Patient Voices Leading Change

Plain English summaryRetinoblastoma is a rare eye cancer that occurs in one or both eyes of infants and young children as a result of errors in the RB1 gene. There are approximately 2000 retinoblastoma survivors in Canada. Those with the heritable form of the disease are at risk of passing the gene to the next generation and developing a second cancer. Many retinoblastoma survivors and families therefore interact with the healthcare system throughout their lives.The retinoblastoma community has a longstanding history of engaging patients in research, however without any formal process. The literature establishes benefits to patient engagement such as research results which are more applicable, credible, and transparent. Building on the established interest among stakeholders, the Canadian Retinoblastoma Research Advisory Board (CRRAB) was established in 2016 to foster sustainable and meaningful collaboration between patients (survivors and family members), advocacy groups, healthcare professionals, and researchers in the retinoblastoma community.The aim of this study was to evaluate the utility of CRRAB in fostering patient engagement in research. Members of CRRAB were surveyed to uncover their attitudes towards and experience with patient engagement in research. Participants perceived CRRAB to provide diverse and accessible opportunities for patient engagement in research and perceived their participation to have a meaningful impact. The results suggest that CRRAB promotes patient engagement in retinoblastoma research, and provides direction to sustain and enhance future patient engagement.BackgroundThe Canadian Retinoblastoma Research Advisory Board (CRRAB) is a multidisciplinary group, including patients (survivors and family members), advocacy groups, healthcare professionals, and researchers, which aims to establish and sustain patient engagement in retinoblastoma research. The purpose of this study was to describe the development of CRRAB and to uncover members’ understanding of and attitudes towards patient engagement in research. As well, to determine their level of engagement.MethodsRetinoblastoma patients, healthcare professionals, and researchers provided leadership to co-develop CRRAB. CRRAB members were surveyed by pre- and post-test questionnaire at the 2016 Annual General Meeting to assess experience with, understanding of, and attitudes towards patient engagement in research. A second questionnaire was administered before the 2017 CRRAB meeting to assess awareness and perceived impact of CRRAB activities, and individual engagement in research. Data were analyzed by descriptive statistics and paired t-test (for pre/post-test). Thematic analysis of chart board discussions at both meetings revealed the joint goals of CRRAB and reasons for and barriers to patient engagement.ResultsIn 2016, 21 individuals participated and self-identified as patients (11, 52%), healthcare professionals (6, 29%), and/or researchers (7, 33%) (participants could overlap stakeholder groups). Overall, participants believed that research is relevant to all stakeholders and that patients can have meaningful impact on research. In 2017, 35 individuals participated and identified as patients (21, 60%), healthcare professionals (9, 26%), and/or researchers (8, 23%). 94% of participants were aware of CRRAB initiatives and 67% had participated in at least one over the previous year. Participants perceived that CRRAB provides diverse opportunities and increases accessibility for patient engagement in research, and perceived patient engagement to have meaningful impact on retinoblastoma research. Chart board discussions revealed that participants wanted to be part of CRRAB to increase knowledge, support innovation and patient engagement, and be part of a community. Members most commonly faced barriers including time and cost restraints.ConclusionsThe results of this study suggest that CRRAB has supported the engagement needs of patients affected by retinoblastoma, and has provided an opportunity for engaging patients in retinoblastoma research. CRRAB will continue to be used as a framework for patient engagement, with improvements based on participant feedback.

CIHR) -supports research that focuses on cardiovascular health, respiratory health, blood and blood vessels, stroke, critical/intensive care, and sleep/circadian rhythms.Together, the disease areas that are covered under the mandate of the ICRH represent conditions with the largest health, societal, and economic burden of any of the other Institutes within CIHR.This reality and the extraordinary diversity of these medical conditions present a major operational challenge for the ICRH.Despite these difficulties and limited resources, the Institute's 2013-2016 Strategic Plan1 demonstrates a commitment to ensuring that research excellence, capacity, competitiveness, innovation, and impact are enhanced across all research fields affiliated with the Institute.Moreover, building on previous successes, the ICRH continues to nurture past collaborations and work to establish new meaningful national and international alliances with the research community, partners, and other stakeholders to develop interdisciplinary, integrative health research that reflects Canada's emerging health needs, gaps, and opportunities.The ICRH also strongly supports partnerships with relevant stakeholders to accelerate the transfer of new knowledge into benefits for Canadians.Notably, patient engagement is an important priority that will serve as 1 of the major building blocks of all clinically related initiatives of the ICRH.Based on identified gaps, opportunities, and feedback received from the International External Review Panel,2 ICRH research communities and partners, and in alignment with the CIHR 2014-2018 Strategic Plan, the ICRH identified 4 research priorities for 2013-2016.The first priority focuses on enhancing capacity, competitiveness, and impact of our communities through networking.This objective is consistent with the importance of aligning with CIHR's largest comprehensive Roadmap Signature Initiative (RSI) the Strategy for Patient-Oriented Research (SPOR).In 2013, the Institute launched the ICRH Emerging Networks program in 3 gap areas, funding the Canadian Vascular Network, Canadian Respiratory Research Network, and the Canadian Stroke Prevention Intervention Network.In addition, through focused community development programs in critical care, stroke treatment and recovery, sleep and circadian rhythms, blood diseases, and resuscitation clinical research, the ICRH has targeted 5 additional research communities in need of networking.Aligned with CIHR's Patient Engagement Framework,3 the ICRH will use specific actions and tools to enhance patient engagement in health research, implement patient engagement as a core priority in strategic initiatives, and offer programs that will allow patients to be true partners in research.Finally, the ICRH is continuing its collaboration with the National Heart, Lung, and Blood Institute of the National Institutes of Health in the ongoing support of 2 large Canada-US networks--the Resuscitation Outcomes Consortium and the Cardiothoracic Surgical Network, and will explore other international network opportunities.

Patient-Centered Outcomes Research: Early Evidence From A Burgeoning Field.

The engagement of patients and other stakeholders is a critical element in the design of patient-centered outcomes research studies. However, methodology for scalable engagement in research management particularly activities such as operationalization of principles and setting of priorities is not well-developed. The objective of this study is to describe a novel approach for scalable stakeholder engagement in research aligned with the Patient-Centered Outcomes Research Institute (PCORI) engagement principles, which was evaluated in a national clinical data research network. Patient, patient advocate, clinician, and researcher stakeholders were recruited from clinical sites, as well as social media sites related to the 3 conditions of focus, heart failure, obesity, and Kawasaki disease. The engagement strategy was designed, implemented, and mapped to the PCORI engagement principles. Evaluation included internal assessment and quantitative measures of online engagement. We operationalized the PCORI principles with 12 stakeholder engagement strategies and convened stakeholder advisory boards and online research prioritization panels to determine research priorities in a rigorous, deliberative process. A total of 46 advisors (20 patients) and 339 panelists (159 patients) actively participated. There were not significant differences between patients and clinicians in level of online engagement. Nonetheless, while patients reported a slightly greater challenge with following online discussion, they overall had a more favorable opinion about use of the online format. An efficient way to engage large numbers of representative stakeholders in research is a necessary first step to assure the public of trustworthy use of data networks for health research. This paper describes a comprehensive approach to engagement in patient-centered outcomes research management that informs ongoing development of rigorous methodologies in this area.

For the past two decades, the National Cancer Institute (NCI) has supported the involvement of patient advocates in both internal advisory activities and funded research projects to provide a patient perspective. Implementation of the inclusion of patient advocates has varied considerably, with inconsistent involvement of patient advocates in key phases of research such as concept development. Despite this, there is agreement that patient advocates have improved the patient focus of many cancer research studies. This commentary describes our experience designing and pilot testing a new framework for patient engagement at SWOG, one of the largest cancer clinical trial network groups in the United States and one of the four adult groups in the NCI's National Clinical Trials Network (NCTN). Our goal is to provide a roadmap for other clinical trial groups that are interested in bringing the patient voice more directly into clinical trial conception and development. We developed a structured process to engage patient advocates more effectively in the development of cancer clinical trials and piloted the process in four SWOG research committees, including implementation of a new Patient Advocate Executive Review Form that systematically captures patient advocates' input at the concept stage. Based on the positive feedback to our approach, we are now developing training and evaluation metrics to support meaningful and consistent patient engagement across the SWOG clinical trial life cycle. Ultimately, the benefits of more patient-centered cancer trials will be measured in the usefulness, relevance, and speed of study results to patients, caregivers, and clinicians.

Plain English summaryIncreasingly, funders and researchers want to partner with patients in health research, but it can be challenging for researchers to find patient partners. More than taking part in research as participants, patient partners help design, carry out and manage research projects. The goal of this study was to describe ways that patient partners have been recruited by researchers and patient engagement leads (individuals within organizations responsible for promoting and supporting patients as research partners).We talked with researchers and patient engagement leads in Canada and the United Kingdom, as well as a patient representative. We found three ways that could help researchers and patients find each other. One way is a case-by-case basis, where patients are often sought with experience of a health condition that is the focus of the research. The other ways involved directories where projects were posted and could be found by patients and researchers, or a third party matched patients with research projects. We found four recruitment strategies:Social marketingCommunity outreachHealth systemPartnering with other organizations (e.g., advocacy groups)There are many influences on finding, selecting and retaining patient partners: patient characteristics, the local setting, the opportunity, work climate, education and support. We hope study results will provide a useful starting point for research teams in recruiting their patient partners.BackgroundPatient engagement in clinical trials and other health research continues to gain momentum. While the benefits of patient engagement in research are emerging, relatively little is known about recruiting patients as research partners. The purpose of this study was to describe recruitment strategies and models of recruiting patients as partners in health research.MethodsQualitative descriptive study. Thirteen patient engagement leads and health researchers from Canada and the United Kingdom, as well as one patient representative from a national patient organization (7 female) completed semi-structured interviews.ResultsRecruitment infrastructures available to respondents varied, but could be categorized into three models including the traditional, third-party and directory models. Four categories of recruitment strategies were identified, representing multiple ways of recruiting patient partners: social marketing recruitment, community outreach recruitment, health system recruitment, and partnering recruitment.ConclusionsMultiple recruitment strategies were identified for engaging patient partners in research, and some common factors influenced recruitment. Study findings contribute to the evidence base in patient engagement and provide guidance for research teams to help identify potential recruitment methods for their patient partners.

BackgroundOver the last 10 years, patient engagement in health research has emerged as the next evolution in healthcare research. However, limited evidence about the clear role and scope of patient engagement in health research and a lack of evidence about its impact have influenced the uptake, implementation and ongoing evolution of patient engagement. The present study aims to conduct a scoping review to identify methods for and outcomes of patient engagement in health research.MethodsAn adaptation of the scoping review methodology originally described by Arksey and O’Malley and updated by Levac, Colquhoun and O’Brien was applied. Sources from a formal database search and relevant documents from a grey literature search were compiled into data extraction tables. Articles were synthesised into key themes according to the (1) methods and (2) outcomes of patient engagement in health research.ResultsThe total yield for the scoping review was 55 records from across Canada, the United Kingdom and the United States. While evidence about the methods used to engage patients in health research is increasing, stronger evidence of specific patient and healthcare system outcomes is required. This necessitates further mobilisation of research that explores outcomes and that validates specific tools to evaluate engagement. Additionally, theoretical frameworks that can better inform and sustain patient engagement across the lifecycle of health research are lacking.ConclusionFurther increasing the volume and reach of evidence about patient engagement in health research will support the paradigmatic shift needed to normalise the patient’s role in research beyond ‘subject’ or ‘participant’, so as to ultimately improve patient health outcomes and better address healthcare reform in Canada.

Chapter 5 - Patient voice in clinical trial programs in industry

Involving patients and the public in health research helps ensure that studies are of high quality and focus on the issues that matter most to the people affected. However, researchers do not always explain clearly how they have done this, making it hard to understand how these voices have shaped the research. This study looked at over 500 research papers published by authors from a UK Russell Group university to see how well the involvement of patients and the public was reported. Only one in five papers mentioned how patients and the public were involved in the main text, and fewer still acknowledged contributions from patients or public partners. Very few papers included patient authors or collaborators from charities and community organisations. Although most papers were freely available online, only 10% included a plain English summary to help non-specialists understand the research. To better understand these findings, the researchers worked closely with a group of patient co-authors who had experience collaborating with university researchers. Together, they developed seven recommendations to help improve how the involvement of patients and the public is reported in future studies. This work highlights the need for universities and research institutes to reflect on their practices and provide better support for researchers to work meaningfully and transparently with patients and the public. Clear and consistent reporting of the involvement of patients and the public is essential for building trust, improving research quality, and ensuring that health research truly serves the communities it aims to help. Patient and Public Involvement and Engagement is important to ensure research addresses the issues that matter most to patients and the public. However, reporting and publishing of these activities is variable and inconsistent. This rapid review aimed to assess how effectively, consistently, and transparently Patient and Public Involvement and Engagement is reported on in papers published by authors affiliated with a heath research institute at a UK Russell Group university. A rapid review of all papers published by institute-affiliated authors (1st August 2021 to 31st December 2024) identified using PubMed. We also consulted with a group of patient co-authors to understand their experiences of being a Patient and Public Involvement and Engagement co-author in collaboration with researchers from the same institute. We retained and reviewed 523 papers for inclusion of Patient and Public Involvement and Engagement. There was significant variation in the reporting across the papers included. Overall, 21% of papers had reference to Patient and Public Involvement and Engagement activity in the main body of the paper, and 17% of papers included Patient and Public Involvement and Engagement in the acknowledgements. In terms of co-authorship, 5% of papers included an author with an affiliation to a charity/non-governmental organisation that represented Patient and Public Involvement and Engagement -related interested, e.g. Alzheimer’s Society, Caribbean and African Health Network and 1% of papers included a patient/lived-experience author. A total of 97% of papers were published open access but only 10% of articles included in this review had a plain English or lay summary. Only one paper included a published GRIPP2 form. The Patient and Public Involvement and Engagement group reviewed and contextualised our findings, supporting analysis and development of seven key recommendations to improve comprehensive, consistent, and transparent reporting of Patient and Public Involvement and Engagement in health research at an institute level. This work demonstrates the challenges that Patient and Public Involvement and Engagement faces and the importance of institutions reflecting on the practices of their academics, and the structures they put in place to encourage good and equitable practices of working with patents and the public.

Patient engagement is increasingly considered to be an important element in the treatment of brain disorders to optimise outcomes for patients, society, and healthcare systems. Nonetheless, scientific research examining methodologies to engage patients with brain diseases in Research and Innovation (R&I) is scarce. To review existing scientific evidence regarding the engagement of patients with brain disorders in research and innovation. Studies were retrieved from several bibliographic databases (publication date between January 2016 and April 2019) with pre-specified selection criteria. In total, 49 articles were identified as meeting the inclusion criteria and were reviewed systematically. Results showed that there is limited evidence available on the impact and (cost-) effectiveness of patient engagement in (brain) research and innovation. Most published studies are protocols, guidelines, and discussion articles for patient engagement in health research and innovation. Overall, there exists a general consensus to engage patients in every step of the research procedure. Relevant evidence identified includes principles of engagement, definitions of stakeholder types, key considerations for planning, conducting and disseminating engaged research, potential engagement activities, and examples of promising practices. Findings are inconclusive due to methodological differences. Comparison between studies was difficult due to differences in patients, form of engagements, and total duration of engagement of patients. Experiences of patient engagement mainly concern adherence to medical treatments or participation of "expert patients" in clinical trials, but very rarely the governance of R&I according to the dictates of Responsible Research and Innovation (RRI). More structuralized, well-conducted and comparable Randomized Controlled Trials (RCTs) are needed to be able to make evidence-based recommendations on how to increase effective patient engagement in research and innovation and assess the impact and (cost)-effectiveness.

In the United States, 42% of adults, and 81% of adults over 65 years of age live with multiple chronic condition (MCC). Current interventions to facilitate engagement in care focus primarily on the patient; however, many individuals with MCC manage and live with their conditions within the context of their family. This review sought to identify interventions used to facilitate patient and/or family engagement among adults with MCC. We adhered as closely as possible to PRISMA guidelines and conducted a systematic scoping review using a modified approach by Arksey and O'Malley. We searched PubMed, Web of Science, and Scopus using terms related to MCC, patient and family engagement, and intervention. We included articles that: (1) were published in English; (2) were peer-reviewed; (3) described an engagement intervention (with or without a comparator); and (4) targeted individuals with MCC. We abstracted data from included articles and classified them using the Multidimensional Framework for Patient and Family Engagement in Health and Health Care, and the Classification Model of Patient Engagement. We identified 21 discrete interventions. Six (29%) were classified as having the highest degree of engagement. Eighteen (85%) focused on engagement at the direct care level. Only one was specifically designed to engage families. Many engagement interventions currently exist for adults with MCC. Few of these interventions foster the highest degree of engagement; most focus on engagement at the level of direct care and do not specifically target family member involvement.

There is an increasing recognition of the importance of patient engagement and involvement in health research, specifically within the field of rheumatology. In general, researchers in this specialty appreciate the...

Engaging with patients and families is largely routine at the healthcare service or provision level. The concept of patient engagement in health research has a more recent history but is now often endorsed by leading journals and funders. Often described as research being done with patients, rather than "to" or "about" them, patient-oriented research is distinguished by patients serving as research partners rather than passive study subjects. It is generally accepted that patient engagement in health research is worthwhile and can contribute to better quality research. However, questions remain about how to do it, what values underlie it, and how patients can be engaged as partners in health research. In this chapter, we attempt to answer these questions and provide tangible examples of patient-oriented research in our province of Newfoundland and Labrador (NL), Canada. While there is no single approach to patient engagement in health research, we hope the examples provided herein help demonstrate how it might be done and assist research teams to better integrate and utilize patients' valuable input.

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.

Patient empowerment through engagement in bladder cancer research