Patient and public involvement and engagement in target trial emulation framework: a scoping review protocol.

Objectives The objective of this rapid review is to understand the reporting, role and quality of patient and public involvement and engagement (PPIE) in real world data and evidence (RWDE) research across the medicines development cycle. Methods We comprehensively searched, with no date restrictions, Medline and Embase databases for peer-reviewed literature and conference abstracts (Embase only) reporting PPIE in RWD studies. We also assessed PPIE in a sample of 100 NICE technology appraisals (TAs) comprising both single technology appraisals (STAs) and highly specialized technologies (HSTs). We used standard methods for screening and data extraction. In addition, we used the Patient Focused Medicines Development (PFMD)’s Patient Engagement Quality Guidance (PEQG) as a framework to assess the quality of PPIE. We planned to conduct narrative synthesis of included studies, however there were insufficient studies and data reported. Results We included three RWD studies that reported PPIE from the peer-reviewed literature and two NICE HSTs. One of the HSTs included data from one of the peer-reviewed journal articles. Reporting of PPIE in included studies was limited. No studies reported a PPIE framework and it was unclear how integrated and meaningful PPIE was. Four out of seven of PFMD’s quality criteria for PPIE were poorly reported by included studies. This suggests reporting and/or conduct of PPIE requires improvement in RWD studies. Conclusions Our review found that PPIE was rarely reported in RWDE research and uncovers a need for consistent reporting. For most publications there was insufficient information to judge the extent to which patients and carers, were considered meaningful partners. However, our review provided preliminary evidence that PPIE can influence protocol development, recruitment, and retention methods in RWD studies. More inclusive approaches to PPIE would help interpretation of RWE regarding relevance and importance to patients and carers.

Background Patient and public involvement and engagement (PPIE) in research is a collaboration between researchers, patients, and the public, enhancing research acceptability, relevance, and impact. There is a growing prevalence of PPIE in high-income country research; however, its integration in low- and middle-income countries (LMICs) remains poorly understood. Recognising this gap, the Ziauddin University Clinical Trials Unit in Karachi, Pakistan, launched a dedicated PPIE initiative in 2022. This study evaluates the engagement process and experiences of patient and public members and researchers to identify barriers and facilitators to participation within the PPIE group. Methods The evaluation uses an explanatory sequential mixed-method design. First, the Public and Patient Engagement Evaluation Tool (PPEET) questionnaire will be administered online to group members, coordinators, and senior institutional leads. Insights from questionnaires will be further explored during semi-structured interviews, with questions guided by the Patient Engagement in Research (PEIR) framework, supplemented with analysis of project documentation. Study activities will be conducted in both English and Urdu. The study has been co-designed with PPIE members and is co-led with a public partner. Findings will highlight areas for improvement, inform best practices, and guide the development of more effective engagement strategies. Outcome Although focused on a single group, this evaluation lays the groundwork for understanding PPIE practices in LMIC contexts. It provides valuable insights into developing equitable partnerships and improving patient-centred research. This study contributes to a growing body of knowledge, offering practical guidance for implementing PPIE in settings with unique socioeconomic challenges and cultural realities. The findings are expected to benefit the local research community and similar initiatives globally, particularly in regions with comparable challenges.

To identify and map the existing literature on the conduct of patient and public involvement and engagement (PPIE) for statistical methodology. PPIE refers to the consideration of patient and public perspectives into research development, conduct and dissemination and, while commonly integrated in applied healthcare research, it currently remains underutilised in statistical methodology research. Many statistical methodologists lack confidence in conducting PPIE citing barriers such as insufficient training, unclear tasks, and concerns about impact. This review will examine literature on PPIE in the context of statistical methodology aiming to inform the design or analysis of healthcare research studies and related methodological research domains such as trials methodology and health data science. A three-step search strategy, developed with an information specialist and based on JBI guidelines, will identify published and unpublished PPIE literature. This will include database searches, hand-searching key journals, and grey literature searches via search engines. The authors will contact their professional networks to identify additional material. The literature will be screened, selected and extracted by two independent researchers. Results will be presented in an evidence map and using a qualitative, thematic analysis.

BackgroundPatient and Public Involvement and Engagement (PPIE) is an essential element of any clinical trial, to ensure that the research design and resources are acceptable and the trial produces findings that are relevant to research participants and service-users. The WILL (When to Induce Labour to Limit risk in pregnancy hypertension) Trial Management Team involved a group of patient and public members to develop an infographic, for service-users and -providers, to communicate the risks of chronic and gestational hypertension at term gestational age.MethodsBased on national resources in the UK for PPIE, including those from the National Institute for Health and Care Research (NIHR) and Health Research Authority, active trial sites (all in the UK) were approached to invite women with pregnancy hypertension to the PPIE group, and advertisements were placed on the Action on Pre-Eclampsia Charity (APEC) and NIHR People in Research websites. PPIE resources were established by the Trial Management Team and virtual meetings with the PPIE members held to co-design and develop the infographic from existing trial materials, using Microsoft Publisher.ResultsSeven diverse PPIE members were involved, six from NIHR People in Research and one from APEC. No members were engaged from active sites. From initial set-up to REC approval of the infographic took 9 months, and a considerable time commitment from the Trial Manager and Senior Research Midwives, to navigate practicalities (e.g. team consensus-building). Four meetings were held and 15 iterations of the infographic were developed with the group. The infographic incorporated requests from PPIE members for a design that was inclusive of all races, cultures, abilities and genders. Discussions with PPIE group members improved our understanding of the acceptability of the trial intervention to some cultures. The Research Ethics Committee (REC) requested revisions to PPIE-approved wording about the risk of stillbirth, from ‘1 in 1000’ to ‘small increased risk’ prior to issuing approval for use.ConclusionsMore practical support would be useful for researchers seeking to establish PPIE groups, and RECs reviewing resources that are co-produced. Our case study provides a practical, step-by-step account of establishing a PPIE group without a funded co-lead, highlighting strengths such as the diversity of members, the incorporation of cultural perspectives, and the development of inclusive patient-facing materials. These findings add to PPIE practice by offering operational guidance, lessons learned and a summary checklist as an additional resource that can complement existing high-level frameworks.Trial registrationISRCTN77258279, registered on 05 December 2018.Supplementary InformationThe online version contains supplementary material available at 10.1186/s13063-025-09218-6.

Introduction Patient and Public Involvement and Engagement (PPIE) involves working ‘with’ or ‘by’ patients and the public, rather than ‘to,’ ‘about,’ or ‘for’ them, and is integral to neurological and digital health research. This rapid review examined PPIE integration in the development and implementation of digital health technologies for neurological conditions. Methods Key terms were input into six databases. Included articles were qualitative studies or PPIE activities involving patient perspectives in shaping digital health technologies for neurological conditions. Bias was evaluated using the NICE qualitative checklist, with reporting following PRISMA guidelines. Results 2,140 articles were identified, with 28 included. Of these, 25 were qualitative studies, and only three were focused PPIE activities. Patient involvement was mostly limited to one-off consultations during development.There was little evidence of PPIE during implementation, and minimal reporting on its impact. Conclusions PPIE has been inconsistently reported in this research area, highlighting the need for more guidance and best-practice examples This review used a UK-based definition of PPIE, which may have excluded relevant activities from other countries. Future reviews should broaden terminology to capture PPIE integration globally.

Patient and Public Involvement and Engagement (PPIE) – sometimes called CommunityEngagement and Involvement (CEI) – comes as a big challenge but one that can bevery helpful for health care professionals and stakeholders in planning betterhealth policies for attending to the main needs of the community. PPIE involvesthree pillars: public involvement, public engagement, and participation. Publicinvolvement occurs when members of the general population are actively involvedin developing the research question, designing, and conducting the research.Public engagement tells people about new studies, why they are important, theimpact of results, the possible implication of the main findings for thecommunity, and the possible impact of these new findings in society, as well as,in the dissemination of knowledge to the general population. Participation isbeing a volunteer in the study. Our experience with PPIE, to the best of ourknowledge the first initiative in Brazil, is a partnership with the Universityof Birmingham, the University of Liverpool, and the NIHR Global Health Group onAtrial Fibrillation (AF) Management focusing on the AF care pathway exploringthe important aspects of diagnosis and treatment in the primary care system froma low-middle income area in São Paulo. The involvement of patients/public in theresearch represents a new step in the process of inclusion of all segments ofour society based on patient illness and the gaps in knowledge aiming to opennew horizons for continuous improvement and better acceptance of researchprojects.

Patient and public involvement and engagement (PPIE) improves research quality but is often limited in scale. This study explored the potential for large-scale PPIE using a Web-based approach. We created an online portal to gather public views on dementia research and a UK-based adaptive platform trial testing repurposed Alzheimer's disease drugs. Participants ranked four anonymized drugs and completed discrete choice experiments on treatment trade-offs. Analyses were stratified by sex, age, and dementia experience. Among 3250 people across 27 countries (87.4% UK-based), 79.6% expressed positive attitudes toward the trial. Metformin was the most preferred drug, followed by atomoxetine, isosorbide mononitrate, and levetiracetam. Probability of severe side effects was the most influential treatment attribute, followed by probability of mild side effects and type of evidence. Subgroup analyses supported the main findings. Web-based PPIE can effectively inform dementia research at scale and provides a reusable resource for other studies.

BackgroundThere is an established history of patient and public involvement and engagement (PPIE) in academic and clinical research. As the National Institute for Health and Care Research (NIHR) expands its investment in research on and by local authorities (LAs), NIHR PPIE frameworks are increasingly being applied in this new context. This article examines if and how the relationship between the public varies across universities, the NHS and LA and what this means for PPIE.MethodsTo analyse differences in institutional structures, we reviewed organisational websites, comparing the purpose and responsibilities of the institution, funding sources, governance structures, ability to directly action research findings, the role of public collaborators and duration of this relationship. We then systematically analysed these differences against the six UK Standards for Public Involvement: inclusive opportunities, working together, support and learning, governance, communications and impact. We also held a group discussion with nine PPIE Research Advisory Panel members to sense check if and how they perceived differences across these three institutional contexts and to refine and identify additional hypotheses about what might need to be adapted for PPIE in a LA setting.ResultsThe three institutions generally fall along a continuum, with universities having the most bounded relationship with the public and LAs the most expansive and enduring. The NHS and LAs have statutory responsibilities to the public, who finance their services and whose rights are articulated in institutional constitutions. Reflective of the service delivery responsibilities of both institutions, they are able to directly implement research findings, whereas university research outputs predominantly aim to inform others’ service design and delivery. Given these differences, our analysis suggests that the three standards on working together, governance and PPIE impact may require greater adaptation in LA settings. At the heart of the challenge is role clarification, since public contributors to research may also be council tenants, taxpayers and voters.ConclusionsPPIE in LA research offers new opportunities and challenges, requiring tailored guidance that accounts for the unique relationship between LAs and the public. We encourage PPIE contributors, coordinators and scholars across institutional settings to work together to fill this gap.Supplementary InformationThe online version contains supplementary material available at 10.1186/s40900-025-00732-0.

Background Alcohol use is the third leading risk factor of death and disability in the UK and costs the NHS £3.5 billion per year. Despite the high prevalence and healthcare burden of Alcohol-related Liver Disease (ArLD), there has been minimal research addressing prevention, morbidity and mortality. Reasons for this include stigma and lack of interest from public, charitable and commercial funding bodies. The objectives of this project were to identify and develop interdisciplinary partnerships, to explore stigma in ArLD, to develop a representative Patient and Public Involvement and Engagement (PPIE) group, to build research capacity, and to develop interdisciplinary research proposals targeting key research priorities. Methods ArLD networks were identified by members of the Project Steering Group. Health Care Professionals (HCPs) from different backgrounds were invited to join the ARMS-Hub. PPIE representatives were invited through charities and support groups. Research areas were identified, discussed, prioritised and ranked. Research questions were refined during an in-person symposium. A mentorship programme was created to encourage and facilitate networking and knowledge exchange for early career researchers. Results We established the ARMS-Hub with 31 HCPs and 40 PPIE members. There were five stakeholder meetings, which included PPIE representation. Three virtual and three in-person PPIE meetings took place. Topics relevant to stigma in ARLD identified during the meetings were education and awareness, language, and access. Priorities identified were the disconnect between mental health and liver services, education around the wider harms of alcohol, and education of HCPs regarding stigma. We established a mentorship network that regularly meets to support development of new research ideas. Conclusions Stigma is central to lack of research engagement from professionals and PPIE. The main priority identified relates to the disconnect between mental health and liver services. This collaborative study has allowed development of a research agenda to address this priority.

BackgroundMost implementation research focuses on understanding and changing health professionals’ work practices. While Patient and Public Involvement and Engagement (PPIE) in applied health research is recognised as best practice, and is often a requirement for funders globally, little is known about the role of patients and the public in implementation research.MethodsGuided by Arksey and O’Malley’s framework, we conducted a scoping review to categorise PPIE in the design and conduct of implementation research, including how patients and the public have been involved, the reported impact of patient and public involvement, and the reported benefits and challenges to involving patients and the public in implementation research. We searched four databases: MEDLINE, Embase, CINAHL, and SCOPUS. To be included, studies had to report some form of PPIE in the design and conduct of implementation research. Information about each study was extracted using a structured data extraction form. Data was collated and summarised.ResultsOf the 535 unique records identified, 12 were included. Analysis of the eligible studies found eight different types of PPIE activities. Researchers mostly consulted with patients and members of the public via feedback sessions, committee representation and roundtable discussions. Barriers and enablers were usually researcher related and their attempts to build, maintain, and negotiate relationships with public contributors over time. Resources and financial remuneration were also key. Most studies (n = 7) reported that engaging community members in the design and implementation of community-based programs and trials enhanced cultural appropriateness, and the likelihood of sustainability. However, there was little formal evaluation of the use of PPIE.ConclusionThis study can be used to design and guide future PPIE in implementation research. Given the inconsistent, and often absent, reporting of PPIE activities and barriers and enablers across the included studies, future studies should describe and evaluate the execution of PPIE in implementation research to advance practices in this field.RegistrationThe review was registered on Research Registry (reviewregistry1552).

Patient and public involvement and engagement (PPIE) can provide valuable insights into the experiences of those living with and affected by a disease or health condition. Inclusive collaboration between patients, the public and researchers can lead to productive relationships, ensuring that health research addresses patient needs. Guidelines are available to support effective PPIE; however, evaluation of the impact of PPIE strategies in health research is limited. In this Review, we evaluate the impact of PPIE in the 'Therapies for Long COVID in non-hospitalised individuals' (TLC) Study, using a combination of group discussions and interviews with patient partners and researchers. We identify areas of good practice and reflect on areas for improvement. Using these insights and the results of a survey, we synthesize two checklists of considerations for PPIE, and we propose that research teams use these checklists to optimize the impact of PPIE for both patients and researchers in future studies.

BackgroundThe EJS ACT-PD initiative is designing a multi-arm multi-stage trial for disease modification in Parkinson’s. A patient and public involvement and engagement (PPIE) working group (WG) comprises 2-3 people with...

BackgroundThe UNderstanding Factors that explain Avoidable hospital admission Inequalities – Research study (UNFAIR)’s Patient and Public Involvement and Engagement (PPIE) representatives raised that health inequalities (HI) are complex and can...

UNderstanding Factors that explain Avoidable hospital admission Inequalities-Research study (UNFAIR) addresses how to reduce health inequalities, particularly for avoidable hospital admissions. Our Patient and Public Involvement and Engagement (PPIE) members broached that health inequalities are complex, challenging to understand and communicate. They identified a need to explore diverse views, including people who have a higher risk of health inequalities. With limited public-facing resources relating to the public's understanding or emotions around health inequalities, this project aimed to fill this gap using co-leadership and co-production. Members of the public worked with researchers to co-produce and run PPIE workshops. Thisproject was co-led by a member of the public and a researcher. One online workshop open to anyone in England accompanied by three face-to-face workshops were held. Public contributors, including people living in diverse communities, were invited. Inclusive involvement opportunities were offered including flexible ways of involvement and remuneration. To strengthen the key messages' rigour, transcriptions of the audio-recordings from each workshop, with facilitator notes, were analysed using thematic analysis. From the key messages, an animation was co-produced with public contributors with the public's voice being integral throughout. A total of 58 people took part capturing intersecting and multiple dimensions of marginalisation including people with a range of ages, genders, ethnicities, socioeconomic backgrounds, and members of communities who face exclusion (including people with learning difficulties and experiencing ill-health). The animation highlighted powerful lived experience, for example, some people are dying earlier than expected. Health inequalities conjured up powerful emotions, such as anger and hopelessness. Public views of how to address health inequalities included respecting, accepting and valuing everyone, regardless of, for example, where people live. The animation is publicly available for use by anyone, including decision makers across the health and care system. Through co-leadership and co-production, this project is an example of inclusive PPIE. This project provided a way for the public's voice to influence policy and practice to inform understanding and action to address health inequalities. The animation provides powerful insights into what health inequalities mean to people with examples of lived experience and corroborates the moral argument for action by decision makers. Members of the public, including people who were affected or at higher risk of health inequalities, co-led this project and were involved as co-creators and developers from the inception of the project to completion. Their involvement was integral and documented in full throughout the project.

BackgroundEULAR guidelines for osteoarthritis (OA) endorse high quality care and support to self-manage with core recommended treatments such as exercise, weight loss and the provision of written information and education....