‘It’s a nice thing to do but…’: exploring the methods and impact of patient and public involvement (PPI) in trials

Background and Aims: Patient and Public Involvement (PPI), defined as research carried out ‘with’ or ‘by’ members of the public rather than ‘to’, ‘about’ or ‘for’ them, is increasingly recognized as an essential component of health research. The rationale for PPI is based on a moral argument where the people whose lives are most affected by research should have a say in what is researched and how it is carried out, and a pragmatic argument that PPI can improve research quality. Although PPI is now required by many research funders, academic journals, and ethics committees, progress to achieve greater involvement has been patchy and slow. There is a lack of clarity on how to conduct ‘strong’ PPI and on why PPI should be used. Research is needed on suitable PPI methodologies and on the impact of PPI if we are to develop a shared understanding of what works, when, how and why. Therefore, the overarching aim of this thesis was to contribute to the evidence on the methods and impact of PPI by exploring PPI contributors’ experiences and contributions at the design, conduct and dissemination stages of trials. Methods: At the design stage, two Studies Within A Trial (SWAT) were conducted within the intervention development phase of the Improving Diabetes Eye-screening Attendance (IDEAs) pilot trial. The first used a mixed methods convergent design to compare people with diabetes and healthcare professionals’ experiences of taking part in three different types of consensus meetings to inform intervention development and assess whether their experiences differed according to group composition. The second used a qualitative design to compare people with diabetes and healthcare professionals’ contributions to the intervention content and assess whether their contributions differed according to group composition. At the conduct stage, a systematic review and narrative synthesis was conducted on trial researchers’ perceptions of the impact of PPI on trial retention. At the dissemination stage, a mixed methods SWAT, including an embedded randomised trial, was conducted within the Thyroid Hormone Replacement for Subclinical Hypo-Thyroidism (TRUST) trial to identify, develop, and evaluate a patient preferred method of receiving trial results. Results: Involving PPI contributors simultaneously with other stakeholders led to a perceived lack of common ground where both stakeholders felt reluctant to fully express their opinions. It also led to conflicting opinions which were difficult to incorporate into the intervention being developed. Researchers perceived PPI to have a positive impact on trial retention as it helped trial researchers to foster a trusting relationship and improve communication with trial participants. PPI was also perceived to improve trial retention by ensuring the trial location was suitable and accessible and enabling researchers to establish cultural appropriateness by ensuring that community customs, norms and social activities were considered in the research design. Although, PPI contributors were involved in the development of the trial result letter, the results of the embedded randomised trial suggested that PPI did not make a difference to participants’ understanding of trial results. Conclusions This research shows that although there are a wide variety of methods used to involve PPI contributors, the method used can have an important influence on the impact of involvement. The results suggest that it may be more suitable and useful to involve PPI contributors separately rather than simultaneously with other stakeholder groups. This finding may assist researchers and PPI contributors in designing and conducting more meaningful and effective involvement activities. This research found that PPI can influence the research process by creating and fostering trust between researchers and participants and PPI contributors can help researchers to communicate more effectively with research participants. Although, the results suggest that PPI did not make a difference to participants’ understanding of results, suggestions for how researchers should approach future evaluations of the methods and impact of PPI have been put forward. This research paves the way forward for building an evidence base for PPI to ensure that a shared understanding of what works, when, how and why is developed among researchers, patients, members of the public and research funders

Participants’ perspectives and preferences on clinical trial result dissemination: The TRUST Thyroid Trial experience

Background: While there is an increasing consensus that clinical trial results should be shared with trial participants, there is a lack of evidence on the most appropriate methods. The aim of this study is to use a patient and public involvement (PPI) approach to identify, develop and evaluate a patient-preferred method of receiving results of the Thyroid Hormone Replacement for Subclinical Hypo-Thyroidism Trial (TRUST). Methods: This is a mixed methods study with three consecutive phases. Phase 1 iteratively developed a patient-preferred result method using semi-structured focus groups and a consensus-orientated-decision model, a PPI group to refine the method and adult literacy review for plain English assessment. Phase 2 was a single-blind parallel group trial. Irish TRUST participants were randomised to the intervention (patient-preferred method) and control group (standard method developed by lead study site). Phase 3 used a patient understanding questionnaire to compare patient understanding of results between the two methods. Results: Patients want to receive results of clinical trials, with qualitative findings indicating three key themes including ‘acknowledgement of individual contribution’, ‘contributing for a collective benefit’ and ‘receiving accessible and easy to understand results’. Building on these findings, a patient-preferred method of receiving results was developed as described above. TRUST participants (n=101) were randomised to the intervention. The questionnaire response rate was 74% for the intervention group and 62% for the control group. There were no differences in patient understanding between the two methods. Conclusions: We have demonstrated that it is feasible to conduct PPI with regard to the dissemination of results. The study identified and developed a patient-preferred method of receiving clinical trial results for older adults over 65 years. Although, in this study PPI did not influence patients’ final understanding of results, it provides a record of the process of conducting PPI within the clinical trial setting

Perceived barriers and facilitators to Risk Based Monitoring in academic-led clinical trials: a mixed methods study.

BACKGROUND: In November 2016, the ICH published a requirement for sponsors to develop a systematic, prioritised, risk-based approach to monitoring clinical trials. This approach is more commonly known as risk-based monitoring (RBM). However, recent evidence suggests that a 'gold standard', validated approach to RBM does not exist and it is unclear how sponsors will introduce RBM into their organisations. A first step needed to inform the implementation of RBM is to explore academic trialists' readiness and ability to perform RBM. The aim of this paper is to identify the attitudes and perceived barriers and facilitators to the implementation of RBM in academic-led clinical trials in Ireland. METHODS: This is a mixed-methods, explanatory sequential design, with quantitative survey followed by semistructured interviews. Academic clinical researchers (N = 132) working in Ireland were surveyed to examine their use and perceptions of RBM. A purposive sample of survey participants (n = 22) were then interviewed to gain greater insight into the quantitative findings. The survey and interview data were merged to generate a list of perceived barriers and facilitators to RBM implementation, with suggestions for, and solutions to, these issues. RESULTS: Survey response rate was 49% (132/273). Thirteen percent (n = 18) of responders were not familiar with the term risk-based monitoring and less than a quarter of respondents (21%, n = 28) had performed RBM in a clinical trial. Barriers to RBM implementation included lack of RBM knowledge/training, increased costs caused by greater IT demands, increased workload for trial staff and lack of evidence to support RBM as an effective monitoring approach. Facilitators included participants' legal obligation to perform RBM under the new ICH-GCP guidelines, availability of RBM guidance and perception of cost savings by performing RBM in future trials. CONCLUSION: The results of this study demonstrate a need for training and regulatory-endorsed guidelines to support the implementation of RBM in academic-led clinical trials. The study provides valuable insights to inform interventions and strategies by policy-makers and clinical trial regulators to improve RBM uptake

Study within a trial (SWAT) protocol. Participants' perspectives and preferences on clinical trial result dissemination: The TRUST Thyroid Trial experience.

INTRODUCTION: Dissemination of results of randomised controlled trials is traditionally limited to academic and professional groups rather than clinical trial participants. While there is increasing consensus that results should be communicated to trial participants, there is a lack of evidence on the most appropriate methods of dissemination. This study within a trial (SWAT) aims to address this gap by using a public and patient involvement (PPI) approach to identify, develop and evaluate a patient-preferred method of receiving trial results of the Thyroid Hormone Replacement for Subclinical Hypothyroidism Trial (TRUST). METHODS: An experimental (intervention) study will be conducted using mixed methods to inform the development of and evaluation of a patient-preferred method of communication of trial results. The study will involve three consecutive phases. In the first phase, focus groups of trial participants will be conducted to identify a patient-preferred method of receiving trial results. The method will be developed and then assessed and refined by a patient and public expert group. In the second phase participants will be randomly assigned to the intervention (patient-preferred method) and comparison groups (standard dissemination method as developed by the lead study site in Glasgow, Scotland). In the third phase, a quantitative questionnaire will be used to measure and compare patient understanding of trial results between the two groups. DISCUSSION: This protocol provides a template for other trialists who wish to enhance patient and public involvement and additionally, will provide empirical evidence on how trialists should best disseminate study results to their participants

Understanding the uptake of a national retinopathy screening programme: An audit of patients with diabetes in two large primary care centres [version 1; peer review: awaiting peer review

Background: Diabetic retinopathy (DR) affects 8.2% of the Irish population with type 2 diabetes over 50 years and is one of the leading causes of blindness among working-age adults. Regular diabetic retinopathy screening (DRS) can reduce the risk of sight loss. In 2013, the new national screening programme (RetinaScreen) was introduced in Ireland. Maximising DRS uptake (consent to participate in the programme and attendance once invited) is a priority, therefore it is important to identify characteristics which determine DRS uptake among those with diabetes in Ireland. We report uptake in an Irish primary care population during the initial phase of implementation of RetinaScreen and investigate factors which predict consenting to participate in the programme. Methods: In two primary care practices, data were extracted from records of people with diabetes (type 1 and type 2) aged ≥18 years who were eligible to participate in RetinaScreen between November 2013 and August 2015. Records were checked for a RetinaScreen letter. RetinaScreen were contacted to establish the status of those without a letter on file. Multivariable Poisson regression was used to examine associations between socio-demographic variables and consenting. Adjusted incident rate ratios (IRR) with 95% CI were generated as a measure of association. Results: Of 722 people with diabetes, one fifth (n=141) were not registered with RetinaScreen. Of 582 who were registered, 63% (n=365) had participated in screening. Most people who consented subsequently attended (n=365/382, 96%). People who had attended another retinopathy screening service were less likely to consent (IRR 0.65 [95%CI 0.5-0.8]; p<0.001). Other predictors were not significantly associated with consent. Conclusions: Over one third of people eligible to participate in RetinaScreen had not consented. Research is needed to understand barriers and enablers of DRS uptake in the Irish context. Implementing strategies to improve DRS uptake (consent and attendance) should be a priority

Participants' perspectives and preferences on clinical trial result dissemination: The TRUST Thyroid Trial experience.

Background: While there is an increasing consensus that clinical trial results should be shared with trial participants, there is a lack of evidence on the most appropriate methods. The aim of this study is to use a patient and public involvement (PPI) approach to identify, develop and evaluate a patient-preferred method of receiving results of the Thyroid Hormone Replacement for Subclinical Hypo-Thyroidism Trial (TRUST). Methods: This is a mixed methods study with three consecutive phases. Phase 1 iteratively developed a patient-preferred result method using semi-structured focus groups and a consensus-orientated-decision model, a PPI group to refine the method and adult literacy review for plain English assessment. Phase 2 was a single-blind parallel group trial. Irish TRUST participants were randomised to the intervention (patient-preferred method) and control group (standard method developed by lead study site). Phase 3 used a patient understanding questionnaire to compare patient understanding of results between the two methods. Results: Patients want to receive results of clinical trials, with qualitative findings indicating three key themes including 'acknowledgement of individual contribution', 'contributing for a collective benefit' and 'receiving accessible and easy to understand results'. Building on these findings, a patient-preferred method of receiving results was developed as described above. TRUST participants (n=101) were randomised to the intervention. The questionnaire response rate was 74% for the intervention group and 62% for the control group.  There were no differences in patient understanding between the two methods.  Conclusions: We have demonstrated that it is feasible to conduct PPI with regard to the dissemination of results. The study identified and developed a patient-preferred method of receiving clinical trial results for older adults over 65 years. Although, in this study PPI did not influence patients' final understanding of results, it provides a record of the process of conducting PPI within the clinical trial setting

Participants’ perspectives and preferences on clinical trial result dissemination: The TRUST Thyroid Trial experience [version 2; peer review: 2 approved]

Background: While there is an increasing consensus that clinical trial results should be shared with trial participants, there is a lack of evidence on the most appropriate methods. The aim of this study is to use a patient and public involvement (PPI) approach to identify, develop and evaluate a patient-preferred method of receiving results of the Thyroid Hormone Replacement for Subclinical Hypo-Thyroidism Trial (TRUST). Methods: This is a mixed methods study with three consecutive phases. Phase 1 iteratively developed a patient-preferred result method using semi-structured focus groups and a consensus-orientated-decision model, a PPI group to refine the method and adult literacy review for plain English assessment. Phase 2 was a single-blind parallel group trial. Irish TRUST participants were randomised to the intervention (patient-preferred method) and control group (standard method developed by lead study site). Phase 3 used a patient understanding questionnaire to compare patient understanding of results between the two methods. Results: Patients want to receive results of clinical trials, with qualitative findings indicating three key themes including ‘acknowledgement of individual contribution’, ‘contributing for a collective benefit’ and ‘receiving accessible and easy to understand results’. Building on these findings, a patient-preferred method of receiving results was developed as described above. TRUST participants (n=101) were randomised to the intervention. The questionnaire response rate was 74% for the intervention group and 62% for the control group. There were no differences in patient understanding between the two methods. Conclusions: We have demonstrated that it is feasible to conduct PPI with regard to the dissemination of results. The study identified and developed a patient-preferred method of receiving clinical trial results for older adults over 65 years. Although, in this study PPI did not influence patients’ final understanding of results, it provides a record of the process of conducting PPI within the clinical trial setting

Feasibility of an implementation intervention to increase attendance at diabetic retinopathy screening: protocol for a cluster randomised pilot trial

Background: Diabetic retinopathy screening (DRS) leads to the earlier detection of retinopathy and treatment that can prevent or delay the development of diabetes-related blindness. However, uptake continues to be sub-optimal in many countries, including Ireland. Routine management of type 2 diabetes largely takes place in primary care. As such, there may be an opportunity in primary care to introduce interventions to improve DRS uptake. However, few studies test the feasibility of interventions to enhance DRS uptake in this context. Our aim is to investigate the feasibility of an implementation intervention (IDEAs (Improving Diabetes Eye screening Attendance)) delivered in general practice to improve the uptake of the national DRS programme, RetinaScreen. Methods: The IDEAs study is a cluster randomised pilot trial with an embedded process evaluation and economic evaluation. Following stratification by practice size, eight general practices (clusters) will be randomly allocated to intervention (n = 4) or wait-list control groups (n = 4). The intervention will be delivered for 6 months, after which, it will be administered to wait-list control practices. The intervention is multi-faceted and comprises provider-level components (training, audit and feedback, health care professional prompt, reimbursement) and patient-level components (GP-endorsed reminder with information leaflet delivered opportunistically face-to-face, and systematically by phone and letter). Patient inclusion criteria are type 1 or type 2 diabetes and DRS programme non-attendance. A multi-method approach will be used to determine screening uptake, evaluate the trial and study procedures and examine the acceptability and feasibility of the intervention from staff and patient perspectives. Quantitative and qualitative data will be collected on intervention uptake and delivery, research processes and outcomes. Data will be collected at the practice, health professional and patient level. A partial economic evaluation will be conducted to estimate the cost of delivering the implementation intervention in general practice. Formal continuation criteria will be used to determine whether IDEAs should progress to a definitive trial. Discussion: Findings will determine whether IDEAsis feasible and acceptable and will be used to refine the intervention and study procedures. A definitive trial will determine whether IDEAs is a cost-effective intervention to improve DRS uptake and reduce diabetes-related blindness. Trial registration: ClinicalTrials.gov NCT03901898. Registered 3rd April 2019

Community research report

University College Cork introduced its first Community-based Participatory Research (CBPR) module in 2016. The module was funded and supported by Horizon2020 funding, specifically the EnRRICH project (Enhancing Responsible Research and Innovation through Curricula in Higher Education). The module is a 5-credit module for PhD students from all disciplines in the early stages of their PhD at University College Cork. Following two fruitful partnerships in the areas of social justice / equality, community family support services and older persons, there was a keen interested to explore partnerships in markedly different areas such as environmental sustainability. A dialogue ensued with CEF where the opportunity and feasibility to collaborate on the CBPR module was explored