标题： 使用贝叶斯方法在罕见疾病中试点可行性试验的效率 显示英文标题

标题： The efficiencies of pilot feasibility trials in rare diseases using Bayesian methods

摘要： 试点可行性研究在罕见疾病临床试验的开发中起着关键作用，其中小样本量和缓慢的招募往往威胁试验的可行性。 虽然这类研究通常用于评估操作参数，但它们也提供了一个宝贵的机会，以指导后续确定性试验的设计和分析，特别是通过使用贝叶斯方法。 在本文中，我们展示了如何通过使用稳健的元分析预测先验信息，将来自单一、符合方案的试点研究的数据纳入确定性试验中。 我们关注二分类疗效结果的情况，这是由静脉注射免疫球蛋白在自身免疫性炎症肌病中的可行性试验所引发的。 通过模拟研究，我们评估了由试点数据指导的试验的操作特性，包括样本量、预期试验持续时间和达到招募目标的概率。 我们的研究结果突显了通过稳健贝叶斯先验信息利用试点数据的操作和伦理优势，并为其在罕见疾病环境中的应用提供了实用指导。 显示英文摘要

摘要： Pilot feasibility studies play a pivotal role in the development of clinical trials for rare diseases, where small populations and slow recruitment often threaten trial viability. While such studies are commonly used to assess operational parameters, they also offer a valuable opportunity to inform the design and analysis of subsequent definitive trials-particularly through the use of Bayesian methods. In this paper, we demonstrate how data from a single, protocol-aligned pilot study can be incorporated into a definitive trial using robust meta-analytic-predictive priors. We focus on the case of a binary efficacy outcome, motivated by a feasibility trial of intravenous immunoglobulin tapering in autoimmune inflammatory myopathies. Through simulation studies, we evaluate the operating characteristics of trials informed by pilot data, including sample size, expected trial duration, and the probability of meeting recruitment targets. Our findings highlight the operational and ethical advantages of leveraging pilot data via robust Bayesian priors, and offer practical guidance for their application in rare disease settings.