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

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

Research output: Contribution to journalComment/debatepeer-review

9 Scopus citations


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.

Original languageEnglish (US)
Article number164
JournalHealth and Quality of Life Outcomes
Issue number1
StatePublished - Dec 2021


  • Clinical trials
  • Patient-reported outcome measures
  • Think Tank meeting results

ASJC Scopus subject areas

  • Public Health, Environmental and Occupational Health


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