Mercieca-Bebber Rebecca, King Madeleine T, Calvert Melanie J, Stockler Martin R, Friedlander Michael
NHMRC Clinical Trials Centre, The University of Sydney, Sydney, NSW, Australia,
Central Clinical School, Sydney Medical School, The University of Sydney, Sydney, NSW, Australia.
Patient Relat Outcome Meas. 2018 Nov 1;9:353-367. doi: 10.2147/PROM.S156279. eCollection 2018.
Patient-reported outcomes (PROs) can be included in clinical trials as primary or secondary endpoints and are increasingly recognized by regulators, clinicians, and patients as valuable tools to collect patient-centered data. PROs provide unique information on the impact of a medical condition and its treatment from the patient's perspective; therefore, PROs can be included in clinical trials to ensure the impact of a trial intervention is comprehensively assessed. This review first discusses examples of how PRO endpoints have added value to clinical trial interpretation. Second, it describes the problems with current practices in designing, implementing, and reporting PRO studies, and how these problems may be addressed by complying with guidance for protocol development, selecting appropriate PRO measures to match clinically motivated PRO hypotheses, minimizing the rates of avoidable missing PRO data, analyzing and interpreting PRO data, and transparently reporting PRO findings.
患者报告结局（PROs）可作为主要或次要终点纳入临床试验，并且越来越受到监管机构、临床医生和患者的认可，被视为收集以患者为中心数据的宝贵工具。PROs从患者角度提供了有关疾病及其治疗影响的独特信息；因此，PROs可纳入临床试验，以确保全面评估试验干预的影响。本综述首先讨论PRO终点如何为临床试验解读增添价值的示例。其次，描述了当前在设计、实施和报告PRO研究方面的做法存在的问题，以及如何通过遵循方案制定指南、选择合适的PRO测量方法以匹配具有临床意义的PRO假设、尽量降低可避免的PRO数据缺失率、分析和解读PRO数据以及透明报告PRO结果来解决这些问题。
