Patient reported outcome measures in clinical trials should be initially analyzed as continuous outcomes for statistical significance and responder analyses should be reserved as secondary analyses

2021 ◽  
Vol 134 ◽  
pp. 95-102
Author(s):  
David Collister ◽  
Shrikant Bangdiwala ◽  
Michael Walsh ◽  
Rajibul Mian ◽  
Shun Fu Lee ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Statistical Significance ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Continuous Outcomes ◽  
Secondary Analyses

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

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Theresa M. Coles ◽  
Adrian F. Hernandez ◽  
Bryce B. Reeve ◽  
Karon Cook ◽  
Michael C. Edwards ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Contextual Factors ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Think Tank ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Evidentiary Standards

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.

scholarly journals International guidance on the selection of patient-reported outcome measures in clinical trials: a review

2020 ◽  
Author(s):  
Norah L. Crossnohere ◽  
Michael Brundage ◽  
Melanie J. Calvert ◽  
Madeleine King ◽  
Bryce B. Reeve ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Patient Reported ◽  
Selection Of

Gaps in patient-reported outcome measures in randomized clinical trials of cardiac catheter ablation: a systematic review

2020 ◽  
Vol 6 (4) ◽  
pp. 234-242
Author(s):  
Yang Chen ◽  
Myura Nagendran ◽  
Manuel Gomes ◽  
Peter V Wharton ◽  
Rosalind Raine ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Catheter Ablation ◽  
Outcome Measures ◽  
Comparative Effectiveness ◽  
Randomized Clinical Trials ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Cardiac Catheter ◽  
Patient Reported

Abstract The aim of this systematic review was to evaluate randomized clinical trials (RCTs) of cardiac catheter ablation (CCA) and to assess the prevalence, characteristics and reporting standards of clinically relevant patient-reported outcome measures (PROMs). Electronic database searches of Medline, Embase, CENTRAL, and the WHO Trial Registry were conducted in March 2019. The study protocol was registered on PROSPERO (CRD42019133086). Of 7125 records identified, 237 RCTs were included for analysis, representing 35 427 patients with a mean age of 59 years. Only 43 RCTs (18%) reported PROMs of which 27 included a generic PROM that measured health-related quality of life (HRQL) necessary to conduct comparative effectiveness research. There was notable under-representation of certain patient groups—only 31% were women and only 8% were of non-Caucasian ethnicity, in trials which reported such data. The reporting standard of PROMs was highly variable with 8–62% adherence against CONSORT PRO-specific items. In summary, PROMs play a crucial role in determining the clinical and cost-effectiveness of treatments which primarily offer symptomatic improvement, such as CCA. Their underuse significantly limits evaluation of the comparative effectiveness of treatments. Using CCA as an exemplar, there are additional issues of infrequent assessment, poor reporting and under-representation of many population groups. Greater use of PROMs, and specifically validated HRQL questionnaires, is paramount in giving patients a voice in studies, generating more meaningful comparisons between treatments and driving better patient-centred clinical and policy-level decision-making.

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