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Abstract
Objectives (a) To adapt the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-patient-reported outcome (PRO) Extension guidance to a user-friendly format for patient partners and (b) to codesign a web-based tool to support the dissemination and uptake of the SPIRIT-PRO Extension by patient partners.
Design A 1-day patient and public involvement session.
Participants Seven patient partners.
Methods A patient partner produced an initial lay summary of the SPIRIT-PRO guideline and a glossary. We held a 1-day PPI session in November 2019 at the University of Birmingham. Five patient partners discussed the draft lay summary, agreed on the final wording, codesigned and agreed the final content for both tools. Two additional patient partners were involved in writing the manuscript. The study compiled with INVOLVE guidelines and was reported according to the Guidance for Reporting Involvement of Patients and the Public 2 checklist.
Results Two user-friendly tools were developed to help patients and members of the public be involved in the codesign of clinical trials collecting PROs. The first tool presents a lay version of the SPIRIT-PRO Extension guidance. The second depicts the most relevant points, identified by the patient partners, of the guidance through an interactive flow diagram.
Conclusions These tools have the potential to support the involvement of patient partners in making informed contributions to the development of PRO aspects of clinical trial protocols, in accordance with the SPIRIT-PRO Extension guidelines. The involvement of patient partners ensured the tools focused on issues most relevant to them.
- protocols & guidelines
- quality in health care
- qualitative research
Data availability statement
Data sharing not applicable as no datasets generated and/or analysed for this study. All data relevant to the study are included in the article or uploaded as supplemental information.
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/.
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Strengths and limitations of this study
Two user-friendly tools were codeveloped with patient and public involvement (PPI) partners for the use of patient partners involved in the codesign of clinical trials collecting patient-reported outcomes.
The research was reported according to Guidance for Reporting Involvement of Patients and the Public 2 checklist and adhered to INVOLVE recommendations.
The user-friendly tools were not tested among a wider patient partner group.
In addition, the PPI partners included in the codevelopment of the tools were mainly oncology patients.
Introduction
Patient-reported outcomes (PROs) provide information about the status of a patient’s health, directly from the patient, without interpretation by a clinician.1 PROs are collected in clinical trials to provide evidence of the impact of disease treatment on functional health, well-being, severity of symptoms or side effects, and psychological impact of the disease and/or the treatment.2
Clinical trials are medical research studies carried out to determine the activity, safety, efficacy, effectiveness and adverse effects of diagnostic and therapeutic interventions.3 Clinical trial protocols describe the objective(s), design, procedures and statistical considerations needed to conduct a specific clinical trial. Recent research suggests important PRO protocol-items, such as hypotheses, data collection methods and statistical plans are often missing from trial protocols.4–7 Furthermore, rates of avoidable missing PRO data are often high4 5 8 and PRO data publications are reported long after other outcomes or not at all9 10; if reported, the PRO reporting is often inadequate.7–9 11–14
A recent review of 228 National Institute of Health Research Cancer portfolio studies identified that PRO data were left unreported for studies involving nearly 50 000 patients, which is unacceptable and unethical.9 Moreover, such failures and omissions compromise the impact of PROs on future patient care and health policy, and also waste valuable resources in terms of patient and researcher time and funding.
In 2018, the SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials)-PRO Extension was published with the aim to provide recommendations for researchers on which items should be addressed in clinical trial protocols with primary or key secondary PRO endpoints. However, there is a lack of training materials and tools to support the uptake of the SPIRIT-PRO guidance to promote quality and to simplify the approach for patient partners who are involved in the review and codesign of clinical trials with PRO objectives.15 The aim of this research was to: (a) adapt the SPIRIT-PRO Extension guidance to a user-friendly format for patient partners and (b) codesign a web-based tool to support the dissemination and uptake of the SPIRIT-PRO Extension by patient partners.
Methods
A patient partner (GP) produced an initial lay summary of the SPIRIT-PRO guideline and drafted a glossary with support from academic coauthors (MC and SCR). The patient partner selected to produce the initial lay summary and glossary was originally involved in the development of the SPIRIT-PRO Extension guideline. In addition, the patient partner has experienced completing PRO questionnaires and has been involved in different PRO-specific projects to provide his perspective from a patient’s perspective.
A 1-day PPI (patient and public involvement) session was held with patient partners in November 2019 at the University of Birmingham, UK. The aim of the PPI session was to adapt the SPIRIT-PRO Extension guidance to a user-friendly format for patient partners, and codesign a tool to aid patient partners in the codesign of PRO clinical trials. The PPI session was conducted and reported according to the Guidance for Reporting Involvement of Patients and the Public (GRIPP) 2 reporting checklists. This international provides guidance on the key reporting items for reporting PPI in health and social care research.16 In addition, the PPI session complied with the INVOLVE guideline, a government supported programme that promotes active public involvement in National Health Service, public health and social care research.17
Patient and public involvement
Seven PPI partners who were already known to the team, who had relevant experience in clinical trials, were recruited by the research team to assist at different stages in the development of the tools. The PPI partners were six patients and one carer with personal experience of different health conditions including oncology (four PPI partners), Parkinson’s (one PPI partner) and chronic kidney disease (one PPI partner). Six PPI partners identified themselves as white and one as Sikh British. Only three of the PPI partners were previously involved as trial participants. One partner was involved in the development of the first version of the patient-friendly SPIRIT-PRO guidance. Five were involved in the codesign of the patient-friendly SPIRIT-PRO tools, and all seven contributed to writing this manuscript.
During the session, five PPI partners (GP/LR/LG/RV/PE) and two academics (MC and SCR) discussed the original SPIRIT-PRO Extension guideline and contrasted it with the initial lay summary drafted. PPI partners commented on the comprehension and refined and agreed the wording and clarity of the lay version of the SPIRIT-PRO guideline and glossary (figure 1). Following the PPI session, attendees commented on the wording and agreed on the penultimate version of the user-friendly SPIRIT-PRO Extension content. Broader feedback on final guidance was sought from two additional patient partners (RW/RS).
User-friendly SPIRIT-PRO Extension and glossary methods. PPI, patient and public involvement; PRO, patient-reported outcome; SPIRIT, Standard Protocol Items: Recommendations for Interventional Trials.
During the PPI session, patient partners discussed the design and content of a previously published diagram (PRO learn resource for patient advocates involved in coproduction of research or review, online supplemental appendix 1) on the PRO considerations for PPI partners in the design and review of trials collecting PROs.18 PPI partners highlighted key SPIRIT-PRO items and additional information that should be incorporated in the published diagram. These changes led to the development of the web-tool.
Supplemental material
Results
Seven PPI partners were involved in the codesign of two tools to promote the uptake and dissemination of the SPIRIT-PRO Extension guidance by patient partners involved in the codevelopment of clinical trials. PPI partners highlighted specific priorities and preferred formats. In addition, PPI partners contributed to the writing up of the discussion section and in particular around the benefits of the development of these tools.
User-friendly version of the SPIRIT-PRO Extension guidance
This tool was developed to adapt the SPIRIT-PRO Extension guidance to a user-friendly format for patient partners. The user-friendly tool (table 1) presents five different key items for PPI partners to consider while involved in the codesign and/or review of trials collecting PROs: (a) SPIRIT-PRO item number and description; (b) questions for PPI partner(s) to consider; (c) key considerations for PPI partner(s); (d) considerations for the lay summary and (e) considerations for the participant information sheet and consent form. A glossary (online supplemental appendix 2) was also codeveloped to aid PPI partners in the implementation of the user-friendly tool.
Supplemental material
User-friendly version of the SPIRIT-PRO Extension guidance
Web-based tool
The web-based tool, presented in concertina style, illustrates the main key items PPI partners considered most relevant from the user-friendly SPIRIT-PRO Extension version. The web-tool aimed at supporting the dissemination and uptake of the SPIRIT-PRO Extension by patient partners, provides PPI partners with six general PRO-specific questions to facilitate their role as codesigners and interaction with the trial team. PPI partners are not expected to answer these questions but to raise these questions with the research team while codeveloping the clinical trial.
The main six SPIRIT-PRO items included were: (a) does the team have a clear reason for assessing PROs in the trial? And has the team clearly stated the purpose of the research? (b) which questionnaire(s) are they considering using? (c) are there any reasons why a patient might not be able to complete the PRO questionnaire? (d) how often, when and where will patients be asked to complete the questionnaire(s)? (e) what languages are the chosen questionnaire(s) available in? and (f) how will the team ensure that they collect high quality data that can meaningfully inform future patient care? The diagram provides further detail to each question to help PPI partners ask more in depth questions and better understand the importance of capturing PROs in trials. In addition, the web-tool includes ‘other considerations’ and ‘other resources’ for PPI partners to facilitate their understanding and participation in the design of the trial. For instance, ‘other considerations’ includes key elements that should be covered in the participant information sheet for potential trial participants. ‘Other resources’ include web resources such as ePROVIDE and GRIPP 2 checklist.19 The webtool is available from the Centre for Patient Reported Outcomes Research website.20 Figure 2 presents an overview of the codeveloped web-tool.
Web-tool for patient advocates involved in coproduction of PRO research or review. PRO, patient-reported outcome.
Discussion
Two user-friendly tools were codesigned with the assistance of seven patient partners to assist PPI partners involved in the design or review of clinical trials and provide informed, patient-centred input into development of PRO aspects of clinical trial protocols. PPI in this research was essential to ensure that the tools were comprehensive and user friendly for PPI partners. In addition, it was essential to enhance the dissemination and uptake of the SPIRIT-PRO Extension guidance.
The involvement of PPI partners helped ensure that the tools focused on issues that matter most to them. PPI should go beyond involvement; it should be a platform for patients to influence, design processes, identify relevant content and to make decisions significant for and acceptable to end users.21 22 PPI partners raised important concerns related to the completion of PRO questionnaires such as: time needed to complete the PRO questionnaire(s) and frequency patients need to complete the questionnaire(s). Although these are covered by the SPIRIT-PRO Extension guidance, they were included in the patient information sheet section under the ‘other resources’ section.
Patients have recently advocated against regulatory agencies for approving oncology drugs based on surrogate endpoints rather than the value they add to patients’ lives.23 24 In addition, patients frequently do not completely understand their diagnostics and are not aware of the side effects of the interventions, as they are occasionally not effectively communicated by healthcare professionals.24 Therefore, patient and public awareness and their involvement can help tackle these issues.23 24 Currently, PRO stakeholders are making concerted efforts to incorporate the patients’ experience into the drug development process, which has the potential to better inform shared decision-making.25 For instance, the Food and Drug Administration is patient-focused drug development guidance to address how stakeholders can collect and include PROs from patients and caregivers in the development and regulation of medical products.26 In 2016, the European Medicine Agency published Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. Appendix 2 describes the use of PRO endpoints in oncology studies and the value of PRO data from the regulatory perspective.27
PROs carry the ‘voice’ of the patients; hence, trials collecting PROs should include patients and carers as codesigners to inform PRO measure development, selection, and implementation and ensure that PRO data are analysed and published.21 28 Thus, maximising the impact on future patient benefit and reducing research waste. The design of trials collecting PROs without patient input can be considered unreasonable and unacceptable.9 21 PPI partners should be empowered to be involved in the design of trials collecting PROs and their content, and make decisions by using the two different tools developed, while following the SPIRIT-PRO Extension guidance. The strengths of the research include the participation of seven PPI partners, who were selected with a range of levels of experience and exposure to trial development to ensure the outputs were well-informed, but also accessible for new patients and public. Adherence to GRIPP 2 guidance to report PPI involvement in research was a further strength of the study.16 The tools presented in this manuscript were developed to aid patient partners in the codevelopment or review of clinical trials collecting PROs. Nonetheless, these tools have the potential to be used in other types of clinical studies in which the participation of patients and carers is essential.
However, the tools developed were not tested among patient partners with less trial experience or less experience with research, which could have helped in the refinement of the tools. A further limitation is that two PPI partners involved in the codevelopment of the user-friendly version of the SPIRIT-PRO Extension guidance were involved in the development of the original guidance. This previous knowledge and understanding of the SPIRIT-PRO items might have influenced the selection of lay vocabulary. However, to tackle these four additional PPI partners were included to agree on the best wording of the guidance. Patient partners were involved in the same way in both research projects. However, patient partners drove the agenda more during the codevelopment of the tools for patients as the aim of the research was to develop tools for them to use. An additional limitation is that PPI partners’ perspectives may not be reflective of a larger patient population as the majority of the participants were oncology partners and only one carer was included.
In conclusion, the tools developed, if used appropriately, have the potential to facilitate the involvement of patient partners in providing informed input into the development of PRO aspects of clinical trial protocols, in accordance with the SPIRIT-PRO Extension guidelines.
Next steps
Feedback can be provided on the resource using an anonymised survey https://www.smartsurvey.co.uk/s/SPIRIT-PRO_Tools_for_patients/, which will help inform future developments. We encourage PPI partners and researchers involved in the design or review of trials collecting PROs to provide further feedback to the research team.
Data availability statement
Data sharing not applicable as no datasets generated and/or analysed for this study. All data relevant to the study are included in the article or uploaded as supplemental information.
Ethics statements
Ethics approval
Ethical approval for this study was gained from the University of Birmingham, UK (ERN_19-0939).
References
Supplementary materials
Supplementary Data
This web only file has been produced by the BMJ Publishing Group from an electronic file supplied by the author(s) and has not been edited for content.
Footnotes
Twitter @samsamcr
Contributors SCR lead the conceptualisation, data curation, formal analysis, investigation, methodology and writing of the original draft. RS critically reviewed and edited the manuscript. GP contributed to the conceptualisation of the manuscript and reviewed the draft. PE, LG, LR and RV contributed to the conceptualisation of the manuscript. RW, RM-B, CR, OLA and AS contributed to the conceptualisation and reviewed and edited the manuscript. MC acquired funding, lead the conceptualisation and reviewed and edited the manuscript.
Funding This work was funded by an unrestricted educational research grant from UCB Pharma. Award/Grant number is not applicable.
Competing interests MC and AS receive 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 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. The views expressed in this article are those of the author(s) and not necessarily those of the NIHR, or the Department of Health and Social Care. MC has received personal fees from Astellas, Takeda, Merck, Daiichi Sankyo, Glaukos, GlaxoSmithKline and the Patient-Centered Outcomes Research Institute (PCORI) outside the submitted work. RM-B is supported by the Australian Government by a National Health and Medical Research. OLA declares personal fees from Gilead Sciences Ltd and GlaxoSmithKline outside 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.