Importance: Patient-reported outcome (PRO) data from clinical trials can provide valuable evidence to inform shared-decision making, labelling 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 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: (i) a systematic review of existing PRO-specific protocol guidance to generate a list of candidate PRO-specific protocol items (published 2014); (ii) refinements to the list and removal of duplicate items by the International Society for Quality of Life Research (ISOQOL) PROtocol Checklist Taskforce; (iii) 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, October 2016); (iv) an international Delphi exercise (n=137 invited; October 2016 to February 2017)and 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 three 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 stakeholders and 99 Delphi panelists. The final wording of the SPIRIT-PRO Extension was agreed at a consensus meeting (n=29 participants) and reviewed by external stakeholders during a consultation period. Eleven extensions and five elaborations to the SPIRIT 2013 checklist are recommended for inclusion in clinical trial protocols where 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, timepoints for assessment, PRO instrument selection and measurement properties, data collection plan, translation to other languages, proxy completion, strategies to minimise missing data and whether PRO data will be monitored during the study to inform clinical care. Conclusions and relevance: These 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.
CORE (COnnecting REpositories)
CORE (COnnecting REpositories)