The specialty of rheumatology takes care of people with disabling and long-term musculoskeletal conditions. For these patients, good healthcare requires the establishment of a sustainable partnership with healthcare professionals. For over two decades, rheumatology has been a frontrunner in piloting and implementing new kinds of partnerships in scientific research. In this viewpoint paper, we provide evidence for the leading role of rheumatology in developing strategies for engaging patients in research agenda setting, outcome research, developing treatment recommendations, assessing grant applications, conducting patient-centred research and transferring knowledge from research into practice. Experiences and lessons learnt in rheumatology are regularly published and are currently widely adapted and implemented in other specialties and research contexts. Challenges still exist and it is expected that rheumatology, as a leading discipline in this field, may further enhance our knowledge, expertise and understanding of the conditions for relational empowerment and meaningful patient involvement.
- patient involvement
- Patient Research Partner
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The specialty of rheumatology takes care of people with disabling and long-term musculoskeletal conditions. Good healthcare for these people requires the establishment of sustainable multidisciplinary partnerships between patients and healthcare professionals. This may explain the prominent place of concepts such as self-management, shared decision-making and multidisciplinary team care in many treatment guidelines for rheumatic diseases. Patients need to be involved in their own treatment because perspectives, knowledge and values of patients and healthcare professionals differ.1 Patients have personal, unique knowledge about the impact of disease and healthcare services and this experiential knowledge is indispensable for making individual treatment plans, but it is also a valid source of knowledge for educating health professionals2 and designing and conducting scientific research.3
For over two decades, rheumatology has taken a leading role in piloting and implementing new kinds of partnerships in research initiatives (table 1). Traditionally, the patients’ perspective is captured through the participation of patients as study participants or through consultative methods. However, there is growing evidence that combining consultation with the active involvement of patients as collaborative partners has substantial benefits for the quality, acceptability and impact of studies. Much of this evidence has been obtained in the field of rheumatology.
The aim of this viewpoint article is to outline the leading role of rheumatology in developing strategies to engage patients in research agenda setting, outcome research, developing treatment recommendations, assessing grant applications, conducting patient-centred research and promoting knowledge transfer, and to advocate continuing progress in these areas.
Patient Research Partners
Patient Research Partners (PRP) contribute their experiential knowledge in research projects by actively collaborating with researchers as equal stakeholders.4 They have obtained the competence to compartmentalise their roles as a patient in the clinic and an equal colleague in the research team. However, their input goes beyond the individual patient perspective, based on introspection and the shared reflection of illness experiences. PRPs provide valuable expertise in the development of patient-reported outcome (PRO) instruments, strategies for consulting patients during different stages of research, proposing ways to include difficult-to-reach target audiences, assistance in analysing and interpreting research findings and then making sure that the results become available and accessible for patients and members of the public.
One of the first organisations that started to involve patients was Outcome Measures in Rheumatology (OMERACT).5 In 2002, OMERACT facilitated 11 patients to participate in their biennial meeting to develop Core Outcome Sets for clinical trials. Inspired by experiences of Bristol University (UK) and based on the positive outcomes of this first meeting with patient delegates, OMERACT became a pioneer in building sustainable partnerships with patients.6 Several manuscripts evaluate the conditions for achieving these successful partnerships7 and provided evidence for the impact of patient involvement in research practices.8 A clear example is the story of fatigue which was reported by patients as an often neglected symptom in clinical practice and then recognised by OMERACT as an under-researched domain. A consecutive series of studies explored its severity and impact on daily life of people with RA9 and prompted the development of new disease-specific PRO measures such as the Bristol Rheumatoid Arthritis Fatigue score.10 Ultimately, in 2006, OMERACT concluded there was sufficient evidence to add fatigue as a mandatory outcome to the rheumatoid arthritis (RA) core set.11 A responsive evaluation of 10 years of patient involvement in OMERACT showed that a majority of OMERACT delegates confirmed that this would not have happened without the participation and persistence of PRPs who kept reminding researchers of the importance of fatigue.8
European League Against Rheumatism (EULAR) is an umbrella organisation that fosters the collaboration of patient representatives, health professionals and rheumatologists in all its activities. This inclusivity is reflected in the governance structure of the organisation12 and makes EULAR different from many other international medical societies by incorporating the perspectives of people with a musculoskeletal condition in all its endeavours. It encourages the direct dialogue between the medical professionals and patient representatives in the standing committee structure, the EULAR congress and EULAR task forces and study groups. The involvement of patients is perhaps most tangible in the development of its international management recommendations for rheumatic diseases. According to its standardised operational procedures, involvement of PRPs is pivotal in every phase of the development project.13 To provide guidance to PRPs and task force leaders, EULAR commissioned in 2010 the development of a set of practical recommendations14 that are broadly implemented in EULAR scientific projects and widely followed by national and international research consortia.
These recommendations have led to the establishment of a EULAR network of motivated and competent PRPs that currently comprises 59 persons with a variety of musculoskeletal conditions and representing all regions of Europe. Ongoing education is provided through the People with Arthritis/Rheumatism in Europe classroom of the EULAR School of Rheumatology. Between 2014 and 2018, ten PRPs followed the extensive online course about the innovative medicine development process,15 provided by the European Patients’ Academy.16 This education enables PRPs to provide high-quality input regarding the patient perspective in international fora such as the European Union, European Medicines Agency (EMA) and Innovative Medicine Initiative as well as to third parties such as pharmaceutical companies.
The Bristol disease-specific fatigue questionnaire was simultaneously published with a comprehensive description of the different methods of capturing the full lived experience of fatigue of people with RA.17 By providing a strategy for generating input from patients and PRPs, they established a solid methodology for patient involvement in PRO development. PRPs were involved in decisions regarding design of interview and focus group protocols, analysis of transcripts, draft language of items and also the dissemination of findings through coauthorship. Comprehension of items was checked for through 15 cognitive interviews. This strategy of patient involvement ensures that PROs are grounded in patient data, have face and content validity and are comprehensive.
Another illustrative example of full PRP participation in accordance with the EULAR recommendations has been the development of two patient-derived impacts of disease scores for rheumatoid arthritis18 and psoriatic arthritis.19 The process of engaging patients in the elaboration and validation of these composite indices has been described in detail.20
Since the establishment of the Patient-Centred Outcomes Research Institute in the USA, two patient-powered research networks in the field of rheumatology were funded. One project explored tools to identify and incorporate the patient voice in PROs of four different diseases, including RA and vasculitis.21 Creaky Joints, a patient organisation founded by a person with RA, was one of the collaborating partners. Through surveys and qualitative research, patients were asked to provide feedback on feasibility and personal and clinical relevance of PROs in making health decisions. Creaky Joints also became a successful applicant of the second project with the objective of developing strong partnerships with trialists to conduct patient-centred comparative effectiveness research.22
Research agenda setting
Although there is relatively less experience with research agenda setting in rheumatology, there have, and continue to be, several efforts to involve patients in this process. A Scandinavian study23 explored the research priorities of people with rheumatic diseases and found that new pharmacological treatments, physical exercise and management of pain and fatigue are among the most important outcomes for research on treatment and disease impact. The medical charity versus Arthritis (UK) has consistently sought to incorporate the perspectives and views of patients into its research agenda and priority funding setting. On World Arthritis Day 2017, EULAR presented the RheumaMap at the European Parliament,24 a document to support the prioritisation of topics and unmet needs for research applications.
Reviewing grant applications is an effective way for patients to influence research agendas. From its inception, the Foundation for Research in Rheumatology involves PRPs in its governing bodies and in the review of research proposals. Members of the Consumer Advisory Group (CAG) of the Australian and New Zealand Musculoskeletal Clinical Trials network operate as PRPs and review all trials from a patient’s perspective. Clinical trialists need to respond to their feedback before they are endorsed by the network. The chair of the CAG sits on the Steering Committee and presents each year at the annual forum for collaborators.
The Cochrane Musculoskeletal Consumer Group was among the first groups in Cochrane to get involved in dissemination of research evidence.25 Their purpose is to facilitate the use of research findings in decision-making, although their impact since its inception is much wider. The members have promoted access to summaries of high-quality systematic reviews, and they stimulated the development of relationships with fellow patients to enhance collaborative research all over the world.
The Assessment of SpondyloArthritis international Society published in 2009 a lay summary of treatment recommendations that was developed with and for people with ankylosing spondylitis.26 Since 2012, EULAR develops public summaries of all its management recommendations that are freely available on their website. These summaries are produced with the active involvement of PRPs who take responsibility for checking relevance, comprehension and completeness. They also assist in reviewing summaries of scientific publications of the Annals of Rheumatology. Finally, three PRPs wrote a lay version of the GRAPPA treatment recommendations for psoriatic arthritis.27
There is a fast growing number of PRP networks in rheumatology that strengthen the quality and success of local, national and international research initiatives. They benefit from published recommendations for patient–researcher partnerships that were developed and applied first in the specialty of rheumatology.14 28 An important recommendation relates to acknowledgement through coauthorship of peer-reviewed manuscript. Since PRPs started to collaborate with researchers, over 500 publications incorporated their name as coauthors, representing more than 200 individual persons with a rheumatic condition. Coauthorship is a recognition of the contributions made by patients, and is the ultimate proof of equal and meaningful partnerships.
This overview demonstrates that the specialty of rheumatology has been a pioneer in implementing strategies of patient involvement in health research and that the experiences gained offer a springboard for many others. There are some reasons that may explain this leading role. First, rheumatic diseases, unlike many other diseases, tend to be long term, disabling and requiring an intense relationship between patient and clinician to achieve the best health outcomes.12 Therefore, collaboration in the consultation room is pivotal. In addition, certainly 20 years ago, treatment options were still limited and while arthritis patient organisation started to grow and became more professional, they watched the developments in adjacent disease areas (such as HIV and cancer) and chose less aggressive forms of advocacy seeking partnership rather than confrontation.3 Their strategy was based on the principles they were familiar with: trust, collaboration, partnership and sustainability. After the introduction of biological drugs, more funding for research became available that additionally facilitated the inclusion of a strong patient perspective in research. Finally, we should not underestimate the role of individual researchers and patients who became trailblazers in the field.
Major challenges still exist and we are confident that they can be solved. Over the years, we have learnt that facilitators of successful and sustainable collaboration have been transparent procedures for decision-making, equal partnerships on all levels, support and education of PRPs, trust and mutual respect for the competences and limitation of other stakeholders, and the willingness to learn from each other. These facilitators are key features of the process of relational empowerment, a dynamic process during which the quality and strength of the collaboration increase as the result of intertwining learning curves. Relational empowerment occurs when people enter into a direct dialogue, not once, but over a sustained period. In this situation, a learning process arises in which one gets to know each other’s interests and in which the individual perspectives gradually melt into a new, shared perspective. The result is mutual understanding for the other. This process is described by the German philosopher Hans-George Gadamer as a 'fusion of horizons’,29 something that can regularly be seen in rheumatology research practices. We believe that this has been a feature of the pioneering role that the specialty of rheumatology has historically played in patient involvement in research, and that this process is continuing within the specialty. As the role of PRPs has become more diverse, new challenges have emerged which are now at the forefront of the current research agenda (table 2).
We hope that the specialty of rheumatology will retain its enviable leading role in patient and researcher collaborative research in the future, and will remain an exemplar to many other specialties.
Handling editor Josef S Smolen
Contributors Both authors were equally involved in the conception, writing and reviewing of this Viewpoint article.
Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests MdW has been the EULAR vice president representing People with Arthritis/Rheumatism in Europe, is an Executive Committee member of OMERACT and a Patient Research Partner in several scientific initiatives among which GRAPPA is one. MdW is a volunteer and board member of Stichting Tools. AA is a UK NIHR INVOLVE associate, having previously been a UK NIHR INVOLVE member and board member. AA is also the Patient and Public Involvement strategic lead for the UK Yorkshire and Humber CLAHRC.
Patient consent for publication Not required.
Provenance and peer review Not commissioned; externally peer reviewed.