Abstract
Objective. Since 2002, 58 patients have participated as collaborating partners in 6 Outcome Measures in Rheumatology (OMERACT) conferences. Little is known about how they engage with researchers and how they have influenced conference outcomes.
Methods. A responsive evaluation was carried out, including a thematic document analysis of conference proceedings and gray literature, participant observation, and 38 interviews with patients and professionals representing research, industry, and regulators. Interview transcripts were subjected to an inductive content analysis.
Results. The role of patients has evolved from a single focus group in 2002 to full integration in all parts of the conference in 2012. Longterm engagement has made a significant change in the scope and conduct of rheumatology research. It has enriched the research agenda by identifying previously neglected outcome domains such as fatigue, sleep disturbances, and flares, and it has contributed to more patient-relevant outcomes in clinical trials. Facilitating factors have been a strong commitment of the leadership, adequate selection procedure, inclusive conference design, interactive and encouraging moderation style, and self-organized support. The intensity of the program and doubts regarding the representativeness of the patient group were still seen as challenges for the future.
Conclusion. Making patient participation an integral part of the vision and procedures of OMERACT has significantly contributed to the success of OMERACT. It has changed the perceptions and beliefs of many participants. Full use of patients’ experiential knowledge before and during the conference is still challenging.
- OMERACT
- OUTCOME RESEARCH
- PATIENT PARTICIPATION
- PATIENT RESEARCH PARTNER
- RHEUMATOLOGY RESEARCH
- USER INVOLVEMENT
There is a growing interest in actively engaging with patients in the context of scientific research1,2. Involving patients in the development of patient-reported outcomes (PRO) ensures their relevance, acceptability, and quality2,3,4. OMERACT (Outcome Measures in Rheumatology) has been a pioneer in the field of rheumatology in organizing longterm involvement of patients as collaborating partners5,6,7,8 in its biannual, worldwide conferences. Since 2002, 58 patients with different rheumatic conditions have participated in 6 conferences with the purpose of providing the patient perspective9. Their involvement has been reported as advantageous, a “success,”10 and “the beginning of a paradigm shift”11. In 2011, OMERACT formalized its policy toward patient participation, recognizing the essential role of patients in outcome research12. Patients’ input is indispensable when identifying domains that are important from the perspective of patients and when assessing feasibility of measurement tools; moreover, it provides face validity to the OMERACT process. It also enables OMERACT to ground theoretical discussions “in the lived experience of arthritis, and in concepts which can be readily communicated to patients to help with therapeutic decision making”12.
Despite a widespread belief among experienced OMERACT delegates that patient participation is worthwhile, robust evidence for its effectiveness is lacking. Little is known in general about how patients engage with researchers, the specific features that promote collaborative research, and the overall effect and consequences of patient involvement. OMERACT, because of its long history of patient participation, provides a unique opportunity to explore these issues by analyzing the effect of patient participation on the nature of its research activities. In addition, the inhibiting and facilitating conditions for this process can be elucidated.
This article contains the preliminary findings of a PhD project as presented by the first author during OMERACT 11 (2012). We describe the combined results of a thematic document analysis of conference proceedings and 38 interviews during OMERACT 10 (May 2010, Malaysia) based on 3 empirical substudies12a,12b,12c.
OMERACT 10 took place over 6 days at a residential conference center and focused on 4 diseases — rheumatoid arthritis (RA), psoriatic arthritis (PsA), ankylosing spondylitis and gout — and on the methodology of choosing domains of interest and instruments appropriate to measuring these domains. As in previous meetings, the program consisted of a mixture of plenary sessions, intense small group discussions, and toward the end, formal presentations and proposals that were voted on to achieve international consensus13. Twenty of the 200 places were allocated to patients.
MATERIALS AND METHODS
To assess the process and effect of patient involvement in OMERACT, a responsive evaluation14,15 was carried out, starting with a thematic document analysis including scientific literature on patient participation, OMERACT conference proceedings, and gray literature such as correspondence, invitations, session reports, and policy documents. The review focused on the reception and evolution of patient involvement in OMERACT conferences and the contributions made by patients.
Data collection was based on 38 semistructured interviews (Table 1) before, during, and after OMERACT 10 (Malaysia, 2010), and included researchers (n = 12), representatives of the pharmacological industry, regulators, and staff (n = 4), experienced patients (n = 8), and new patients (n = 8). To gain insights into changing perceptions, 3 new patients were interviewed 3 times: before the start of the conference; on the third day; and immediately after the end. Finally the opinions and experiences of 16 professionals and 16 patients were collected (Table 2).
Summary of the interview protocols for new patients, experienced patients, and researchers.
Characteristics of interviewees.
Selection of interviewees aimed at maximum variation and followed an emergent purposive sampling approach taking into account background, opinion about patient involvement, sex, geographical spread, and number of OMERACT conferences attended. A preliminary analysis of the first data revealed an unexpected response shift. The attitude toward patient involvement of 2 professionals had changed from presumed skeptic to supportive. For this reason we identified 2 new interviewees who were known for their critical perceptions. After analyzing the additional transcripts, saturation was achieved.
Broad interview protocols were developed16 (Table 1) based on the document analysis, 4 pilot interviews, and the first author’s personal experience during 3 OMERACT conferences. The protocols differed for professionals, new patients, and experienced patients. The interviews were recorded, transcribed, and subjected to a responder check. One interview took place without protocol and, on request of the interviewee, without recording. The average duration of the interviews was about 50 min. Finally, participant observation took place during 6 patient meetings by the first author.
Interview transcripts were subjected to an inductive content analysis17. Coding of all interviews was done separately by the first author and an independent health researcher with extensive experience in qualitative research (MK). To increase the relevance and validity of the data analysis, one of the patients who attended OMERACT 10 for the first time (SC) joined the research team. Codes were compared and discussed during several face-to-face meetings, resulting in sets of main categories. To reduce the risk of observer bias18,19, all authors, representing different backgrounds, randomly cross-checked the coding and confirmed that there were no significant discordances. Triangulation took place by synthesizing data derived from document analysis, interviews, and participant observation.
RESULTS
Influence of Patient Participation
Widening the research agenda
The main findings are shown in Table 3. There was broad consensus about the significant influence of patients on the research agenda by identifying previously neglected outcome domains such as sleep disturbances, flares, and particularly fatigue20,21,22,23. Increased research in the domain of fatigue has been reported unanimously as the most illustrative result of this process, which would not have occurred without the structural involvement of patients being listened to by receptive researchers. It has led to extensive studies into the nature, influence, and measurement of fatigue in RA24,25,26,27,28. In 2006, fatigue was added to the RA core set as a recommended outcome for clinical trials29,30. More powerful instruments for measuring fatigue have been devised and validated31,32 with the active involvement of patients33. The issue of fatigue was not new for rheumatologists34,35,36. During OMERACT 3 (1996), delegates carried out a ranking exercise where fatigue was already identified as a major concern for patients37. Eight measurement instruments were given for fatigue38. However, after this workshop, there were no other developments for 6 years. Retrospectively, professionals admitted they had a blind spot for fatigue in RA and only hearing about it from patients at OMERACT 6 made them change their perception of fatigue as an important outcome.
Main findings of the study.
Core sets and outcome measures
Patients have identified domains that are relevant for disease-specific core sets for PsA, fibromyalgia, gout, and vasculitis. Further, they contributed to the development of core sets for clinical concepts such as minimum clinically important difference and remission, and played an important role in the assessment of the feasibility of core sets and outcome measures, one of the key components of the OMERACT filter39. Patients have also been helpful in the development of PRO in the field of work productivity, adverse events, flares, and psychosocial interventions.
Changing perspectives
Longterm engagement with patients has changed the culture of OMERACT and the perceptions of its participants. Initially some researchers were concerned about inviting patients to the conference. They feared that patients were not able to transcend their personal experience, to generalize, and to understand the rigor of the methodological discussions. After a while, when patients started to organize themselves and learned to apply OMERACT procedures, researchers became less reserved and started to see the benefits. They confirmed that it changed their way of thinking and talking. Patients improved communication and brought dynamics to the dialogue because they were motivated, constructive, and had no personal agenda. When discussions became extremely technical, patients reminded participants of the common goal of the conference by providing the human face of a person living with the condition day by day. Their presence made participants more explicit about the objectives of sessions and more explanatory about terms and concepts under discussion. Together with a reduced use of jargon, this “forced” simplification resulted in fewer misunderstandings for everyone.
Patients attending OMERACT for the first time reported significant learning curves and a variety of personal benefits. In fact all participants learned from the contact with other parties. During this process participants gained trust, respect, and understanding. One interviewee mentioned “a reality check” as an important benefit. For professionals, patient participation offered the opportunity to check the relevance and scope of their research: are we doing the right things according to patients and are we using the right tools and methods?
Consequences outside OMERACT
Patient involvement in OMERACT has stimulated more patient-oriented health research in many countries. Patients returning home after the conference have introduced the concept of participatory research in local projects or established networks of patient research partners40,41,42,43. With the input from several OMERACT participants, the European League Against Rheumatism developed recommendations for patient involvement in scientific projects6. Inspired by the experiences of OMERACT, the organizing committee of the 6th International Shared Decision Making conference decided in 2011 to officially invite and facilitate for the first time 4 patient participants44. In the same year, OMERACT delegates, patients as well as researchers, participated in the 2nd Core Outcome Measures in Effectiveness Trials (COMET) conference, demonstrating how the OMERACT methodology can be used in other disease areas45.
Facilitators
Leadership
The patient role evolved from a single focus group in 2002 to full participation in all parts of the conference in 2012. The longterm commitment of the OMERACT organizing committee has been important for this process. The organizers were intrinsically motivated to foster patient participation in the conference because they believed that patient input is essential to decide on the right measurement tools46. The decision to invite patients was not taken lightly, but once OMERACT leaders were determined, they wanted to do it properly. They provided full support to their designated patient participation leader and guaranteed funds for patients to attend the conference. The positive contributions made by patients were regularly acknowledged and stimulated OMERACT in 2006 to formulate basic principles for patient participation. This internal policy document represented a milestone by making patient participation part of the vision of OMERACT, embedded in a set of operational procedures. In 2011 OMERACT decided on the principle that active involvement of patients would be a prerequisite for working groups suggesting themes in the OMERACT program12.
Patient selection
Since 2004 OMERACT has aimed for 10% of conference participants to be patients with a mixture of conditions, geographical spread, and OMERACT experience. From the start it was clear that patients were expected not to represent any advocacy group or organization. Although they might be active in patient organizations, they should adopt a strict individual role at OMERACT. To avoid self-selection, recruitment is done through the clinics of participating physicians, who proved to be ideally positioned to identify eligible patients who are able to make a valuable contribution12. A potential disadvantage of this is the possibility that patients feel unable to decline an invitation to please their consultant or to prevent jeopardizing their care.
Conference design
Constructive engagement with patients was enhanced by the small size of the conference, an open mind among delegates, and the spirit of OMERACT as a consensus-oriented initiative based on the involvement of many parties. From the start, patients have felt “part of the family”47. The conference layout and the focus on active interaction and debate in small groups10 have been facilitators for accepting and incorporating new perspectives. Some of the patients, all of whom were officially invited as full delegates with voting rights and access to all sessions, felt equal to professionals and acted as co-researchers. A majority of the patients saw their role as giving information or as an adviser. In particular, new partners initially felt the same unequal relationship toward professionals as is experienced in clinical practice. The interviews revealed that new researchers and research fellows sometimes also experienced these disparities. However, participants who had attended several conferences confirmed that inequalities disappear over time.
Support
According to the organizers of OMERACT, the first conference with patient involvement was the easiest and most successful because there were no expectations. Partners formed a homogeneous group and the program was not demanding. Although support was limited, patients felt welcome and accepted47. During subsequent conferences partners were extremely motivated to learn the OMERACT way and started to organize themselves. They formed a patient liaison group and developed introduction packs, information modules to bring newcomers up to speed, and a glossary in lay language48. In 2010 new patients were matched with an experienced patient who took on the role of a buddy. To prevent overburdening, personalized programs were developed that helped patients to pace themselves and to prioritize sessions to attend. Finally, patients have insisted on being involved in the preconference work, saying they felt that early participation in working groups will best prepare them for worthwhile participation during the conference.
Facilitative style
The breakout discussion sessions form the heart of OMERACT, and the quality of the moderation emerged as of utmost importance for patients to contribute to their full potential. From the perspective of patients, a style of moderation, both interactive and encouraging, that fosters mutual learning through open dialogue was seen as an important facilitator of confidence and greater input. Sometimes patients were confronted with an apparently patronizing attitude of a moderator by whom they felt ignored or disqualified. Language was an underestimated barrier in discussion groups for those whose first language is not English. Some patients tended to hold back when they were afraid of not using the right words or expressions.
Barriers
Overburdening
OMERACT was an overwhelming experience for all patients attending the conference for the first time. Before the conference they felt privileged to be invited but also insufficiently prepared and uncertain about what was expected from them. They anticipated a learning experience and hoped to be able to make a difference. In fact most experienced the conference program as physically and mentally challenging. They had to adjust to the use of medical jargon and scientific terminology, and they were not always familiar with the rules for clinical research. The (intercontinental) travel, the early and late starting times of sessions, and the duration of the conference increased the risk of overburdening. In particular, new patients often participated beyond their physical capacities. Dealing with hierarchical power relations and strongly opinionated professionals was experienced as mentally challenging. A recurring barrier reported by patients was a lack of feedback on provided contributions. At times they felt that their experiential knowledge was not accepted as a valid source for scientific research, nor seen as relevant compared to the expert knowledge of professionals. Experienced patients coped better with the requirements of the conference. After the conference almost all patients described their participation as having been a valuable learning experience. Although new patients presumed that they had not been very productive, they expected their contribution would be more effective at future conferences. Experienced patients as well as researchers confirmed that expectation.
Skepticism
In 2010 a few respondents said they believed that the disadvantages of patient participation outweighed the benefits. They judged the influence of partners out of balance and feared less interest in solving methodology challenges leading to a less diverse research agenda. They said they felt forced to engage with patients in areas of research where they saw little added value, such as imaging and biomarkers. Most of the patients confirmed this opinion and said that they thought they could offer little benefit to domains that were distant from their daily life. They said they believed that they could contribute most to research focused on their own condition and were uncertain about providing meaningful input during sessions dedicated to other conditions. To a great extent, physicians agreed with this.
Lack of diversity
Institutionalization of patient participation gave rise to a debate regarding the representativeness of the patient group. OMERACT encouraged patients to provide the “naive” dimension of being familiar with the disease although there has been a tendency to raise the selection criteria to recruit better-educated patients. A small number of respondents objected to the participation of patients who did not understand the rigor of scientific research and slowed down the process, thus causing irritation during breakout sessions. They argued that the patient perspective should be obtained during the preconference research work. Other participants emphasized the irreplaceable value of new patients bringing in authentic experiences, crucial for generating new ideas. They reported potential risks of patients becoming too professional, identifying themselves with researchers, and finally losing touch with their peers. Some respondents warned that patients who started acting as advocates might compromise the outcomes of OMERACT as a data-driven conference.
DISCUSSION
Our study has proven that building patient participation structurally into the OMERACT conference program has guaranteed that themes that are important for patients were not only added to the research agenda, but increased a sense of urgency and prompted sustainable collaboration in new research programs. From the example of fatigue we learn that it takes more than a decade to identify a new domain of interest for patients, develop validated outcome measures, and design intervention trials that target that new domain as a primary outcome. Patient involvement in OMERACT has enriched the research agenda and changed the perceptions of all those involved. Thus patient participation in a research conference is effective and complementary to other structured and deliberate procedures for agenda setting used in other disease areas49. In research agenda setting, various models and strategies have been developed with the help of different methods50; these may vary from more to less structured approaches, or from one-time consultation to regular advice from beginning to end. We see similarities between OMERACT and the dialogue model for agenda setting with patients, developed in The Netherlands: a stepwise approach for articulating patients’ priorities in homogeneous groups followed by a dialogue with other people involved51,52,53,54,55. The dialogue model strives toward equal partnerships among patients and researchers in which control over the process is shared. In taking part in a dialogue, participants listen, try to understand each other, and find a common ground56. Participants engage in a mutual learning process leading to a new perspective that is acceptable and recognizable for all involved57. The strength of OMERACT has been the vision that patient participation is an integral part of the conference that requires extensive support and continuous reflection by all.
An important additional finding of our study is that patients and researchers said they feel patients’ knowledge and competences could be even better used. Based on the results of our study and using the 5 categories of the FIRST model (facilitate, identify, respect, support, and train5), we have produced a list of recommendations that should be taken forward for consideration during future OMERACT conferences (Table 4). We summarize the remaining challenges for OMERACT as: (1) to study how experiential knowledge can be better incorporated in the discussions during breakout sessions. It is known from the literature that patients’ views are often excluded unintentionally4,58, and OMERACT should develop more effective inclusion strategies; (2) to facilitate patient participation in working groups that meet between conferences, to better prepare patients for their attendance at the conference; (3) to identify type and phases of research that are likely to benefit more from full involvement of patients; (4) to start reporting on the benefits and limitations of patient involvement; and (5) to guarantee a better representation of patients by inviting patients from continents that are currently absent.
Recommendations for improving patient participation at future OMERACT conferences structured according to the FIRST model5.
Although we refer to examples outside OMERACT where conference organizers have taken notice of the way OMERACT has organized structural involvement of patients, the transferability of these recommendations may be limited to the specific context in which the data have been collected. They have been formulated by the authors and have not gone through a thorough consensus process. This should be taken forward during future OMERACT conferences. Further, based on the contingency of our findings, we cannot provide a simple template for patient participation in all conferences. Nevertheless, there may be lessons that other researchers can apply to the process of developing patient involvement.
Acknowledgment
The authors thank all OMERACT delegates who have given their unconditional support and open participation during the interviews.
