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Research methods
Communication
SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials
  1. Melanie Calvert1,2,3,4,5,
  2. Madeleine King6,
  3. Rebecca Mercieca-Bebber7,
  4. Olalekan Aiyegbusi1,3,
  5. Derek Kyte1,
  6. Anita Slade1,4,
  7. An-Wen Chan8,
  8. E Basch9,
  9. Jill Bell10,
  10. Antonia Bennett11,
  11. Vishal Bhatnagar12,
  12. Jane Blazeby13,14,
  13. Andrew Bottomley15,
  14. Julia Brown16,
  15. Michael Brundage17,
  16. Lisa Campbell18,
  17. Joseph C Cappelleri19,
  18. Heather Draper20,
  19. Amylou C Dueck21,
  20. Carolyn Ells22,
  21. Lori Frank23,
  22. Robert M Golub24,
  23. Ingolf Griebsch25,
  24. Kirstie Haywood26,
  25. Amanda Hunn27,
  26. Bellinda King-Kallimanis12,
  27. Laura Martin28,
  28. Sandra Mitchell29,
  29. Thomas Morel30,
  30. Linda Nelson31,
  31. Josephine Norquist32,
  32. Daniel O'Connor18,
  33. Michael Palmer33,
  34. Donald Patrick34,
  35. Gary Price1,
  36. Antoine Regnault35,
  37. Ameeta Retzer1,
  38. Dennis Revicki36,
  39. Jane Scott37,
  40. Richard Stephens38,
  41. Grace Turner1,4,
  42. Antonia Valakas39,
  43. Galina Velikova40,
  44. Maria von Hildebrand1,
  45. Anita Walker1,
  46. Lari Wenzel41
  1. 1Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, UK
  2. 2Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, Birmingham, UK
  3. 3NIHR Biomedical Research Centre, Institute of Translational Medicine, Birmingham, UK
  4. 4NIHR Surgical Reconstruction and Microbiology Research Centre, Birmingham, UK
  5. 5NIHR Applied Research Collaboration West Midlands, University of Birmingham, Birmingham, UK
  6. 6Faculty of Science, School of Psychology, University of Sydney, Sydney, New South Wales, Australia
  7. 7Faculty of Medicine and Health, NHMRC Clinical Trials Centre, University of Sydney, Sydney, New South Wales, Australia
  8. 8Women’s College Research Institute, Department of Medicine, University of Toronto, Toronto, Ontario, Canada
  9. 9University of North Carolina, Chapel Hill, North Carolina, USA
  10. 10Oncology Digital Health, AstraZeneca, Gaithersburg, Maryland, USA
  11. 11Cancer Outcomes Research Program, University of North Carolina, Chapel Hill, North Carolina, USA
  12. 12US Food and Drug Administration, Silver Spring, Maryland, USA
  13. 13NIHR Bristol Biomedical Research Centre, University of Bristol, Bristol, UK
  14. 14Division of Surgery, Head and Neck, University Hospitals Bristol NHS Foundation Trust, Bristol, UK
  15. 15Department of Quality of Life, European Organisation for Research and Treatment of Cancer, Brussels, Belgium
  16. 16Clinical Trials Research Unit, University of Leeds, Leeds, UK
  17. 17Department of Oncology, Queen’s University Cancer Research Institute, Kingston, Ontario, Canada
  18. 18Medicines and Healthcare Products Regulatory Agency, London, UK
  19. 19Global Biometrics & Data Management—Statistics, Pfizer Inc, New York City, New York, USA
  20. 20University of Warwick, Coventry, UK
  21. 21Department of Health Sciences Research, Mayo Clinic, Scottsdale, Arizona, USA
  22. 22School of Population and Global Health, McGill University, Montreal, Quebec, Canada
  23. 23Patient-Centered Outcomes Research Institute, Washington, DC, USA
  24. 24JAMA, Chicago, Illinois, USA
  25. 25Boehringer Ingelheim International GmbH, Ingelheim, Germany
  26. 26Warwick Research in Nursing, University of Warwick, Warwick Medical School, Coventry, UK
  27. 27Independent Advisor, Manchester, UK
  28. 28Daiichi Sankyo Europe GmbH, Munchen, Germany
  29. 29National Cancer Institute, Rockville, Maryland, USA
  30. 30Patient-Centered Outcomes Research, UCB Pharma, Brussels, Belgium
  31. 31Value Evidence and Outcomes—Patient Centered Outcomes, GSK, Collegeville, Pennsylvania, USA
  32. 32Center for Observational Real-world Evidence (CORE), Patient-Centered Endpoints & Strategy, Merck & Co Inc, Kenilworth, New Jersey, USA
  33. 33Cancer Research Institute, Queen's University, Kingston, Ontario, Canada
  34. 34Department of Health Services, University of Washington, Seattle, Washington, USA
  35. 35Modus Outcomes, Lyon, France
  36. 36Outcomes Research Consulting, Sarasota, Florida, USA
  37. 37Johnson and Johnson, Janssen Global Services LLC, High Wycombe, UK
  38. 38National Cancer Research Institute Consumer Forum, London, UK
  39. 39EMD Serono Inc, Healthcare Business of Merck KGaA, Darmstadt, Germany
  40. 40Leeds Institute of Medical Research at St James’s, University of Leeds, Leeds, UK
  41. 41University of California, Irvine, California, USA
  1. Correspondence to Professor Melanie Calvert; M.Calvert{at}bham.ac.uk

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.

  • statistics & research methods
  • education & training (see medical education & training)
  • protocols & guidelines
  • clinical trials
http://creativecommons.org/licenses/by-nc/4.0/

This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, appropriate credit is given, any changes made indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.

https://doi.org/10.1136/bmjopen-2020-045105

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    • Contributors MC and MK had full access to all of the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. MC and MK are cochairs of the SPIRIT-PRO Group. Concept and design: MC, MK. Acquisition, analysis or interpretation of data: MC, MK, RM-B, OA, DK, AS, A-WC, EB, JBe, ABe, VB, JBl, ABo, JBr, MB, LC, JCC, HD, ACD, CE, LF, RMG, IG, KH, BK-K, LM, SM, TM, LN, JN, DO’C, MP, DP, GP, AReg, ARet, DR, JS, RS, GT, AV, GV, MvH, AW, LW. Drafting of the manuscript: MC, DK, RM-B, AS, A-WC, MK+section writers GV, AReg, DR, ABe, SM, LW, MP, JBr, GT, ARet, AW. Critical revision of the manuscript for important intellectual content: MC, MK, RM-B, OA, DK, AS, A-WC, EB, JBe, ABe, VB, JBl, ABo, JBr, MB, LC, JCC, HD, ACD, CE, LF, RMG, IG, KH, BK-K, LM, SM, TM, LN, JN, DO’C, MP, DP, GP, AReg, ARet, DR, JS, RS, GT, AV, GV, MvH, AW, LW. Obtained funding: MC, DK, MK, Doug Altman, JBl, JBr, MB, Joanna Coast, HD, MvH, J Ives, RM-B, GP, L Roberts, AS. Supervision: MC.

    • Funding This SPIRIT-PRO Extension was funded by Macmillan Cancer Support (grant 5592105) and the University of Birmingham and was sponsored by the University of Birmingham. Development of the PRO protocol template was funded by an unrestricted educational research grant from UCB Pharma. MC receives funding from the National Institute for Health Research (NIHR) Birmingham Biomedical Research Centre, the NIHR Surgical Reconstruction and Microbiology Research Centre and NIHR ARC West Midlands at the at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, Health Data Research UK, Innovate UK (part of UK Research and Innovation), Macmillan Cancer Support, UCB Pharma. MK is supported by the Australian government through Cancer Australia. RM-B is supported by the Australian Government by a National Health and Medical Research Council (NHMRC) research fellowship. JBl is supported by the NIHR Biomedical Research Centre at the University Hospitals Bristol NHS Foundation Trust. She is also an NIHR Senior Investigator. Work was funded by an unrestricted grant from UCB Pharma, and a participant (TM) contributed as a coauthor and member of the industry advisory group.

    • Disclaimer The study funders/sponsors had no role in the design and conduct of the study; collection, management, analysis and interpretation of the data; preparation, review or approval of the manuscript or decision to submit the manuscript for publication.

    • Competing interests All authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest and declare: SPIRIT-PRO group members were reimbursed for travel/subsistence at the consensus meeting. MC is Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for the Centre for Patient Reported Outcomes Research and is a National Institute for Health Research (NIHR) Senior Investigator. MC has received personal fees from Astellas, Takeda, Merck, Daiichi Sankyo, Glaukos, GSK and the Patient-Centred Outcomes Research Institute (PCORI) outside the submitted work. RM-B reports non-financial support from University of Birmingham. OA, DK and ARet report grants from NIHR. OA and DK report grants from Birmingham Biomedical Research Centre (BRC). OA reports grants from UCB Pharma and also receives funding from the Health Foundation and declares personal fees from Gilead Sciences Ltd. DK and ARet report grants from Innovate UK and Macmillan Cancer Support. DK reports grants from Kidney Research UK, NIHR SRMRC at the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, personal fees from Merck, GSK. EB declares personal fees from Navigating Cancer, Sivan Healthcare, CareVive Systems and AstraZeneca. Bell reports other from AstraZeneca, is an employee with stock ownership and/or stock options in the company. JCC reports other from Pfizer Inc., and is an employee and a stockholder of Pfizer Inc. IG is a fully paid employee of Boehringer Ingelheim International GmbH. CE is Chair of the Government of Canada Interagency Advisory Panel on Research Ethics. LM reports non-financial support from Daiichi-Sankyo and Cell & Gene Therapy Catapult. Morel reports other from UCB. LN reports other from GlaxoSmithKline, including employment and ownership of stock in GSK. RS reports personal fees from BioMed Central and Pfizer, other from NHS England, NHSx, NDC, NCRI, NIHR, MRC CTU, GeL, Glasgow CTU, UCLH, LSHTM, Cancer Research UK, Macmillan, Warwick University, Warwick CTU and University of Birmingham. AW reports grants from NIHR and Innovate UK. All other authors have completed the ICMJE uniform disclosure form and declare no support from any organisation for the submitted work; no financial relationships with any organisations that might have an interest in the submitted work in the previous 3 years, no other relationships or activities that could appear to have influenced the submitted work.

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.

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