Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2021 Jan;30(1):21-40.
doi: 10.1007/s11136-020-02625-z. Epub 2020 Sep 14.

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

Norah L Crossnohere  1 , Michael Brundage  2 , Melanie J Calvert  3 , Madeleine King  4 , Bryce B Reeve  5 , Elissa Thorner  6 , Albert W Wu  7   8 , Claire Snyder  7   6   8
Affiliations
Review

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

Norah L Crossnohere et al. Qual Life Res. 2021 Jan.

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.

Keywords: Clinical trials; Guidance as topic; Health care; Oncology; Outcome assessment; Quality of life.

PubMed Disclaimer

References

    1. Acquadro, C., et al. (2003). Incorporating the patient's perspective into drug development and communication: An ad hoc task force report of the patient-reported outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. Value Health, 6(5), 522–531. - PubMed
    1. Au, H. J., et al. (2010). Added value of health-related quality of life measurement in cancer clinical trials: The experience of the NCIC CTG. Expert Review of Pharmacoeconomics & Outcomes Research, 10(2), 119–128.
    1. Till, J. E., et al. (1994). Research on health-related quality of life: Dissemination into practical applications. Quality of Life Research, 3(4), 279–283. - PubMed
    1. Lipscomb, J., Gotay, C. C., & Snyder, C. (2004). Outcomes assessment in cancer: Measures, methods and applications. Cambridge: Cambridge University Press.
    1. Brundage, M., et al. (2011). A knowledge translation challenge: Clinical use of quality of life data from cancer clinical trials. Quality of Life Research, 20(7), 979–985. - PubMed

LinkOut - more resources