Outcomes Research, Evidera, Inc., Bethesda, MD, USA.
Patient-Reported Outcome Consortium, Critical Path Institute, Tucson, AZ, USA.
Value Health. 2014 Jul;17(5):501-16. doi: 10.1016/j.jval.2014.06.005.
The objective of this report was to address the use and mixing of data collection modes within and between trials in which patient-reported outcome (PRO) end points are intended to be used to support medical product labeling. The report first addresses the factors that should be considered when selecting a mode or modes of PRO data collection in a clinical trial, which is often when mixing is first considered. Next, a summary of how to "faithfully" migrate instruments is presented followed by a section on qualitative and quantitative study designs used to evaluate measurement equivalence of the new and original modes of data collection. Finally, the report discusses a number of issues that must be taken into account when mixing modes is deemed necessary or unavoidable within or between trials, including considerations of the risk of mixing at different levels within a clinical trial program and mixing between different types of platforms. In the absence of documented evidence of measurement equivalence, it is strongly recommended that a quantitative equivalence study be conducted before mixing modes in a trial to ensure that sufficient equivalence can be demonstrated to have confidence in pooling PRO data collected by the different modes. However, we also strongly discourage the mixing of paper and electronic field-based instruments and suggest that mixing of electronic modes be considered for clinical trials and only after equivalence has been established. If proceeding with mixing modes, it is important to implement data collection carefully in the trial itself in a planned manner at the country level or higher and minimize ad hoc mixing by sites or individual subjects. Finally, when mixing occurs, it must be addressed in the statistical analysis plan for the trial and the ability to pool the data must be evaluated to then evaluate treatment effects with mixed modes data. A successful mixed modes trial requires a "faithful migration," measurement equivalence established between modes, and carefully planned implementation to minimize the risk of increased measurement error impacting the power of the trial to detect a treatment effect.
本报告的目的是解决旨在支持医疗产品标签的患者报告结局（PRO）终点的临床试验中，数据收集模式在试验内和试验间的使用和混合问题。报告首先讨论了在临床试验中选择 PRO 数据收集模式或混合模式时应考虑的因素，这通常是首次考虑混合模式的时候。接下来，总结了如何“忠实地”迁移工具，然后介绍了用于评估新的和原始数据收集模式的测量等效性的定性和定量研究设计。最后，报告讨论了在试验内或试验间认为混合模式是必要或不可避免时必须考虑的一些问题，包括在临床试验项目的不同层次上混合的风险以及在不同类型的平台之间混合的考虑。在没有文件证明测量等效性的情况下，强烈建议在试验中混合模式之前进行定量等效性研究，以确保可以证明足够的等效性，从而有信心对不同模式收集的 PRO 数据进行汇总。但是，我们也强烈反对纸质和电子现场仪器的混合，并建议仅在建立等效性后才考虑在临床试验中混合电子模式。如果要混合模式，重要的是在试验本身中以计划的方式在国家或更高层次上仔细收集数据，并尽量减少由站点或个别受试者进行的临时混合。最后，当发生混合模式时，必须在试验的统计分析计划中解决，并评估混合数据的汇总能力，然后评估混合模式数据的治疗效果。成功的混合模式试验需要“忠实地迁移”、模式之间建立测量等效性以及精心计划的实施，以最大程度地降低增加测量误差影响试验检测治疗效果的能力的风险。
