International guidance on the selection of patient-reported outcome measures in clinical trials: a review
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REVIEW
International guidance on the selection of patient‑reported outcome measures in clinical trials: a review Norah L. Crossnohere1 · Michael Brundage2 · Melanie J. Calvert3 · Madeleine King4 · Bryce B. Reeve5 · Elissa Thorner6 · Albert W. Wu1,7 · Claire Snyder1,6,7 Accepted: 26 August 2020 © Springer Nature Switzerland AG 2020
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 Outcome assessment · Quality of life · Guidance as topic · Health care · Clinical trials · Oncology
* Norah L. Crossnohere [email protected] 1
Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA
2
Queen’s Cancer Research Institute, Queen’s University, Kingston, Canada
3
Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, and NIHR, Birmingham Biomedical Research Centre, University of Birmingham, Birmingham, UK
4
The University of Sydney, Sydney, Australia
5
Duke University School of Medicine, Durham, NC, USA
6
Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, USA
7
Johns Hopkins School of Medicine, Baltimore, MD, USA
Introduction Patient-reported outcome (PRO) measures (PROMs) are a critical aspect of clinical trials given their ability to directly capture the
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