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Cited by 144Meta-analysis and systematic reviewsStatistical Methods in Clinical TrialsDelphi Technique in Research

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SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials

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Calvert, Melanie, King, Madeleine, Mercieca-Bebber, Rebecca, Aiyegbusi, Olalekan, Kyte, Derek, Slade, Anita, Chan, An-Wen, Basch, E, Bell, Jill, Bennett, Antonia, Bhatnagar, Vishal, Blazeby, Jane, Bottomley, Andrew, Brown, Julia, Brundage, Michael, Campbell, Lisa, Cappelleri, Joseph C, Draper, Heather, Dueck, Amylou C, Ells, Carolyn, Frank, Lori, Golub, Robert M, Griebsch, Ingolf, Haywood, Kirstie, Hunn, Amanda, King-Kallimanis, Bellinda, Martin, Laura, Mitchell, Sandra, Morel, Thomas, Nelson, Linda, Norquist, Josephine, O'Connor, Daniel, Palmer, Michael, Patrick, Donald, Price, Gary, Regnault, Antoine, Retzer, Ameeta, Revicki, Dennis, Scott, Jane, Stephens, Richard, Turner, Grace, Valakas, Antonia, Velikova, Galina, von Hildebrand, Maria, Walker, Anita, Wenzel, Lari·2021·BMJ Open
DOI 10.1136/bmjopen-2020-045105PMC PMC8246371
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Abstract

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients’ symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item’s importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.

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  • The development of a validated PROM requires a rigorous, multistep process that incorporates both qualitative and quantitative methods, which is beyond the scope of this study.

    “The development of a validated PROM requires a rigorous, multistep process that incorporates both qualitative and quantitative methods, which is beyond the scope of this study (Calvert et al. 2021; Rothrock et al. 2011).”

    From 1st Global Consensus for Clinical Guidelines: Identifying a Core Outcome Set for Implant Dentistry in Edentulous Maxilla Rehabilitation (2026) · cites this paper as Calvert et al. 2021

    In Methods: data collection, management and analysis > Explanation (page unavailable), Calvert et al. say that a new PRO instrument should have its measurement properties evaluated according to established validation guidelines. That supports the need for careful validation, but our copy does not describe PROM development as a multistep process requiring both qualitative and quantitative methods. The statement that development is beyond the citing study’s scope is the citing authors’ own assessment. (LLM, conf=0.84)

    Experimental — accuracy not yet measured

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Metadata

Authors
Calvert, M., King, M., Mercieca-Bebber, R., Aiyegbusi, O., Kyte, D., Slade, A., Chan, A.-W., Basch, E., Bell, J., Bennett, A., Bhatnagar, V., Blazeby, J., Bottomley, A., Brown, J., Brundage, M., Campbell, L., Cappelleri, J. C., Draper, H., Dueck, A. C., Ells, C., Frank, L., Golub, R. M., Griebsch, I., Haywood, K., Hunn, A., King-Kallimanis, B., Martin, L., Mitchell, S., Morel, T., Nelson, L., Norquist, J., O'Connor, D., Palmer, M., Patrick, D., Price, G., Regnault, A., Retzer, A., Revicki, D., Scott, J., Stephens, R., Turner, G., Valakas, A., Velikova, G., von Hildebrand, M., Walker, A., & Wenzel, L.
Affiliations
  • NIHR Surgical Reconstruction and Microbiology Research Centre (GB)
  • The University of Sydney (AU)
  • NIHR Birmingham Biomedical Research Centre (GB)
  • University of Birmingham (GB)
  • University of Toronto (CA)
  • University of North Carolina at Chapel Hill (US)
  • AstraZeneca (United States) (US)
  • United States Food and Drug Administration (US)
  • University Hospitals Bristol NHS Foundation Trust (GB)
  • European Organisation for Research and Treatment of Cancer (NL)
  • University of Leeds (GB)
  • Queen's University (CA)
  • Medicines and Healthcare products Regulatory Agency (GB)
  • Pfizer (United States) (US)
  • University of Warwick (GB)
  • Mayo Clinic (US)
  • McGill University (CA)
  • Patient-Centered Outcomes Research Institute (US)
  • Boehringer Ingelheim (Germany) (DE)
  • Daiichi Sankyo (Germany) (DE)
  • National Cancer Institute (US)
  • UCB Pharma (Belgium) (BE)
  • GlaxoSmithKline (United States) (US)
  • Merck & Co., Inc., Rahway, NJ, USA (United States) (US)
  • University of Washington (US)
  • Janssen (United Kingdom) (GB)
  • National Cancer Research Institute (GB)
  • Merck KGaA, Darmstadt (Germany) (DE)
  • University of California, Irvine (US)
Corresponding author
Calvert, Melanie
ORCID
Venue
BMJ Open
Publisher
BMJ
Published
2021-06
Volume
11
Issue
6
Pages
e045105
ISSN
2044-6055 (ISSN-L)
DOI
10.1136/bmjopen-2020-045105
PMC
PMC8246371
OpenAlex
W3174588197
Cited by
144
References
14
Citations by year
2026: 19 · 2025: 32 · 2024: 29 · 2023: 30 · 2022: 31 · 2021: 3
FWCI
0.67
Type
journal article
Language
en
Open access
Licence
Locations
  • BMJ Open (publishedVersion, cc-by-nc)
  • PubMed Central (publishedVersion, cc-by-nc)
  • PubMed (publishedVersion)
  • White Rose Research Online (University of Leeds, The University of Sheffield, University of York) (acceptedVersion)
  • University of Birmingham Research Portal (University of Birmingham) (acceptedVersion, cc-by-nc)
  • Warwick Research Archive Portal (University of Warwick) (acceptedVersion)
  • Lirias (submittedVersion, other-oa)
  • Carolina Digital Repository (University of North Carolina at Chapel Hill) (submittedVersion)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
  • eScholarship (California Digital Library) (submittedVersion)
  • eScholarship (California Digital Library) (submittedVersion, cc-by-nc)
  • UNC Libraries
Funders
  • National Institute for Health and Care Excellence
  • Macmillan Cancer Support (5592105)
  • University Hospitals Birmingham NHS Foundation Trust
  • National Institute for Health and Care Research (PDF-2016-09-009, NIHR200268, NIHR201334)
  • University of Birmingham
  • University of Bristol
  • Surgical Reconstruction and Microbiology Research Centre
  • National Institutes of Health
  • Medical Research Council (MR/K025643/1)
  • Health Technology Assessment Programme
  • Birmingham Biomedical Research Centre
  • National Institute for Health Research Applied Research Collaboration West
  • UCB Pharma
Topics
  • Social Sciences › Decision Sciences › Statistics, Probability and Uncertainty › Meta-analysis and systematic reviews
  • Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
  • Social Sciences › Sociology and Political Science › Delphi Technique in Research
Keywords
patient-reported outcomes, research waste, protocol transparency, protocol templates, patient-centered care, quality of life, shared decision-making
MeSH terms
Patient Reported Outcome Measures, Humans, Quality of Life, Research Design, Checklist, Research Report

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