OneArmPhaseTwoStudy: An R Package for Planning, Conducting, and Analysing Single-Arm Phase II Studies
OneArmPhaseTwoStudy: An R Package for Planning, Conducting, and Analysing Single-Arm Phase II Studies
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DOI：
10.18637/jss.v081.i08
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发表时间：
2017-11-01
影响因子：
5.8
通讯作者：
Rauch, Geraldine
中科院分区：
文献类型：
--
作者：
Kieser, Meinhard;Wirths, Marius;Rauch, Geraldine
在临床II期研究中，首次在患者中测试有希望的疗法的疗效。根据结果，决定是否应停止开发项目，或者是否有足够的获益-风险特征来证明启动大型III期研究是合理的。在肿瘤学中，II期试验通常作为单组试验进行，计划进行中期分析，以允许因无效而提前停止。一个适当的研究设计，保证控制I型和II型错误率的规范是这样一个试验的规划阶段的关键任务。存在各种统计方法，可用于优化此类研究的规划和分析。然而，目前既没有商业也没有非商业的软件工具，支持这些方法的全面实际应用。R包OneArmPhaseTwoStudy的实现填补了这一空白。该软件包允许为手头的特定情况确定适当的研究设计，以及监控研究进展，并使用有效和高效的分析方法评估结果。本文介绍了R包的功能及其应用。
In clinical phase II studies, the efficacy of a promising therapy is tested in patients for the first time. Based on the results, it is decided whether the development programme should be stopped or whether the benefit-risk profile is promising enough to justify the initiation of large phase III studies. In oncology, phase II trials are commonly conducted as single-arm trials with planned interim analyses to allow for an early stopping for futility. The specification of an adequate study design that guarantees control of the type I and II error rates is a key task in the planning stage of such a trial. A variety of statistical methods exists which can be used to optimise the planning and analysis of such studies. However, there are currently neither commercial nor non-commercial software tools available that support the practical application of these methods comprehensively. The R package OneArmPhaseTwoStudy was implemented to fill this gap. The package allows determining an adequate study design for the particular situation at hand as well as monitoring the progress of the study and evaluating the results with valid and efficient analyses methods. This article describes the features of the R package and its application.