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

Research output: Contribution to journalArticle

Authors

  • Norah L. Crossnohere
  • Michael Brundage
  • Madeleine King
  • Bryce B. Reeve
  • Elissa Thorner
  • Albert W Wu
  • Claire Snyder

Colleges, School and Institutes

External organisations

  • THE QUEENS UNIVERSITY
  • Psycho-oncology Co-operative Research Group
  • UNIVERSITY OF SYDNEY
  • Department of Health Policy and Management
  • Gillings School of Global Public Health
  • Division of Rheumatology, Department of Medicine, University of North Carolina, Chapel Hill, North Carolina, USA.
  • Institute of Cell Engineering, School of Medicine, Johns Hopkins University, Baltimore, MD, USA.

Abstract

Purpose: Patient-reported outcomes (PROs) are increasingly used in clinical trials to provide patients’ perspectives regarding symptoms, health-related quality of life, and satisfaction with treatments. A range of guidance documents exist for the selection of patient-reported outcome measures (PROMs) in clinical trials, and it is unclear to what extent these documents present consistent recommendations.

Methods: We conducted a targeted review of publications and regulatory guidance documents that advise on the selection of PROMs for use in clinical trials. A total of seven guidance documents from the US Food and Drug Administration, European Medicines Agency, and scientific consortia from professional societies were included in the final review. Guidance documents were analyzed using a content analysis approach comparing them with Minimum Standards recommended by the International Society for Quality of Life Research.

Results: Overall there was substantial agreement between guidance regarding the appropriate considerations for PROM selection for a clinical trial. Variations among the guidance primarily related to differences in their format and differences in the perspectives and mandates of their respective organizations. Whereas scientific consortia tended to produce checklist or rating-type guidance, regulatory groups tended to use more narrative-based approaches sometimes supplemented with lists of criteria.

Conclusion: The consistency in recommendations suggests an emerging consensus in the field and supports use of any of the major guidance documents available to guide PROM selection for clinical trials without concern of conflicting recommendations. This work represents an important first step in the international PROTEUS Consortium’s ongoing efforts to optimize the use of PROs in clinical trials.

Details

Keywords

  • Outcome assessment, Quality of life, Guidance as topic, Health care, Clinical trials, Oncology