Priorities for methodological research on patient and public involvement in clinical trials: A modified Delphi process.

BACKGROUND: Despite increasing international interest, there is a lack of evidence about the most efficient, effective and acceptable ways to implement patient and public involvement (PPI) in clinical trials. OBJECTIVE: To identify the priorities of UK PPI stakeholders for methodological research to help resolve uncertainties about PPI in clinical trials. DESIGN: A modified Delphi process including a two round online survey and a stakeholder consensus meeting. PARTICIPANTS: In total, 237 people registered of whom 219 (92%) completed the first round. One hundred and eighty-seven of 219 (85%) completed the second; 25 stakeholders attended the consensus meeting. RESULTS: Round 1 of the survey comprised 36 topics; 42 topics were considered in round 2 and at the consensus meeting. Approximately 96% of meeting participants rated the top three topics as equally important. These were as follows: developing strong and productive working relationships between researchers and PPI contributors; exploring PPI practices in selecting trial outcomes of importance to patients; and a systematic review of PPI activity to improve the accessibility and usefulness of trial information (eg participant information sheets) for participants. CONCLUSIONS: The prioritized methodological research topics indicate important areas of uncertainty about PPI in trials. Addressing these uncertainties will be critical to enhancing PPI. Our findings should be used in the planning and funding of PPI in clinical trials to help focus research efforts and minimize waste.

Ensuring young voices are heard in core outcome set development: international workshops with 70 children and young people.

PLAIN ENGLISH SUMMARY: Researchers test treatments to ensure these work and are safe. They do this by studying the effects that treatments have on patients by measuring outcomes, such as pain and quality of life. Often research teams measure different outcomes even though each team is studying the same condition. This makes it hard to compare the findings from different studies and it can reduce the accuracy of the treatment advice available to patients. Increasingly, researchers are tackling this problem by developing 'core outcome sets'. These are lists of outcomes that all researchers working on a given condition should measure in their studies. It is important that patients have a voice in the development of core outcome sets and children and young people are no exception. But their voices have rarely been heard when core outcome sets are developed. Researchers are trying to address this problem and make sure that core outcome sets are developed in ways that are suitable for children and young people. As a first step, we held two international workshops with children and young people to listen to their views. They emphasised the importance of motivating young people to participate in developing core outcome sets, making them feel valued, and making the development process more interactive, enjoyable and convenient. We hope this commentary will encourage researchers to include children and young people when developing core outcome sets and to adapt their methods so these are suitable for young participants. Future research is important to examine whether these adaptations are effective. ABSTRACT: Background Different research teams looking at treatments for the same condition often select and measure inconsistent treatment outcomes. This makes it difficult to synthesise the results of different studies, leads to selective outcome reporting and impairs the quality of evidence about treatments. 'Core outcome sets' (COS) can help to address these problems. A COS is an agreed, minimum list of outcomes that researchers are encouraged to consistently measure and report in their studies. Including children and young people (CYP) as participants in the development of COS for paediatric conditions ensures that clinically meaningful outcomes are measured and reported. However, few published COS have included CYP as participants. COS developers have described difficulties in recruiting and retaining CYP and there is a lack of guidance on optimising COS methods for them. We aimed to explore CYP's views on the methods used to develop COS and identify ways to optimise these methods.Main body This commentary summarises discussions during two workshops with approximately 70 CYP (aged 10-18 years old) at the International Children's Advisory Network Research and Advocacy Summit, 2018. Delegates described what might motivate them to participate in a COS study, including feeling valued, understanding the need for COS and the importance of input from CYP in their development, and financial and other incentives (e.g. certificates of participation). For Delphi surveys, delegates suggested that lists of outcomes should be as brief as possible, and that scoring and feedback methods should be simplified. For consensus meetings, delegates advised preparing CYP in advance, supporting them during meetings (e.g. via mentors) and favoured arrangements whereby CYP could meet separately from parents and other stakeholders. Overall, they wanted COS methods that were convenient, enjoyable and engaging.Conclusion This commentary points to the limitations of the methods currently used to develop COS with CYP. It also points to ways to motivate CYP to participate in COS studies and to enhancements of methods to make participation more engaging for CYP. Pending much needed research on COS methods for CYP, the perspectives offered in the workshops should help teams developing COS in paediatrics and child health.

Outcomes for Pressure Ulcer Trials (OUTPUTs): protocol for the development of a core domain set for trials evaluating the clinical efficacy or effectiveness of pressure ulcer prevention interventions.

BACKGROUND: Core outcome sets (COS) are being developed in many clinical areas to increase the quality and comparability of clinical trial results as well as to ensure their relevance for patients. A COS represents an agreed standardized set of outcomes that describes the minimum that should be consistently reported in all clinical trials of a defined area. It comprises a core domain set (defining what core outcomes should be measured) and a core measurement set (defining measurement/assessment instruments for each core domain). For pressure ulcer prevention trials a COS is lacking. The great heterogeneity of reported outcomes in this field indicates the need for a COS. METHODS/DESIGN: The first part of this project aims to develop a core domain set by following established methods, which incorporates four steps: (1) definition of the scope, (2) conducting a scoping review, (3) organizing facilitated workshops with service users, (4) performing Delphi surveys and establishing consensus in a face-to-face meeting with different stakeholders. DISCUSSION: After achieving consensus on the core domain set, further work will be undertaken to determine a corresponding core measurement set. This will lead to better pressure ulcer prevention research in the future. There are a number of methodological challenges in the field of COS development. To meet these challenges and to ensure a high-quality COS, the OUTPUTS project affiliates to current standards and works in close collaboration with international experts and with existing international service user groups. TRIAL REGISTRATION: The OUTPUTs project is registered in the COMET database: ( http://www.comet-initiative.org/studies/details/283 ). Registered on 2015.

The COMET Handbook: version 1.0.

The selection of appropriate outcomes is crucial when designing clinical trials in order to compare the effects of different interventions directly. For the findings to influence policy and practice, the outcomes need to be relevant and important to key stakeholders including patients and the public, health care professionals and others making decisions about health care. It is now widely acknowledged that insufficient attention has been paid to the choice of outcomes measured in clinical trials. Researchers are increasingly addressing this issue through the development and use of a core outcome set, an agreed standardised collection of outcomes which should be measured and reported, as a minimum, in all trials for a specific clinical area.Accumulating work in this area has identified the need for guidance on the development, implementation, evaluation and updating of core outcome sets. This Handbook, developed by the COMET Initiative, brings together current thinking and methodological research regarding those issues. We recommend a four-step process to develop a core outcome set. The aim is to update the contents of the Handbook as further research is identified.

Including patients in core outcome set development: issues to consider based on three workshops with around 100 international delegates.

PLAIN ENGLISH SUMMARY: This commentary article describes three interactive workshops that explored how patients can contribute to decisions about what outcomes are measured in clinical trials across the world. Outcomes like quality of life, side-effects and pain are used in trials to measure whether a treatment is effective. Here, we outline how research groups are increasingly coming together to develop 'core outcomes sets' for particular conditions. Core outcome sets are lists of agreed outcomes. Their use will help in identifying which treatments are effective by enabling people to compare the findings of different clinical trials in the same condition. Currently, it is often very difficult to make these comparisons because different studies often measure different outcomes. Delegates attending the workshops included patients, clinicians and researchers. They discussed ways of making core outcome set development more meaningful and accessible for patients, and ensuring that they have a genuine say in the development process. This article summarises these discussions and concludes by identifying three distinctive challenges in securing patient input to core outcome set development: the process and objectives can seem far removed from the immediate concerns of patients, difficulties can arise in securing patient input on an international scale, and difficulties can also arise in bringing multiple stakeholder groups together to achieve consensus. While patient participation, involvement and engagement in core outcome set development can draw on lessons from other research areas, these distinctive challenges point to the need for distinctive solutions to enable meaningful patient input to core outcome set development. ABSTRACT: Background This article describes three workshops that explored how patients can contribute to decisions about what outcomes are measured in clinical trials. People need evidence about what treatments are best for particular health conditions. The strongest evidence comes from systematic reviews comparing outcomes across different studies of treatments for a particular condition. However, it is often difficult to do these comparisons because the different studies-even though they have all investigated the same condition-often measure different outcomes. To tackle this problem, research teams are increasingly coming together to develop core outcome sets (COS) for particular conditions or treatments. The goal is that across the world, all the research teams working on the same condition or treatment will then use the COS in their research.Main body We report on three interactive workshops that explored how patients and the public can contribute to decision making about what outcomes should be included in a COS. About 100 international delegates, including researchers, clinicians and patients, attended the workshops. The workshops were held in the United Kingdom, Italy and Canada as part of the COMET (Core Outcome Measures in Effectiveness Trials) Initiative annual meetings. Patients who had some experience as research advisors, collaborators, partners or co-ordinators facilitated the workshops together with a researcher. Notes made during each workshop informed the preparation of this article. Workshop discussion focussed on ways of making core outcome set development more meaningful and accessible for patients. Delegates wanted patients to have a genuine say, alongside other stakeholders, in what outcomes are included in COS. Delegates felt that key to ensuring this is recognising that patient participation in COS development alone is not enough, and that patients will also need to be involved in the design of COS development studies.Conclusion We conclude by pointing to some distinctive challenges in including patients in COS development. While the COS development community can draw on the lessons learnt from other research areas about patient participation, involvement and engagement, the distinctive challenges that arise in COS development point to the need for some distinctive solutions too.

A patient and public involvement (PPI) toolkit for meaningful and flexible involvement in clinical trials - a work in progress.

PLAIN LANGUAGE SUMMARY: Funders of research are increasingly requiring researchers to involve patients and the public in their research. Patient and public involvement (PPI) in research can potentially help researchers make sure that the design of their research is relevant, that it is participant friendly and ethically sound. Using and sharing PPI resources can benefit those involved in undertaking PPI, but existing PPI resources are not used consistently and this can lead to duplication of effort. This paper describes how we are developing a toolkit to support clinical trials teams in a clinical trials unit. The toolkit will provide a key 'off the shelf' resource to support trial teams with limited resources, in undertaking PPI. Key activities in further developing and maintaining the toolkit are to: ● listen to the views and experience of both research teams and patient and public contributors who use the tools; ● modify the tools based on our experience of using them; ● identify the need for future tools; ● update the toolkit based on any newly identified resources that come to light; ● raise awareness of the toolkit and ● work in collaboration with others to either develop or test out PPI resources in order to reduce duplication of work in PPI. ABSTRACT: Background Patient and public involvement (PPI) in research is increasingly a funder requirement due to the potential benefits in the design of relevant, participant friendly, ethically sound research. The use and sharing of resources can benefit PPI, but available resources are not consistently used leading to duplication of effort. This paper describes a developing toolkit to support clinical trials teams to undertake effective and meaningful PPI. Methods The first phase in developing the toolkit was to describe which PPI activities should be considered in the pathway of a clinical trial and at what stage these activities should take place. This pathway was informed through review of the type and timing of PPI activities within trials coordinated by the Clinical Trials Research Centre and previously described areas of potential PPI impact in trials. In the second phase, key websites around PPI and identification of resources opportunistically, e.g. in conversation with other trialists or social media, were used to identify resources. Tools were developed where gaps existed. Results A flowchart was developed describing PPI activities that should be considered in the clinical trial pathway and the point at which these activities should happen. Three toolkit domains were identified: planning PPI; supporting PPI; recording and evaluating PPI. Four main activities and corresponding tools were identified under the planning for PPI: developing a plan; identifying patient and public contributors; allocating appropriate costs; and managing expectations. In supporting PPI, tools were developed to review participant information sheets. These tools, which require a summary of potential trial participant characteristics and circumstances help to clarify requirements and expectations of PPI review. For recording and evaluating PPI, the planned PPI interventions should be monitored in terms of impact, and a tool to monitor public contributor experience is in development. Conclusions This toolkit provides a developing 'off the shelf' resource to support trial teams with limited resources in undertaking PPI. Key activities in further developing and maintaining the toolkit are to: listen to the views and experience of both research teams and public contributors using the tools, to identify the need for future tools, to modify tools based on experience of their use; to update the toolkit based on any newly identified resources that come to light; to raise awareness of the toolkit and to work in collaboration with others to both develop and test out PPI resources in order to reduce duplication of work in PPI.

Depressed mood, cognitive impairment, and survival in older people admitted to care homes in England.

OBJECTIVE: To investigate psychiatric morbidity, cognitive impairment, dependency, and survival in residents newly admitted to care homes. METHODS: A total of 308 older people were assessed using measures of cognitive impairment and depressive symptomatology, and interviewable residents completed a quality-of-life interview. Dependency levels were assessed by interviews with staff and medication data were collected from home records. Follow-up assessments were carried out at five and nine months. A telephone follow-up approximately 12 months later augmented the survival data. RESULTS: Of residents whose outcomes were known, 73% survived throughout the nine-month study period. Residents who died before the five-month follow-up had higher scores on the depression measure than those surviving longer. Reduced survival was predicted by greater dependency at baseline. Of 188 surviving residents, 63 (38%) were classified as depressed at baseline. Twenty-seven (43%) of the latter were still classed as depressed at five and nine months. Just 19% of residents rated as depressed at baseline were prescribed antidepressant medication, increasing to 26% at each follow-up. There was significant coexistence of cognitive impairment and depressive symptomatology. CONCLUSION: High levels of mortality, psychiatric morbidity, and chronicity of depressed mood among residents requires care homes to improve access to specialist resources such as geriatric consultation, old-age psychiatry, occupational therapy, and physiotherapy. Findings suggest that future care standards should include external factors, such as the extent of access to relevant specialist services for vulnerable older people.