Jointly assessing multiple endpoints in pilot and feasibility studies.

IF 1.9 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES
Statistical Methods in Medical Research Pub Date : 2025-03-01 Epub Date: 2025-02-12 DOI:10.1177/09622802241311219
Robert N Montgomery, Amy E Bodde, Eric D Vidoni
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引用次数: 0

Abstract

Pilot and feasibility studies are routinely used to determine whether a definitive trial should be pursued; however, the methodologies used to assess feasibility endpoints are often basic and are rarely informed by the requirements of the planned future trial. We propose a new method for analyzing feasibility outcomes which can incorporate relationships between endpoints, utilize a preliminary study design for a future trial and allow for multiple types of feasibility endpoints. The approach specifies a Joint Feasibility Space (JFS) which is the combination of feasibility outcomes that would render a future trial feasible. We estimate the probability of being in the JFS using Bayesian methods and use simulation to create a decision rule based on frequentist operating characteristics. We compare our approach to other general-purpose methods in the literature with simulation and show that our approach has approximately the same performance when analyzing a single feasibility endpoint but is more efficient with more than one endpoint. Feasibility endpoints should be the focus of pilot and feasibility studies. The analyses of these endpoints deserve more attention than they are given, and we have provided a new, effective method their assessment.

在试点和可行性研究中联合评估多个终点。
经常使用试点和可行性研究来确定是否应进行最终试验;然而,用于评估可行性终点的方法通常是基本的,很少根据计划的未来试验的要求进行评估。我们提出了一种分析可行性结果的新方法,该方法可以纳入终点之间的关系,利用未来试验的初步研究设计,并允许多种类型的可行性终点。该方法指定了一个联合可行性空间(JFS),它是使未来试验可行的可行性结果的组合。我们使用贝叶斯方法估计进入JFS的概率,并使用仿真来创建基于频率算子操作特征的决策规则。我们将我们的方法与文献中的其他通用方法进行了仿真比较,并表明我们的方法在分析单个可行性端点时具有大致相同的性能,但在分析多个端点时效率更高。可行性终点应成为试点和可行性研究的重点。对这些终点的分析值得更多的关注,我们提供了一种新的、有效的评估方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Statistical Methods in Medical Research
Statistical Methods in Medical Research 医学-数学与计算生物学
CiteScore
4.10
自引率
4.30%
发文量
127
审稿时长
>12 weeks
期刊介绍: Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)
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