Investigating the Extent to Which Patients Should Control Access to Patient Records for Research: A Deliberative Process Using Citizens� Juries

BackgroundThe secondary use of health data for research raises complex questions of privacy and governance. Such questions are ill-suited to opinion polling where citizens must choose quickly between multiple-choice answers based on little information.ObjectiveThe aim of this project was to extend knowledge about what control informed citizens would seek over the use of health records for research after participating in a deliberative process using citizens' juries.MethodsTwo 3-day citizens' juries, of 17 citizens each, were convened to reflect UK national demographics from 355 eligible applicants. Each jury addressed the mission "To what extent should patients control access to patient records for secondary use?" Jurors heard from and questioned 5 expert witnesses (chosen either to inform the jury, or to argue for and against the secondary use of data), interspersed with structured opportunities to deliberate among themselves, including discussion and role-play. Jurors voted on a series of questions associated with the jury mission, giving their rationale. Individual views were polled using questionnaires at the beginning and at end of the process.ResultsAt the end of the process, 33 out of 34 jurors voted in support of the secondary use of data for research, with 24 wanting individuals to be able to opt out, 6 favoring opt in, and 3 voting that all records should be available without any consent process. When considering who should get access to data, both juries had very similar rationales. Both thought that public benefit was a key justification for access. Jury 1 was more strongly supportive of sharing patient records for public benefit, whereas jury 2 was more cautious and sought to give patients more control. Many jurors changed their opinion about who should get access to health records: 17 people became more willing to support wider information sharing of health data for public benefit, whereas 2 moved toward more patient control over patient records.ConclusionsThe findings highlight that, when informed of both risks and opportunities associated with data sharing, citizens believe an individual's right to privacy should not prevent research that can benefit the general public. The juries also concluded that patients should be notified of any such scheme and have the right to opt out if they so choose. Many jurors changed their minds about this complex policy question when they became more informed. Many, but not all, jurors became less skeptical about health data sharing, as they became better informed of its benefits and risks

Investigating the Extent to Which Patients Should Control Access to Patient Records for Research: A Deliberative Process Using Citizens� Juries (Preprint)

BACKGROUND The secondary use of health data for research raises complex questions of privacy and governance. Such questions are ill-suited to opinion polling where citizens must choose quickly between multiple-choice answers based on little information. OBJECTIVE The aim of this project was to extend knowledge about what control informed citizens would seek over the use of health records for research after participating in a deliberative process using citizens’ juries. METHODS Two 3-day citizens’ juries, of 17 citizens each, were convened to reflect UK national demographics from 355 eligible applicants. Each jury addressed the mission “To what extent should patients control access to patient records for secondary use?” Jurors heard from and questioned 5 expert witnesses (chosen either to inform the jury, or to argue for and against the secondary use of data), interspersed with structured opportunities to deliberate among themselves, including discussion and role-play. Jurors voted on a series of questions associated with the jury mission, giving their rationale. Individual views were polled using questionnaires at the beginning and at end of the process. RESULTS At the end of the process, 33 out of 34 jurors voted in support of the secondary use of data for research, with 24 wanting individuals to be able to opt out, 6 favoring opt in, and 3 voting that all records should be available without any consent process. When considering who should get access to data, both juries had very similar rationales. Both thought that public benefit was a key justification for access. Jury 1 was more strongly supportive of sharing patient records for public benefit, whereas jury 2 was more cautious and sought to give patients more control. Many jurors changed their opinion about who should get access to health records: 17 people became more willing to support wider information sharing of health data for public benefit, whereas 2 moved toward more patient control over patient records. CONCLUSIONS The findings highlight that, when informed of both risks and opportunities associated with data sharing, citizens believe an individual’s right to privacy should not prevent research that can benefit the general public. The juries also concluded that patients should be notified of any such scheme and have the right to opt out if they so choose. Many jurors changed their minds about this complex policy question when they became more informed. Many, but not all, jurors became less skeptical about health data sharing, as they became better informed of its benefits and risks. </sec

The need for ethical guidance for the use of patient-reported outcomes (PROs) in 2 research and clinical practice

To the Editor—Patient-reported outcomes (PROs) are increasingly being used in clinical research to provide evidence of the benefits and risk of therapy from a patient perspective. PRO data from clinical trials can inform regulatory approvals and drug labeling, clinical guideline development and health policy1. Approximately one third of clinical trials include PROs collected through the use of patient questionnaires2. Beyond trials, PRO data are also increasingly captured in observational research and routine clinical care to provide information on the burden of disease and real-world evidence of treatment safety and effectiveness3, for audit and benchmarking1, and to monitor the status of patients and provide timely care tailored to individual needs. For instance, a study demonstrated that systematic web-based collection of information on symptoms led to improved health-related quality of life, survival and quality-adjusted survival, and fewer visits to the emergency room and hospitalization, among patients receiving chemotherapy for advanced solid tumors4. Patients value PRO trial results, as they can enhance clinician–patient communication about treatment options, which helps patients to feel more empowered in shared decision-making around their care5

The ethics of ‘Trials within Cohorts’ (TwiCs): 2nd international symposium - London, UK. 7-8 November 2016

On 7-8 th November 2016, 60 people with an interest in the ‘ Trials within Cohorts ’ (TwiCs) approach for randomised controlled trial design met in London. The purpose of this 2 nd TwiCs international symposium was to share perspectives and experiences on ethical aspects of the TwiCs design, discuss how TwiCs relate to the current ethical frame- work, provide a forum in which to discuss and debate ethical issues and identify future directions for conceptual and empirical research. The symposium was supported by the Wellcome Trust and the NIHR CLAHRC Yorkshire and Humber and organised by members of the TwiCs network led by Clare Relton and attended by people from the UK, the Netherlands, Norway, Canada and USA. The two-day sympo- sium enabled an international group to meet and share experiences of the TwiCs design (also known as the ‘ cohort multiple RCT design ’ ), and to discuss plans for future research. Over the two days, invited plenary talks were interspersed by discussions, posters and mini pre- sentations from bioethicists, triallists and health research regulators. Key findings of the symposium were: (1) It is possible to make a compelling case to ethics committees that TwiCs designs are ap- propriate and ethical; (2) The importance of wider considerations around the ethics of inefficient trial designs; and (3) some questions about the ethical requirements for content and timing of informed consent for a study using the TwiCs design need to be decided on a case-by-case basis

Benchmarking of Online Information Literacy Tutorials to Identify Lessons Learnt and Best Practice.

This paper reports the process and findings of a benchmarking exercise conducted on 10 online information literacy tutorials from around the English speaking World. The benchmarking exercise comprised site visits and the completion of an online survey. The aim of the exercise was to establish best practice and to gather any lessons that could be learnt, with the overall purpose of determining ways that our information literacy tutorial could provide the best quality and performance through the identification of user requirements. The method of selecting the tutorials to benchmark against and the areas to include in the review is described. A summary of results from each of the benchmarked areas is included for: pedagogical issues; design, development and user feedback; content; interactivity; and look and feel

Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial

Background: Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies. Methods: An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions. ORBCEL-C™ is an enriched Mesenchymal Stromal Cells product isolated from human umbilical cord tissue using CD362+ cell selection. Usability testing sessions were conducted using cognitive interviews and the ‘Think Aloud’ method with patient advisory group members and Research Nurses to assess the usability of the system. Results: Nine patient partners and seven research nurses took part in one usability testing session. Measures of fatigue and health-related quality of life, the PRO-CTCAE™ and FACT-GP5 global tolerability question were included in the ePRO system. Alert notifications to the clinical team were triggered by PRO-CTCAE™ and FACT-GP5 scores. Patient participants liked the simplicity and responsiveness of the patient-facing app. Two patients were unable to complete the testing session, due to technical issues. Research Nurses suggested minor modifications to improve functionality and the layout of the clinician dashboard and the training materials. Conclusion: By testing the effectiveness, efficiency, and satisfaction of our novel ePRO system (PROmicsR), we learnt that most people with an inflammatory condition found it easy to report their symptoms using an app on their own device. Their experiences using the PROmicsR ePRO system within a trial environment will be further explored in our upcoming feasibility testing. Research nurses were also positive and found the clinical dashboard easy-to-use. Using ePROs in early phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence based, and inform methodology development. Trial registration: ISRCTN, ISRCTN80103507. Registered 01 April 2022, https://www.isrctn.com/ISRCTN8010350

Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension

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