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Open Access 01-12-2021 | Commentary

Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

Authors: Theresa M. Coles, Adrian F. Hernandez, Bryce B. Reeve, Karon Cook, Michael C. Edwards, Marc Boutin, Elizabeth Bush, Arnold Degboe, Lothar Roessig, Amy Rudolph, Pauline McNulty, Nikunj Patel, Trish Kay-Mugford, Margaret Vernon, Michael Woloschak, Gustavo Buchele, John A. Spertus, Matthew T. Roe, Denise Bury, Kevin Weinfurt

Published in: Health and Quality of Life Outcomes | Issue 1/2021

Abstract

Objectives

There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge.

Methods

Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States.

Results

Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims.

Conclusions

A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

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Title: Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials
Authors: Theresa M. Coles
Adrian F. Hernandez
Bryce B. Reeve
Karon Cook
Michael C. Edwards
Marc Boutin
Elizabeth Bush
Arnold Degboe
Lothar Roessig
Amy Rudolph
Pauline McNulty
Nikunj Patel
Trish Kay-Mugford
Margaret Vernon
Michael Woloschak
Gustavo Buchele
John A. Spertus
Matthew T. Roe
Denise Bury
Kevin Weinfurt
Publication date: 01-12-2021
Publisher: BioMed Central
Published in: Health and Quality of Life Outcomes / Issue 1/2021
Electronic ISSN: 1477-7525
DOI: https://doi.org/10.1186/s12955-021-01800-1

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

Abstract

Objectives

Methods

Results

Conclusions

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Objectives

Methods

Results

Conclusions

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