The importance of patient-reported outcomes in clinical trials and strategies for future optimization

AbstractPatient-reported outcomes (PROs) can be included in clinical trials as primary or secondary endpoints and increasingly recognised by regulators, clinicians and patients as valuable tools to collect patient-centred 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 manuscript first discusses examples of how PRO endpoints have added value to clinical trial interpretation. The second half of this manuscript describes 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, minimising the rates of avoidable missing PRO data, analysing and interpreting PRO data, and transparently reporting PRO findings.