Using a participatory approach to develop and implement the UK ME/CFS Biobank

Since the millennium, we have witnessed an increasing number of biobanks acting as key infrastructure for biomedical research [1]. Biobanks are a cost-effective and nimble means of providing high-quality samples linked to comprehensive clinical and socio-demographic participant data.

This editorial highlights the importance of biobanks for the advancement of scientific knowledge and of patient participation and engagement as drivers of success by outlining the procedures and rationale behind the development of the UK ME/CFS Biobank, which used a participatory approach.

For over 30 years, participatory community research has brought together successfully academics and potential research participants in the planning stages of projects, allowing both parties to share the power of decision-making [2] in the research process, despite a perceived threat to the academic research establishment [3]. Having its roots grounded on two distinct schools of thought, Kurt Levin's cycle of ‘fact finding, action and reflection’ was initiated in the 40's while empowering dialogical research methods originated in developing countries during the 70's [4,5,6]. The participatory research approach incorporated further contributions from feminist and post-colonialist authors, and has evolved into a framework, with the interconnected elements of participation, research, and action at its core [5].

The lack of validation and legitimisation of ME/CFS has resulted in part from an inadequate understanding of its aetiology and pathophysiology as well as the absence of sensitive and specific tests to confirm diagnosis. This in turn has hampered investigation into, and development of, effective treatments. Despite the growing number of published biomedical research studies on ME/CFS [7], advances in understanding disease mechanisms and the translation of research findings into clinical practice have been limited. The lack of coordination in ME/CFS research, of consensus on case definitions, and of attention to methodological rigour have worked together to threaten the interpretation of evidence and the comparability and generalisability of study outcomes [8,9,10].

Such poor understanding of the causality and disease mechanisms of ME/CFS has led to disagreements about its nature and management among professionals, patients, and the general population. These differences have further impeded research progress and affected the healthcare provided to people with ME/CFS (PWME), who often need to overcome significant barriers related to the recognition of their illness and its effect on their lives, as well as to access to care and effective treatment [11].

Particularly problematic have been conflicts between patients, doctors and researchers related to distinct perceptions of the nature of the condition and its management [12,13]. In the clinical context, PWME often have defined ideas about what their issues are and how they might be analysed and managed, which sometimes conflict with those held by medical professionals. Disagreements are no less relevant in ME/CFS research, in which research priorities and conducts have been challenged, and patients have been expressing great dissatisfaction with study conclusions, the lack of transparency, and the methodological rigour of some studies, as exemplified by the controversies and disagreements generated by the PACE-trial [14,15].

Participation as a way forward

With sluggish progress in ME/CFS research frustrating many patients and researchers, we sought solutions that were not only robust from technical and logistical viewpoints, but would also find acceptance among researchers, clinicians, patients and the wider ME/CFS community. We believe that tissue banks, and biobanks in particular, are essential infrastructure resources that will enable the acceleration of ME/CFS research by ensuring high-quality, standardised procedures for the collection, storage, and management of data and samples, while observing ethical, legal and social issues. The sharing of such a resource with both academics and the commercial sector represents a cost-effective way of accelerating science in this area of research.

In planning the proposed tissue bank, we considered a participatory approach to be best practice. Such an approach recognises both the complementary expertise of patients and researchers and that the ultimate beneficiaries of research are the patients themselves. To develop a resource for the study of ME/CFS with no explicit support from the community of beneficiaries would represent a significant risk of being unsuccessful or lacking purpose, and could also risk further deterioration of the relationship between patients and healthcare professionals.

Using a participatory approach, we investigated the acceptability and feasibility of developing a disease-specific biobank for the study of ME/CFS that would be cost-effective in both its implementation and operation. The biobank would aim to be technically robust and responsive to the needs of both patients and scientists. The model would be fully compliant with relevant ethical and legal frameworks and would attract the participation of both PWME and others without ME/CFS as members of control groups.

Project outline and outcomes

The consultation phase consisted of discussions with the project's Steering Committee (comprising researchers, clinicians, representatives of ME/CFS charities, and PWME and their carers, and chaired by a representative from an ME/CFS charity), focus group discussions with PWME, and a workshop with a group of experts, i.e. professionals with expertise in biobanking, ethics, clinical, epidemiology and lab-based research. This was followed by the development of the biobank proposal and its subsequent implementation [16].

We ran focus groups with PWME to identify and understand specific areas of concern that needed addressing to ensure that targeted donors were comfortable in providing biosamples with accompanying personal information for storage and use in future research. The participatory approach we used endorses the ethical principles of ‘self-determination, liberty and equity, and reflects an inherent belief in the ability of people to accurately assess strengths and needs and their right to act upon them’, as described by Leung et al [17].

During the consultation, focus group participants clearly expressed their desire for the establishment of an ME/CFS biobank focused on blood sample collection and believed it would be both feasible and cost-effective. Their recommendation was to create a disease-specific biobank of blood samples from both PWME and healthy controls, including people severely affected by the disease. The various stakeholders were aligned in highlighting the importance of using appropriate case definitions and inclusion/exclusion criteria for PWME and controls together with careful data collection methods and rigorous protocols for the collection, transport, and storage of samples. Discussions also emphasised the importance of ethical and legal issues.

The concerns of focus groups participants and their detailed recommendations, together with those given by professionals, were fully taken into account in designing a biobank protocol that was acceptable both to potential participants and the scientific community and responsive to the pressing need for high-quality ME/CFS biomedical research.

The UK ME/CFS Biobank has, as of May 2017, recruited over 500 participants, including people with a confirmed clinical diagnosis of ME/CFS and others in comparison groups of people with multiple sclerosis and ‘healthy controls’.

Lessons learned

This project resulted from a collaborative process established between the authors, UK ME/CFS charities and support groups. In our study, the ‘community’ was defined by its individuals’ shared experiences of being struck by a ‘not-well-understood-and-stigmatised’ disease, and the lack of effective therapeutic responses.

The positive impacts on the research team, such as, PWME bringing their personal knowledge and perspectives on ME/CFS, and their enthusiastic response for ‘taking part’ and ‘being listened to’, and were similar to those described by researchers involved in other participatory research projects [4, 5]. The researchers were humbled to share and learn from PWME's narratives and experiences. The challenges identified to the establishment of the disease-specific biobank and ongoing ME/CFS research included the potential constraints of those with symptoms so severe they might be unable to take part, the fear of symptom relapse because of the required blood donations, and the potential misuse of samples by researchers. We responded to those challenges with careful planning, including: (i) organising home visits for consenting participants with severe symptoms, (ii) monitoring potential symptoms post-blood donation, and (iii) developing robust research governance mechanisms, including ongoing participation of PWME and carers in decision-making around sample sharing [16].

Conclusion

The implementation of the UK ME/CFS Biobank and the successful recruitment of participants is evidence that the participatory approach has been effective. The development of the protocol that underpinned the launch of the UK ME/CFS Biobank was informed by the participation of a variety of relevant stakeholders.

Acknowledgements

The charity Action for M.E. funded the initial consultation process, including the focus groups and workshop with experts, which resulted in the proposal for the UK ME/CFS Biobank. The Biobank was established with a joint grant from the charities Action for M.E., the ME Association, and ME Research UK. All three charities have been represented on the Steering Committee that oversees the Biobank project and were involved in decision-making processes related to the biobank development; however, the researchers are solely responsible for collecting, analysing, and interpreting data and writing up manuscripts.