Systematic evaluation of Patient-Reported Outcome protocol content and reporting in cancer trials

Derek Kyte, Ameeta Retzer, Khaled Ahmed, Thomas Keeley, Jo Armes, Julia M Brown, Lynn Calman, Anna Gavin, Adam Glaser, Diana M Greenfield, Anne Lanceley, Rachel M Taylor, Galina Velikova, Michael Brundage, Fabio Efficace, Rebecca Mercieca-bebber, Madeleine T. King, Grace Turner, Melanie Calvert

Research output: Contribution to journalArticlepeer-review

21 Citations (Scopus)
163 Downloads (Pure)

Abstract

Background: Patient-reported outcomes (PROs) are captured within cancer trials to help future patients and their clinicians make more informed treatment decisions. However, variability in standards of PRO trial design and reporting threaten the validity of these endpoints for application in clinical practice. Methods: We systematically investigated a cohort of randomized controlled cancer trials that included a primary or secondary PRO. For each trial, an evaluation of protocol and reporting quality was undertaken using standard checklists. General patterns of reporting where also explored. Results: Protocols (101 sourced, 44.3%) included a mean (SD) of 10 (4) of 33 (range = 2-19) PRO protocol checklist items. Recommended items frequently omitted included the rationale and objectives underpinning PRO collection and approaches to minimize/address missing PRO data. Of 160 trials with published results, 61 (38.1%, 95% confidence interval = 30.6% to 45.7%) failed to include their PRO findings in any publication (mean 6.43-year follow-up); these trials included 49 568 participants. Although two-thirds of included trials published PRO findings, reporting standards were often inadequate according to international guidelines (mean [SD] inclusion of 3 [3] of 14 [range = 0-11]) CONSORT PRO Extension checklist items). More than one-half of trials publishing PRO results in a secondary publication (12 of 22, 54.5%) took 4 or more years to do so following trial closure, with eight (36.4%) taking 5-8 years and one trial publishing after 14 years. Conclusions: PRO protocol content is frequently inadequate, and nonreporting of PRO findings is widespread, meaning patient-important information may not be available to benefit patients, clinicians, and regulators. Even where PRO data are published, there is often considerable delay and reporting quality is suboptimal. This study presents key recommendations to enhance the likelihood of successful delivery of PROs in the future.

Original languageEnglish
Pages (from-to)1170-1178
Number of pages9
JournalJournal of the National Cancer Institute
Volume111
Issue number11
Early online date11 Apr 2019
DOIs
Publication statusPublished - Nov 2019

Bibliographical note

© The Author(s) 2019. Published by Oxford University Press.

Keywords

  • cancer
  • follow-up
  • guidelines
  • surrogate endpoints
  • patient self-report
  • reporting standards
  • performance reporting

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Fingerprint

Dive into the research topics of 'Systematic evaluation of Patient-Reported Outcome protocol content and reporting in cancer trials'. Together they form a unique fingerprint.

Cite this