies, sequential, parallel, and a unified adult-pediatric bayesian adaptive design, to incorporate children into clinical trials and transform this current state of evidence inequity. A unified adult-pediatric whole-of-life clinical trial is demonstrated through the Staphylococcus aureus Network Adaptive Platform (SNAP) trial. Observations Bayesian methods provide a framework for synthesizing data in the form of a probability model that can be used in the design and analysis of a clinical trial. Three trial design strategies are compared: (1) a sequential adult-pediatric bayesian approach that involves a separate, deferred pediatric trial that incorporates existing adult trial data into the analysis model to potentially reduce the pediatric trial sample size; (2) a parallel adult-pediatric bayesian trial whereby separate pediatric enrollment occurs in a parallel trial, running alongside an adult randomized clinical trial; and (3) a unified adult-pediatric bayesian adaptive design that supports the enrollment of both children and adults simultaneously in a whole-of-life bayesian adaptive randomized clinical trial. The SNAP trial whole-of-life design uses a bayesian hierarchical model that allows information sharing (also known as borrowing) between trial age groups by linking intervention effects of children and adults, thereby improving inference in both groups. Conclusion and Relevance Bayesian hierarchical models may provide more precision for estimates of safety and efficacy of treatments in trials with heterogenous populations compared to traditional methods of analysis. They facilitate the inclusion of children in clinical trials and a shift from children deemed therapeutic orphans to the vision of no child left behind in clinical trials to ensure evidence for clinical practice exists across the life course. The SNAP trial provides an example of a bayesian adaptive whole-of-life inclusion design that enhances trial population inclusivity and diversity overall, as well as generalizability and translation of findings into clinical practice.

中文翻译：

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贝叶斯自适应平台临床试验的终身包容性。


重要性 临床试验为婴儿、儿童和青少年提供循证护理的需求尚未得到满足。本特别通讯概述了 3 种不同的试验设计策略（顺序、并行和统一的成人-儿童贝叶斯自适应设计）的能力，以将儿童纳入临床试验并改变目前证据不公平的状态。通过金黄色葡萄球菌网络自适应平台 (SNAP) 试验证明了统一的成人-儿童终身临床试验。观察贝叶斯方法提供了一个以概率模型形式合成数据的框架，可用于临床试验的设计和分析。比较了三种试验设计策略：（1）连续的成人-儿童贝叶斯方法，涉及单独的、延期的儿科试验，将现有的成人试验数据纳入分析模型，以潜在地减少儿科试验样本量； (2) 平行成人-儿童贝叶斯试验，其中单独的儿童入组在平行试验中进行，与成人随机临床试验同时进行； (3) 统一的成人-儿童贝叶斯适应性设计，支持儿童和成人同时参加终身贝叶斯适应性随机临床试验。 SNAP 试验的终身设计采用贝叶斯分层模型，通过将儿童和成人的干预效果联系起来，允许试验年龄组之间进行信息共享（也称为借用），从而改善两组的推理。 结论和相关性 与传统分析方法相比，贝叶斯分层模型可以更精确地估计异质人群试验中治疗的安全性和有效性。它们促进将儿童纳入临床试验，并从被视为治疗孤儿的儿童转向临床试验中不留任何儿童的愿景，以确保在整个生命过程中存在临床实践的证据。 SNAP 试验提供了贝叶斯自适应终身纳入设计的示例，该设计增强了试验人群的总体包容性和多样性，以及研究结果的普遍性和转化为临床实践。