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

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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 within 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.

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Data availability

All data generated or analyzed during this study are included in this published article and its supplementary information files.

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Acknowledgements

We would like to thank the many guidance authors who responded to our requests for feedback.

Funding

Funded by a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington Engagement Award (12565-JHU). Dr. Snyder is a member of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (NCI P30 CA006973).

Author information

Authors and Affiliations

  1. Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA

    Norah L. Crossnohere, Albert W. Wu & Claire Snyder

  2. Queen’s Cancer Research Institute, Queen’s University, Kingston, Canada

    Michael Brundage

  3. Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, and NIHR, Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK

    Melanie J. Calvert

  4. The University of Sydney, Sydney, Australia

    Madeleine King

  5. Duke University School of Medicine, Durham, NC, USA

    Bryce B. Reeve

  6. Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, USA

    Elissa Thorner & Claire Snyder

  7. Johns Hopkins School of Medicine, Baltimore, MD, USA

    Albert W. Wu & Claire Snyder

Authors
  1. Norah L. Crossnohere
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  2. Michael Brundage
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  8. Claire Snyder
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Corresponding author

Correspondence to Norah L. Crossnohere.

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Conflict of interest

MC reports grants from Macmillan Cancer Support, Innovate UK, Health Data Research UK, UCB Pharma the NIHR, NIHR Birmingham Biomedical Research Centre and NIHR Surgical Reconstruction and Microbiology Research Centre (SRMRC) at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, and personal fees from Astellas, Takeda, Glaukos and Merck outside the submitted work.

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Crossnohere, N.L., Brundage, M., Calvert, M.J. et al. International guidance on the selection of patient-reported outcome measures in clinical trials: a review. Qual Life Res 30, 21–40 (2021). https://doi.org/10.1007/s11136-020-02625-z

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