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Review
. 2018 Nov 1:9:353-367.
doi: 10.2147/PROM.S156279. eCollection 2018.

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

Rebecca Mercieca-Bebber  1 , Madeleine T King  2   3 , Melanie J Calvert  4   5 , Martin R Stockler  1 , Michael Friedlander  1   6
Affiliations
Review

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

Rebecca Mercieca-Bebber et al. Patient Relat Outcome Meas. .

Abstract

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.

Keywords: clinical trials as topic; patient-reported outcomes; quality of life; research practices; trial conduct.

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Conflict of interest statement

Disclosure Unrelated to the current work: Professor Calvert has received personal fees from Astellas Pharmaand Ferring and chairs the ISOQOL Best Practice for PROs in Trials Taskforce and Standards and Best Practice Committee.Professor Stockler’s institution has received research funding from Astellas Pharma, Celgene, Bayer, Bionomics, Medivation, Sanofi,Pfizer, AstraZeneca, Bristol-Myers Squibb, Roche, Amgen, Merck Sharp& Dohme (MSD), and Tilray and has received travel support from Medivation/Pfizer.Professor Friedlander has received honoraria for advisory boards for Astra Zeneca and MSD. The authors report no other conflicts of interest in this work.

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