Patient participation in ERS guidelines and research projects: the EMBARC experience

Patients and carers are the key stakeholders in every aspect of medical care; yet, until recently, direct involvement of patients in clinical guidelines, clinical research studies or research consortia was uncommon [1]. Patients have typically been viewed as participants, e.g. subjects in a clinical trial, or involvement has been characterised as “tokenism” [1]. This is changing, as the value of patient involvement is being recognised, as physicians are being provided with the tools and guidance required to achieve meaningful patient engagement, and as research funders increasingly encourage or require patient involvement during the development of research projects [2–5].

The Value+ toolkit defines meaningful patient engagement as giving patients an active role in activities or decisions that will have consequences for the patient community, because of their specific knowledge and relevant experience as patients [6].

Despite an increase in literature regarding patient involvement in projects, one of the major barriers to patient engagement is a lack of understanding from healthcare professionals about how to do it, and a lack of support in terms of funding for patient activities and tools and advice on how to do it [7–9]. From a patient’s perspective, while involvement in clinical projects can be a very positive experience, it is important to consider barriers, such as available time, self-confidence, stigma associated with illness and financial considerations, and to design patient involvement in a way that ensures that patients are adequately supported.

In this article, we describe a successful European Respiratory Society (ERS)/European Union (EU) project focussed on bronchiectasis, in which patients have been closely involved at every stage [10]. The purpose of this article is to highlight the multiple aspects of clinical guidelines and clinical research where patients can make a positive contribution, to highlight the lessons learnt from the European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC) project in terms of how to support patient engagement, and finally to reflect upon the patients’ experience of these projects, in order to help future patients and professionals to do better.

Patient involvement in EMBARC

EMBARC was one of the first ERS Clinical Research Collaborations awarded in 2013 [10, 11]. The specific objectives of EMBARC defined in its original constitution were: to create a European Bronchiectasis Registry to facilitate research and quality improvement initiatives across different healthcare systems; to build a network of researchers and experts in bronchiectasis to guide future research and clinical priorities; to attract new researchers and clinicians to the field of bronchiectasis; to support and encourage early career researchers in the field of bronchiectasis through involvement in network activities; and to build bronchiectasis research capacity in Europe [10].

Bronchiectasis is a poorly studied “orphan” condition with limited research and a poorly defined standard of care [12–14]. Patients often have poor quality of life, and may have poor experiences of healthcare due to delayed diagnosis, ineffective treatments, lack of understanding of their condition by the general public and by non-specialist doctors, and a lack of access to quality information [15–18]. Therefore, a decision was made early in the EMBARC project to engage directly with patients to ensure that activities within the project were patient focussed and, where appropriate, patient led. EMBARC has been a multifaceted project with a range of different activities, all of which have involved patients. These are summarised in figure 1 and each is discussed in the following sections.

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Figure 1

Summary of patient involvement in the EMBARC project.

Patient involvement in the European Bronchiectasis Registry

The formation of the EMBARC registry was a major undertaking, involving over 150 centres in more than 40 countries. As bronchiectasis is a poorly researched disease, there have been limited epidemiological data and we know very little about the natural history [29–31]. The initial development of the registry required the development of a case report form and a governance structure that would allow the registry to succeed over the long term. This, in turn, needed to be focussed on the outputs that we wished to achieve through the research. To inform this, we conducted an online patient survey co-ordinated by ELF. The survey was sent to over 100 European physicians caring for bronchiectasis patients, and the online survey was ultimately completed by more than 1000 patients and carers of those with bronchiectasis, with 711 included in the final analysis [11]. The questions asked in this survey were developed by a small working group composed of three physicians, representatives of ELF and four patients [11].

The result has been published as a research priorities document in the European Respiratory Journal and this “research roadmap” defined by patients and physicians is now used as the basis for the EMBARC strategy and analysis plan [11].

The case report form of the registry was developed with input from patients and directed by the results of the patient survey. The study documents, such as the patient information sheet and consent form, were also reviewed and amended by patients [10].

The governance structure of the registry included patients, and patients have attended each of the registry steering committee meetings. The patient representatives also gave a closing address at the general assembly of the EMBARC project in 2015, and an opening address at the EMBARC general assembly in 2016 (figure 2).

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Figure 2

A group of bronchiectasis patients address the EMBARC annual meeting.

Patients have a stake in ensuring that their data are used to benefit the whole research community and, ultimately, other patients. The patient advisory group therefore played a key role in EMBARC adopting a very open and transparent data access process, which allows any research with a legitimate scientific question to access the data [32].

The EMBARC registry is ongoing and patients continue to play a key role in its governance and development as they have done since its inception.

Patient involvement in education initiatives, the ERS Annual Congress and World Bronchiectasis Conference

There is a significant need for physician education in bronchiectasis, as highlighted by the patient priorities survey, in which one of the top patient priorities was that their primary care physician should have greater knowledge and understanding of their condition [11]. Patients have been integral to the development of a physician education platform that is due for launch in 2017. In addition, patients consistently report a lack of high-quality educational material available for them to understand their own condition. Patients have therefore led the development of a patient education website to be launched by ELF in 2017 to provide high-quality peer-reviewed information about bronchiectasis targeted at the general public. These initiatives have been conducted under the same principles as the task force, with multidisciplinary working groups that include patients as equal partners. Patients also participated in the first World Bronchiectasis Conference, held in Hannover, Germany, in 2016. The conference was opened by a lecture by Marta Almagro, a patient with bronchiectasis who gave a powerful, personal presentation about the impact of the disease and the importance of encouraging greater awareness of bronchiectasis (figure 3). Patients have also attended EMBARC meetings at the past three ERS International Congresses.

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Figure 3

Marta Almagro opens the first World Bronchiectasis Conference.

Experience of patient involvement: the clinician’s perspective

In part as a result of the strong support from patients, the EMBARC project has been a major success. The European Bronchiectasis Registry is now well established with >8000 patients enrolled at the time of writing, and the project has produced a large number of important publications. Although the benefit of patient involvement is clear from the information presented here, the process presented many challenges.

The EMBARC study team had limited prior experience of patient involvement in research projects or guidelines. There was no established pathway or guidelines for how patients should be involved in research or task forces, and as a result the “rules of engagement” had to be generated by EMBARC and ELF on a bespoke basis to suit the project. There was some initial resistance to patient involvement from some quarters, because of concerns that patients may be upset by some aspects of discussion or that the presence of patients may stifle debate. As an example, if a member of a guideline task force is receiving a drug that the guideline task force wants to recommend is ineffective or potentially harmful, how would that patient react, and would this harm their relationship with their existing healthcare professional?

Additional barriers include the need for in-depth scientific discussion, which may not always be accessible for patients or carers, leading to the concern that discussions may be stifled if conversations are constantly being interrupted for lay explanations. Table 1 outlines some of the barriers and concerns and how these were addressed in this project. These can be used as a framework for future projects of this kind, while recognising that barriers may be different depending on the nature of the projects being proposed.

Physician attitudes towards patient involvement have clearly changed over the course of the project, with these concerns addressed. Clinicians and allied health professionals have commented how overwhelmingly positive the experience of patient involvement has been, and the genuine level of enthusiasm this has generated is reflected in increasing requests to involve patients in further events, committees and activities. Nevertheless, EMBARC has made plenty of mistakes along the way and it is hoped that the guidance in this document can help to highlight both the good and bad practices we have identified over the past 3 years so that future projects can learn from our experience.

Experience of patient involvement: the patient’s perspective

Patients become involved in research and guideline projects for a variety of reasons, including their own personal experiences of care and a desire to improve treatment for themselves and for others with a similar condition. Making a decision to become involved in a project like EMBARC is a major undertaking and there are a few things that can be done to make it easier for patients to volunteer to become involved [41]. The EMBARC patients’ key recommendations for patient involvement in research are presented in table 2.

We recommend that clinicians explain very clearly what they expect from patients, which may be helped by generating a job description for patient representatives. It is important to make clear at the beginning what time commitment is needed and also to let patients know if any training is required or will be provided. We recommend involving patients at an early stage, when they have the opportunity to actively influence the research, rather than later on when most of the major decisions have been made. It is also important to make sure that patients know who to contact with questions or for information.

As a result of an open set of medical professionals, the patients’ input and concerns are fully reflected in all aspects of the EMBARC project. A prime example was in agreeing the final draft of the guidelines, where patient acceptability is one of the criteria. At this stage, the healthcare professionals actively sought the input of the patient representatives. The recommendations in the 2017 ERS bronchiectasis guidelines have all been developed with patients and a lay version of the guidelines will published alongside the main document. Another important impact patients made was to request that all publications arising from EMBARC should be freely available to patients, with the effect that all publications from EMBARC have been open access since 2015.

At the start of the patient involvement, however, meetings were difficult and there was a need for clear guidance on the potential role(s) of the patient (from observer to full participant) and how the meetings could be structured to reflect the presence of patients. To many patients, these meetings present a challenge on five fronts: 1) it is a formal meeting and many patients may not attend formal meetings of any type in their day to day life; 2) the group of medical professionals are already known to each other but not to the patient; 3) the medical members may also be treating the patient away from the meeting; 4) the potential for “deep” scientific and medical discussion with associated jargon; and 5) the need to address topics such as life expectancy calculations, which can be upsetting to patients.

The biggest challenge for a patient is the first meeting. This was also highlighted from the clinicians’ perspective in terms of resistance from the clinicians, which may result from meeting a patient out of the context of a clinic visit, or from the instant access of the patient to a club that the clinicians have invested many years to gain entry into. In most cases it was clear that this was the clinician’s first experience of involving patients in research and clinical projects.

Each patient will be different in their experience of meetings, their subject matter knowledge, how outgoing they are, and their financial circumstance. An underestimated impact of involvement in research projects is the impact that medical data can have on patients. For example, several patients involved in the EMBARC project are chronically infected with Pseudomonas aeruginosa. Across multiple discussions in both research and guideline meetings, it became clear that the physicians regard P. aeruginosa as a poor prognostic sign, a sign of increased mortality and more rapid disease progression requiring more intensive treatment [38, 39, 42]. Hearing this can be very upsetting for a patient who knows that they are infected with P. aeruginosa but who may not have been previously made aware of its health consequences. The “clinical” tone of these discussions was described by one patient as “brutal”. This is not a scenario unique to bronchiectasis, as the reaction would no doubt be similar for a patient with lung cancer on hearing lung cancer 5-year mortality rates, or a patient with sleep apnoea learning about their cardiovascular risks, if they are not warned beforehand that such information will be discussed [43, 44]. It is important to inform patients that aggregated research data show effects across large populations, and do not necessarily have implications for them personally. It is important to emphasise that all patients are different and have different needs for treatment. To give another example, physicians will often discuss that a therapy “has no evidence to support it” or “does not work” when the patients attending the meeting are receiving that therapy. The patient’s natural reaction is to question whether they should be taking the therapy, and whether their doctor may have made a wrong decision in prescribing it. One patient in the bronchiectasis guidelines task force was receiving a therapy that the panel determined should not be used due to an increase in side-effects in clinical trials. After discussion, it was very clear that the patient had benefited from this treatment. It is important to understand that research and guideline projects look at the balance of risks and benefits across a population, and do not determine whether an individual patient will benefit from a treatment. This distinction is not immediately obvious unless clearly explained to patients. Patients told us that they would prefer to have a single point of contact with whom they can discuss issues as they arise, and from whom they can obtain information. This can be a member of the panel, or someone from a patient organisation such as ELF.

The European Lung Foundation perspective

ELF was founded by the ERS to bring together patients and the public with respiratory professionals to positively influence lung health. ELF encourages healthcare professionals to engage with patients and patient organisation representatives to ensure that patient perspectives are at the centre of their project set-up, decision making and outcomes.

Over the last 5 years, ELF’s remit has expanded significantly as its role has become better known within the ERS, resulting in an increasing number of ERS project chairs engaging with ELF for support in facilitating patient input into their activities. This has resulted in a steep learning curve for ELF and, with increasing experience in this area, and building on the facilitation of patient input into over 15 ERS task forces and projects to date, means that we are continually looking to review and refine our approach.

Our experience of working with EMBARC, which has been a very successful collaboration, forms the basis for the challenges and learning points we have identified for this article and which are outlined in table 3. Many of the recommendations and do’s and don’ts outlined in table 2 also give us an opportunity to improve our ways of working to ensure smoother participation and involvement for patients in the future. Some of the challenges identified here have been responded to already and solutions have been implemented.

Table 3 shows the key challenges we faced specifically in working with EMBARC on their multiple projects and we have illustrated the solutions that have already been implemented in response to these, as well as future recommendations.

One of the solutions we have been able to put in place over the life of the task force project has been to refine our patient input process, which is used as the basis for initial discussions with project leaders and chairs to define the appropriate elements of patient involvement to include within each project. We continually review our approach and ways of working to ensure that we can build on feedback received and adapt where necessary to ensure quality patient input.

Disclosures