Melanie Calvert1, Derek Kyte1, Rebecca Mercieca-Bebber2, Anita Slade1, An-Wen Chan7, Madeleine T King2, Amanda Hunn4, Andrew Bottomley5, Antoine Regnault6, An-Wen Chan7, Carolyn Ells8, Daniel O'Connor9, Dennis Revicki10, Donald Patrick11, Doug Altman12, Ethan Basch13, Galina Velikova14, Gary Price15, Heather Draper16, Jane Blazeby17, Jane Scott18, Joanna Coast17, Josephine Norquist19, Julia Brown14, Kirstie Haywood16, Laura Lee Johnson20, Lisa Campbell9, Lori Frank21, Maria von Hildebrand15, Michael Brundage22, Michael Palmer22, Paul Kluetz20, Richard Stephens23, Robert M Golub24, Sandra Mitchell25, Trish Groves26. 1. Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, England. 2. Sydney Medical School, Faculty of Medicine and School of Psychology, Faculty of Science, University of Sydney, Sydney, Australia. 3. Women's College Research Institute, Department of Medicine, University of Toronto, Toronto, Ontario, Canada. 4. Health Research Authority, London, England. 5. European Organization for Research and Treatment of Cancer, Brussels, Belgium. 6. Modus Outcomes, Lyon, France. 7. University of Toronto, Toronto, Ontario, Canada. 8. Panel on Research Ethics, Toronto, Ontario, Canada. 9. Medicines and Healthcare Products Regulatory Agency, London, England. 10. Evidera, Bethesda, Maryland. 11. University of Washington, Seattle. 12. University of Oxford, Oxford, England. 13. University of North Carolina, Chapel Hill. 14. University of Leeds, Leeds, England. 15. Patient Partner, Centre for Patient Reported Outcome Research, Institute of Applied Health Research, University of Birmingham, Birmingham, England. 16. University of Warwick, Coventry, England. 17. University of Bristol, Bristol, England. 18. Janssen Global Services, Johnson and Johnson, High Wycombe, England. 19. Merck Sharp & Dohme Corporation, Whitehouse Station, New Jersey. 20. US Food and Drug Administration, Silver Spring, Maryland. 21. Patient-Centered Outcomes Research Institute, Washington, DC. 22. Queen's University, Kingston, Ontario, Canada. 23. National Cancer Research Institute Consumer Forum, London, England. 24. The JAMA Network, Chicago, Illinois. 25. National Cancer Institute, Rockville, Maryland. 26. , London, England.
Abstract
Importance: Patient-reported outcome (PRO) data from clinical trials can provide valuable evidence to inform shared decision making, labeling claims, clinical guidelines, and health policy; however, the PRO content of clinical trial protocols is often suboptimal. The SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) statement was published in 2013 and aims to improve the completeness of trial protocols by providing evidence-based recommendations for the minimum set of items to be addressed, but it does not provide PRO-specific guidance. Objective: To develop international, consensus-based, PRO-specific protocol guidance (the SPIRIT-PRO Extension). Design, Setting, and Participants: The SPIRIT-PRO Extension was developed following the Enhancing Quality and Transparency of Health Research (EQUATOR) Network's methodological framework for guideline development. This included (1) a systematic review of existing PRO-specific protocol guidance to generate a list of potential PRO-specific protocol items (published in 2014); (2) refinements to the list and removal of duplicate items by the International Society for Quality of Life Research (ISOQOL) Protocol Checklist Taskforce; (3) an international stakeholder survey of clinical trial research personnel, PRO methodologists, health economists, psychometricians, patient advocates, funders, industry representatives, journal editors, policy makers, ethicists, and researchers responsible for evidence synthesis (distributed by 38 international partner organizations in October 2016); (4) an international Delphi exercise (n = 137 invited; October 2016 to February 2017); and (5) consensus meeting (n = 30 invited; May 2017). Prior to voting, consensus meeting participants were informed of the results of the Delphi exercise and given data from structured reviews evaluating the PRO protocol content of 3 defined samples of trial protocols. Results: The systematic review identified 162 PRO-specific protocol recommendations from 54 sources. The ISOQOL Taskforce (n = 21) reduced this to 56 items, which were considered by 138 international stakeholder survey participants and 99 Delphi panelists. The final wording of the SPIRIT-PRO Extension was agreed on at a consensus meeting (n = 29 participants) and reviewed by external group of experts during a consultation period. Eleven extensions and 5 elaborations to the SPIRIT 2013 checklist were recommended for inclusion in clinical trial protocols in which PROs are a primary or key secondary outcome. Extension items focused on PRO-specific issues relating to the trial rationale, objectives, eligibility criteria, concepts used to evaluate the intervention, time points for assessment, PRO instrument selection and measurement properties, data collection plan, translation to other languages, proxy completion, strategies to minimize missing data, and whether PRO data will be monitored during the study to inform clinical care. Conclusions and Relevance: The SPIRIT-PRO guidelines provide recommendations for items that should be addressed and included in clinical trial protocols in which PROs are a primary or key secondary outcome. Improved design of clinical trials including PROs could help ensure high-quality data that may inform patient-centered care.
Importance: Patient-reported outcome (PRO) data from clinical trials can provide valuable evidence to inform shared decision making, labeling claims, clinical guidelines, and health policy; however, the PRO content of clinical trial protocols is often suboptimal. The SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) statement was published in 2013 and aims to improve the completeness of trial protocols by providing evidence-based recommendations for the minimum set of items to be addressed, but it does not provide PRO-specific guidance. Objective: To develop international, consensus-based, PRO-specific protocol guidance (the SPIRIT-PRO Extension). Design, Setting, and Participants: The SPIRIT-PRO Extension was developed following the Enhancing Quality and Transparency of Health Research (EQUATOR) Network's methodological framework for guideline development. This included (1) a systematic review of existing PRO-specific protocol guidance to generate a list of potential PRO-specific protocol items (published in 2014); (2) refinements to the list and removal of duplicate items by the International Society for Quality of Life Research (ISOQOL) Protocol Checklist Taskforce; (3) an international stakeholder survey of clinical trial research personnel, PRO methodologists, health economists, psychometricians, patient advocates, funders, industry representatives, journal editors, policy makers, ethicists, and researchers responsible for evidence synthesis (distributed by 38 international partner organizations in October 2016); (4) an international Delphi exercise (n = 137 invited; October 2016 to February 2017); and (5) consensus meeting (n = 30 invited; May 2017). Prior to voting, consensus meeting participants were informed of the results of the Delphi exercise and given data from structured reviews evaluating the PRO protocol content of 3 defined samples of trial protocols. Results: The systematic review identified 162 PRO-specific protocol recommendations from 54 sources. The ISOQOL Taskforce (n = 21) reduced this to 56 items, which were considered by 138 international stakeholder survey participants and 99 Delphi panelists. The final wording of the SPIRIT-PRO Extension was agreed on at a consensus meeting (n = 29 participants) and reviewed by external group of experts during a consultation period. Eleven extensions and 5 elaborations to the SPIRIT 2013 checklist were recommended for inclusion in clinical trial protocols in which PROs are a primary or key secondary outcome. Extension items focused on PRO-specific issues relating to the trial rationale, objectives, eligibility criteria, concepts used to evaluate the intervention, time points for assessment, PRO instrument selection and measurement properties, data collection plan, translation to other languages, proxy completion, strategies to minimize missing data, and whether PRO data will be monitored during the study to inform clinical care. Conclusions and Relevance: The SPIRIT-PRO guidelines provide recommendations for items that should be addressed and included in clinical trial protocols in which PROs are a primary or key secondary outcome. Improved design of clinical trials including PROs could help ensure high-quality data that may inform patient-centered care.
Authors: Goldy C George; Tito R Mendoza; Eucharia C Iwuanyanwu; Meryna Manandhar; Solmaz F Afshar; Sarina A Piha-Paul; Apostolia Tsimberidou; Aung Naing; Charles S Cleeland; David S Hong Journal: Invest New Drugs Date: 2019-11-25 Impact factor: 3.850
Authors: Gita Thanarajasingam; John P Leonard; Thomas E Witzig; Thomas M Habermann; Kristie A Blum; Nancy L Bartlett; Christopher R Flowers; Brandelyn N Pitcher; Sin-Ho Jung; Pamela J Atherton; Angelina Tan; Paul J Novotny; Amylou C Dueck Journal: Lancet Haematol Date: 2020-06 Impact factor: 18.959
Authors: Benjamin A Steinberg; Jeffrey Turner; Ann Lyons; Joshua Biber; Mihail G Chelu; James C Fang; Roger A Freedman; Frederick T Han; Benjamin Hardisty; Nassir F Marrouche; Ravi Ranjan; Rashmee U Shah; John A Spertus; Josef Stehlik; Brian Zenger; Jonathan P Piccini; Rachel Hess Journal: Europace Date: 2020-03-01 Impact factor: 5.214
Authors: David M Condon; Robert Chapman; Sara Shaunfield; Michael A Kallen; Jennifer L Beaumont; Daniel Eek; Debanjali Mitra; Katy L Benjamin; Kelly McQuarrie; Jamae Liu; James W Shaw; Allison Martin Nguyen; Karen Keating; David Cella Journal: Qual Life Res Date: 2019-11-07 Impact factor: 4.147