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

Rebecca Mercieca-Bebber1 NHMRC Clinical Trials Centre, The University of Sydney, Sydney, NSW, Australia, [email protected]; [email protected]Correspondence[email protected] [email protected]

Madeleine T King2 Central Clinical School, Sydney Medical School, The University of Sydney, Sydney, NSW, Australia;3 School of Psychology, The University of Sydney, Sydney, NSW, Australia

Melanie J Calvert4 Centre for Patient-Reported Outcomes Research and NIHR Birmingham Biomedical Research Centre, University of Birmingham, Edgbaston, Birmingham, UK;5 Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, UK

Martin R Stockler1 NHMRC Clinical Trials Centre, The University of Sydney, Sydney, NSW, Australia, [email protected]; [email protected]

Michael Friedlander1 NHMRC Clinical Trials Centre, The University of Sydney, Sydney, NSW, Australia, [email protected]; [email protected];6 Prince of Wales Clinical School, University of New South Wales, Sydney, NSW, Australia, [email protected]Correspondence[email protected] [email protected]

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.

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Acknowledgments

RMB is funded by a NHMRC Early Career Fellowship. MK is funded by the Australian Government courtesy of Cancer Australia. MC is partly funded by the NIHR Birmingham Biomedical Research Centre and the NIHR Surgical Reconstruction and Microbiology Research Centre at the University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham. The views expressed are those of the author and not necessarily those of the NHMRC, Cancer Australia, NHS, the NIHR or the Department of Health.

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.

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

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

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