Evaluation of the quality of patient involvement in a patient-led analysis of the lived experience of a rare disease
Research Involvement and Engagement volume 9, Article number: 35 (2023)
Patients themselves are best placed to provide insights on the lived experience and to lead the analysis of such insights to bring the patient voice into peer-reviewed literature. In doing so, they can meet the authorship criteria for subsequent research publications. It is important to evaluate patient engagement to identify ways to improve future collaborations. Here, we describe the approach taken during a patient-led and patient co-authored analysis of the lived experience of generalized myasthenia gravis, which may be applicable to other indications. We also assessed the quality of patient engagement throughout the research project.
We used self-reported experience surveys based on the Patient Focused Medicines Development Patient Engagement Quality Guidance criteria for assessing patient engagement. The surveys were adapted to focus on individual projects and assessed eight domains using a five-point Likert scale. In September 2020, we invited eight patient council members to complete a self-reported experience survey following qualitative lived experience data generation. We calculated the average experience score as a percentage of the maximum possible score. Patient authors (n = 1) and non-patient authors (n = 3) were invited to complete a similar survey in November 2021, with questions customized for relevance, to evaluate the authorship experience following publication of the research.
Overall, patient council members had a positive experience of taking part in this study, with an average experience score of 90% (71.6/80.0; n = 8). The patient author and non-patient authors rated their authorship experience highly, with average experience scores of 92% (78.0/85.0) and 97% (63.3/65.0), respectively. There were key aspects that contributed to the overall project success (e.g., ensuring that all participants were aligned on the project objectives at the outset and understood their roles and responsibilities). We also identified elements of the approach that could be improved in future collaborations.
In this patient-led analysis, patient council members, patient authors and non-patient authors had a positive experience of being involved in the project. We gained useful insights into elements that contributed to the project’s success and ways to improve future patient-led projects on the lived experience.
Plain English summary
Involving patients in research is becoming more common. Listening to patients can help healthcare teams to better understand the impact of living with a condition. Some patients taking part in research can go on to be an author of an article that describes the findings. We describe how we assessed the quality of patient involvement in a research project to better understand views of patients living with a rare condition.AbstractSection How did we assess patient involvement?
We asked patients to complete surveys to find out about their experience of taking part in a patient-led analysis of a rare condition. This can highlight challenges that they may have faced during the project.AbstractSection What were the main findings?
Patients who took part in this research and those who were authors of the research article had a positive experience. There were several key factors that helped the project to be a success. We also identified ways to improve future projects designed to gain patient insights on living with other conditions.
The importance of involving patients throughout the drug development life cycle is increasingly recognized by multiple stakeholders, including patients [1, 2]. In particular, the benefits of partnering with patients to gain unique insights into their perspectives of living with a condition are well-recognized, especially in rare diseases [3, 4]. Patient-reported outcomes instruments provide useful information on the impact of living with a condition on people’s quality of life, but they do not provide the in-depth understanding gained from learning about the lived experience. Patient insights can help healthcare professionals better understand the unmet needs of patients, which may facilitate shared decision-making [5,6,7]. Importantly, seeking insights from patients themselves encourages patients to actively participate in all aspects of their condition, including advocacy, and draws attention to priorities for their own care . Gaining insights can also empower patients in the knowledge that their lived experience is important and valuable.
Not only are patients themselves best placed to provide insights into their own condition, but they are also best placed to analyze and interpret insights from broader patient populations. Such patient-led research can in turn provide patients with an opportunity to meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship so they go on to author peer-reviewed publications describing the lived experience. However, there are perceived barriers to patient authorship. For example, a recent survey highlighted that only 3.6% of editors-in-chief (4 of 110) have policies regarding patient authorship . Potential risks of including patients as authors have been identified (e.g., increased resource needs and opportunity costs, imbalance of power dynamics in the authorship group and risk of nonrepresentative insights from patient groups that are less diverse than the wider patient population) . Despite these barriers, the number of patients authoring their research is increasing, driven by growing interest from patients themselves and recognition by major funders who support patient authorship .
Our patient-authored, peer-reviewed publication describes an analysis of the lived experience of a rare neuromuscular disease, myasthenia gravis (MG) . MG is a rare, chronic and unpredictable autoimmune disease with a prevalence of around 1–2 per 10,000 people [13, 14]. Clinical symptoms of MG include muscle weakness and fatigue [15, 16], which can be restricted to certain muscle groups, such as the eyes (ocular myasthenia) or involve multiple muscle groups (generalized MG). Many people experience central fatigue (physical or mental) and may have comorbidities that impact their quality of life [15, 17, 18]. Importantly, people with MG may have symptoms that fluctuate over time so their symptoms may not always be evident to healthcare professionals or friends and family. Although there is a wide evidence base on the epidemiology and clinical symptoms of MG, further evidence is needed on the lived experience of MG from the patient’s own perspective.
In 2018, UCB Pharma established an international patient council to facilitate a patient-led approach to improving treatment for people with generalized MG. The patient council was a group of leading, national patient advocates with MG from Belgium, Finland, France, the Netherlands, Spain, the United Kingdom and the United States. Here, we describe the approach we used in the design and conduct of a patient-led and patient co-authored analysis of the lived experience of generalized MG, which may be applicable to other indications. We also describe the self-reported experience of being involved in the project either as a member of the patient council, as a patient author or non-patient author, to understand how this approach could be improved for all involved.
Project approach for the published patient-led analysis
For this research, the patient council was composed of people with MG. Although family and caregiver views were considered, the focus was on those who have MG. We established a patient-led approach to the identification, collation, prioritization, analysis and publication of patient insights to describe the lived experience (Fig. 1). Care was taken to ensure that the proposed approach was discussed with all council members, and that they had an opportunity to input. Further details of the data sources used to extract insights and methods used in this study have been published elsewhere .
Identification of unmet needs
Patient council members highlighted a lack of understanding among many healthcare professionals about the lived experience of MG. They noted a lack of published patient-reported data and identified the need for a qualitative research analysis to better understand and report the patient perspective of living with MG.
Insight collation and categorization
At the start of the patient-led analysis, council members provided suggestions for domains that reflected different aspects of the lived experience of MG. These domains provided a framework representing the lived experience of MG and are described in full detail in the original patient-led analysis . Patient insights into the lived experience of MG based on qualitative data reported in the literature and direct patient quotes were extracted from three sources (Table 1). Insights and quotes extracted from the data sources were categorized into nine descriptive domains: physical, psychological, social, activities and participation, reproduction and parenting, controlled/inadequately controlled, flare-ups and myasthenic crises, treatment burden and unmet needs. Two researchers (Dawn Lobban, Jennie Hepburn) systematically collated and categorized patient insights, with specific quotes noted to support the extracted insights. Researchers discussed and cross-checked extracted insights to ensure the source data were accurately represented and to minimize researcher interpretation.
Generation of summary statements and patient author–led thematic analysis
Insights were reviewed and prioritized by patient council members using an online survey. Patients were asked to consider how well each insight represented the lived experience of MG. Each council member chose five insights that they felt best represented each of the domains from a global view based on their experience as patient advocates, rather than their personal experience. Patient council members had in-depth discussions about the survey findings during two half-day virtual workshops in August 2020 and subsequently developed summary statements of the lived experience.
Patient authors finalized these statements after the meeting. Based on collective experience, the patient council identified 44 summary statements across nine domains that they considered best represented the lived experience of generalized MG. As it has been reported that people living with chronic diseases can experience some sense of personal growth , and to ensure balance in the data, patient council members were also asked to share any positive aspects of living with MG. Patient authors reviewed the statements in an online meeting (October 2020) to further summarize the statements into five overarching themes to best describe the patient perspective of living with MG.
Publishing the research findings
Two patient authors and three non-patient authors worked together to report the findings of the analysis. Although both patient authors did have some prior experience of publications, the company publication lead (Veronica Porkess) explained the publication process to them at the outset to ensure that they both understood their role, responsibilities and the time commitment associated with authorship of company-sponsored research publications. In addition, the publication lead provided plain language versions of some key documents: Good Publication Practice guidelines, author agreement form and confidentiality agreement . Medical writing support was provided to support patient and non-patient authors to publish the research findings.
Initially, the data were submitted as an abstract and presented as an audio-enhanced poster at a large neurology conference in the United States . The full manuscript was subsequently published in a leading neurology journal . A short supporting video was developed to increase interest, reach and accessibility of the data . The patient-led analysis of the lived experience of MG has received high online attention (6504 article accesses, 7 citations and an Altmetric score of 263 as of March 2023 since publication in October 2021). The article received mentions in 35 news outlets and was ranked 1st of the tracked articles of a similar age in Neurology and Therapy. The involvement of patient council members and patient co-authors in this project is summarized in Additional file 1 following the Guidance for Reporting Involvement of Patients and the Public (GRIPP2), which have been developed to improve the reporting of patient and public involvement in health research [23, 24].
Assessing the self-reported experience of patient involvement in the patient-led analysis
We assessed the involvement of patient council members, patient authors and non-patient authors using a self-reported experience online survey (Additional file 2). The survey was based on the Patient Focused Medicines Development (PFMD) Patient Engagement Quality Guidance criteria for planning or assessing patient engagement activities . Information on the survey and how it was developed has been reported previously [20, 26]. Questions on the results and outcomes of this project were also included in the survey based on recommendations in the PFMD Book of Good Practices and GRIPP2 [23, 24, 27].
Overall, eight domains were assessed using a five-point, psychometric Likert scale (strongly disagree to strongly agree) and an overall score was calculated as a percentage of the maximum possible score. Questions were customized for relevance (i.e., experience of being involved “as a member of the patient council” or “as a patient author”) and were reviewed and approved by target audience representatives. In September 2020, nine members of the patient council were invited to complete the patient experience survey comprising 16 questions to evaluate how well researchers worked with patient partners in the qualitative data generation. Eight members of the patient council completed the survey and one patient council member did not complete the survey, despite a follow-up email. Following the publication of the research findings, we gained feedback on the authorship experience from three non-patient authors (13 questions) and one patient author (17 questions) in December 2021. Following preparation of the lived experience manuscript, one of the patient authors sadly passed away so only one patient author completed the authorship experience survey.
Overall, patient council members rated their experience of taking part in this analysis as positive, with an average patient experience score of 90% (71.6/80.0; n = 8). The patient experience survey results from the patient council members are summarized in Fig. 2a. Patients provided additional open-text feedback on their experience of being involved in the analysis (Fig. 2b). Involvement in the patient co-authored publication was also rated highly: experience score was 92% (78/85; n = 1) for the patient author (Fig. 3a) and 97% (63.3/65; n = 3) for non-patient authors (Fig. 3b).
Key learnings to optimize the approach
Based on our own experience of the approach and the feedback provided by the patient council members and authors, Table 2 captures key aspects that contributed to the overall success of the approach. We also reflect on elements of the approach that could be improved in future studies. Consensus on the key aspects leading to success and elements for improvement was achieved through a virtual discussion followed by two rounds of online written review.
The patient co-author’s perspective (Kelly Davio)
This patient-led approach presented a unique opportunity for patient advocates from multiple countries to describe their lived experience (and their broader patient communities’ lived experiences) of MG. It also allowed the insights generated in this process to be documented in the peer-reviewed literature, which I believe will contribute to greater understanding of patients’ experiences, needs and perspectives. As an individual patient, I found the process of engaging with the patient council and of authorship to be a unique and indeed empowering opportunity to share and represent the views and needs of the MG community. I believe this approach can be successfully used among patients with other conditions to amplify the patient voice and to help to create a more nuanced understanding of patients’ perspectives and priorities among healthcare decision-makers
Patient involvement in research publications is a relatively new area. As recently highlighted, patient engagement is not always successful, e.g., there can be an unconscious bias towards patient partners and a lack of support to fully include patients . As such, it is important to reflect on the patient experience of being involved to improve future patient involvement initiatives . We gained useful insights into ways to improve patient involvement in future research and patient co-authored publications. By involving patients at the project outset, clearly outlining roles and responsibilities and being flexible about how patients provided input, we enabled patients to publish valuable insights into the lived experience of MG.
We used a patient-led approach to collate, prioritize and analyze insights from people living with MG. This provided a comprehensive view of the patient perspective of the lived experience of MG. Patient council members had extensive experience as patient advocates and had interacted with a broad range of patients from across the world. This enabled council members to identify wider insights that best represented the patient perspective of living with MG. These insights were published to help healthcare professionals better understand their patients’ experience of living with MG that may not be evident in routine clinical appointments. Importantly, this could facilitate understanding of the impact of symptoms (e.g., on a patient’s quality of life, mental health, relationships, participation in activities). This approach was also a way for patient advocates to raise their voice and ensure their insights were included in the peer-reviewed literature to improve awareness of living with MG for anyone with an interest in the disease.
Although the number of patient authors is currently low and difficult to measure [11, 30], evidence-based guidance and resources to facilitate patient authorship are emerging [10, 31,32,33]. This guidance can help to overcome the perceived barriers to patient authorship. The recent Good Publication Practice guidelines clearly recognize that patients can provide important input into publications, and patients who meet authorship criteria should be listed as authors . For example, a systematic literature review co-authored by patients and non-patient authors explored the risks and benefits of involving patients in preparing health research peer-reviewed publications . The review provides 21 evidence-based recommendations for involving patients before, during and after a manuscript is developed. To assist conversations between researchers and patient authors, members of the Strategy for Patient-Oriented Research Chronic Pain Network's Patient Engagement Committee have published guidance on patient authorship .
Practical open-access resources are now available to support patient authors and companies and academic collaborators working with patients [20, 35]. The self-directed online Patients in Publications course trains patient advocates on how to engage in publications as authors and peer-reviewers . Plain language resources have been co-developed with patient advocates that explain how patients can meet the ICMJE authorship criteria, provide an overview of the publication process, outline the rights and responsibilities and provide guidance on author disclosures . Although we know that patient authorship is increasing, currently it can be difficult and time-consuming to identify patient-authored publications [11, 30]. The affiliation search function in PubMed has been proposed as a quick and easy way to identify patient-authored publications, but consensus is needed for stakeholders on a suitable standard meta tag (such as Patient Author) or set of meta tags to track patient authors . Improving our understanding of the true extent of patient authorship could encourage more patients to contribute their perspectives to the peer-reviewed literature.
Evaluating the experience of patients engaged in research can provide valuable feedback and identify areas to improve future collaborations. Patient experience tools allow patients and non-patients to reflect on their experience of being involved in a research project and can highlight challenges they may have faced as well as benefits of taking part in the research. Overall, in this project, patients involved as a patient council member or as a patient author had a positive experience. Since publication, the patient-led analysis of the lived experience of MG has received high online attention, demonstrating interest in this type of analysis to better understand the lived experience . Whereas similar Altmetric scores may be seen for publications on common conditions, these scores are high for a publication on a rare disease. A limitation of this research is that only nine participants were involved in this research who were all patients living with MG. However, the results of this analysis may be informative for people affected by MG in the wider sense including “the family and those voluntarily caring for those with the medical condition(s), patient advocates, and patient groups” [25, 29]. This patient-led approach provides a framework that other researchers could follow in future research and adapt to gain valuable insights on the lived experience of other conditions.
Availability of data and materials
Guidance for Reporting Involvement of Patients and the Public
International Committee of Medical Journal Editors
Patient Focused Medicines Development
Hoos A, Anderson J, Boutin M, Dewulf L, Geissler J, Johnston G, et al. Partnering with patients in the development and lifecycle of medicines: a call for action. Ther Innov Regul Sci. 2015;49:929–39.
Geissler J, Ryll B, di Priolo SL, Uhlenhopp M. Improving patient involvement in medicines research and development: a practical roadmap. Ther Innov Regul Sci. 2017;51:612–9.
United States Food & Drug Administration. Patient listening sessions. https://www.fda.gov/patients/learn-about-fda-patient-engagement/patient-listening-sessions. Accessed 22 March 2023.
European Medicines Agency. Patients and consumers. https://www.ema.europa.eu/en/partners-networks/patients-consumers. Accessed 22 March 2023.
Olthuis G, Leget C, Grypdonck M. Why shared decision making is not good enough: lessons from patients. J Med Ethics. 2014;40:493–5.
Garrino L, Picco E, Finiguerra I, Rossi D, Simone P, Roccatello D. Living with and treating rare diseases: experiences of patients and professional health care providers. Qual Health Res. 2015;25:636–51.
Keer-Keer T. The lived experience of adults with myasthenia gravis: a phenomenological study. Australas J Neurosci. 2015;25:40–6.
Bombard Y, Baker GR, Orlando E, Fancott C, Bhatia P, Casalino S, et al. Engaging patients to improve quality of care: a systematic review. Implement Sci. 2018;13:98.
Cobey KD, Monfaredi Z, Poole E, Proulx L, Fergusson D, Moher D. Editors-in-chief perceptions of patients as (co) authors on publications and the acceptability of ICMJE authorship criteria: a cross-sectional survey. Res Involv Engagem. 2021;7:39.
Arnstein L, Wadsworth AC, Yamamoto BA, Stephens R, Sehmi K, Jones R, et al. Patient involvement in preparing health research peer-reviewed publications or results summaries: a systematic review and evidence-based recommendations. Res Involv Engagem. 2020;6:34.
Oliver J, Lobban D, Dormer L, Walker J, Stephens R, Woolley K. Hidden in plain sight? Identifying patient-authored publications. Res Involv Engagem. 2022;8:12.
Law N, Davio K, Blunck M, Lobban D, Seddik K. The lived experience of myasthenia gravis: a patient-led analysis. Neurol Therapy. 2021;10:1103–25.
Gilhus NE, Verschuuren JJ. Myasthenia gravis: subgroup classification and therapeutic strategies. Lancet Neurol. 2015;14:1023–36.
Verschuuren JJ, Palace J, Gilhus NE. Clinical aspects of myasthenia explained. Autoimmunity. 2010;43:344–52.
Ruiter AM, Verschuuren J, Tannemaat MR. Fatigue in patients with myasthenia gravis: a systematic review of the literature. Neuromuscul Disord. 2020;30:631–9.
Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren J. Myasthenia gravis. Nat Rev Dis Primers. 2019;5:30.
Tanovska N, Novotni G, Sazdova-Burneska S, Kuzmanovski I, Boshkovski B, Kondov G, et al. Myasthenia gravis and associated diseases. Open Access Maced J Med Sci. 2018;6:472–8.
Cacho Diaz B, Flores-Gavilan P, Garcia-Ramos G, Lorenzana-Mendoza NA. Myasthenia Gravis and its comorbidities. J Neurol Neurophysiol. 2015;6:1–5.
Paterson B, Thorne S, Crawford J, Tarko M. Living with diabetes as a transformational experience. Qual Health Res. 1999;9:786–802.
Envision the Patient. Patient authorship resources. https://www.patientauthorship.com/. Accessed 22 March 2023.
Davio K, Law N, Blunck M, Seddik K. The lived experience of Myasthenia Gravis (MG): a patient-led analysis. Neurology. 2021;96:2272.
Adis journals on behalf of; Law N, Davio K, Blunck M, Lobban D, Seddik K. Patient author video. The lived experience of myasthenia gravis: a patient-led analysis. 2022. https://doi.org/10.6084/m9.figshare.19175462.v2.
Staniszewska S, Brett J, Simera I, Seers K, Mockford C, Goodlad S, et al. GRIPP2 reporting checklists: tools to improve reporting of patient and public involvement in research. BMJ. 2017;358: j3453.
Staniszewska S, Brett J, Simera I, Seers K, Mockford C, Goodlad S, et al. GRIPP2 reporting checklists: tools to improve reporting of patient and public involvement in research. Res Involv Engagem. 2017;3:13.
Patient Focused Medicine Development. Patient engagement quality guidance. 2018. https://patientfocusedmedicine.org/peqg/patient-engagement-quality-guidance.pdf. Accessed 22 March 2023.
Woolley KL, Arnstein L, Hamoirm AM, Lobban D, Stephens R, Yamamoto B. Original abstracts from the 15th Annual Meeting of ISMPP. Development and use of two tools to facilitate and evaluate patient authorship. Curr Med Res Opin. 2019;35:5–29.
Patient Focused Medicine Development. The PFMD Book of Good Practices: Patient involvement in preparing clinical research peer-reviewed publications or results summaries: a systematic review. 2019. https://patientfocusedmedicine.org/bogp/2019/the-book-of-good-practices.pdf. Accessed 22 March 2023.
Richards DP, Poirier S, Mohabir V, Proulx L, Robins S, Smith J. Reflections on Patient Engagement by Patient Partners: How it can go Wrong. 2023. Preprint at https://doi.org/10.31219/osf.io/q3e76.
National Health Council. Patient-focused drug development – recommended language for use in guidance document development. 2017. http://www.nationalhealthcouncil.org/sites/default/files/NHC-GA%20Feb2017.pdf. Accessed 22 March 2023.
Ellis U, Kitchin V, Vis-Dunbar M. Identification and reporting of patient and public partner authorship on knowledge syntheses: rapid review. J Particip Med. 2021;13:e27141.
Richards DP, Birnie KA, Eubanks K, Lane T, Linkiewich D, Singer L, et al. Guidance on authorship with and acknowledgement of patient partners in patient-oriented research. Res Involv Engagem. 2020;6:38.
Woolley K, Yamamoto BA, Stephens R, Lobban D, Ebina H, Arnstein L. Patient authorship: three key questions (& answers!) for medical communication professionals [part A]. The MAP Newsletter. 2020. https://ismpp-newsletter.com/2020/05/13/patient-authorship-three-key-questions-answers-for-medical-communication-professionals-part-a/. Accessed 22 March 2023.
Woolley K, Yamamoto BA, Stephens R, Lobban D, Ebina H, Arnstein L. Patient authorship: three key questions (& answers!) for medical communication professionals [part B]. The MAP Newsletter. 2020. https://ismpp-newsletter.com/2020/05/26/patient-authorship-three-key-questions-answers-for-medical-communication-professionals-part-b/. Accessed 22 March 2023.
DeTora LM, Lane T, Sykes A, DiBiasi F, Toroser D, Citrome L. Good publication practice (GPP) guidelines for company-sponsored biomedical research: 2022 update. Ann Intern Med. 2023;176:eL220490.
WECAN, Envision Pharma Group. Patients in Publications training course. 2021. https://wecanadvocate.eu/patients-in-publications/. Accessed 22 March 2023.
Oliver J, Porkess V, McSorley S, Lobban D. Measuring the quality of patient engagement in a patient-led and co-authored analysis of the lived experience of a rare disease. Original abstracts from the 18th annual meeting of ISMPP. Curr Med Res Opin. 2022;38:5–24.
Following the development of the lived experience analysis manuscript, lead and patient co-author Nancy Law sadly passed away (September 2021). Nancy was key to driving this analysis to bring the voice of people living with MG into the peer-reviewed literature. The authors and UCB both gratefully acknowledge Nancy’s valuable and inspirational contribution to this work. We give special thanks to the people with MG who participated in the qualitative data generation. We thank the patient advocates who were members of the International Myasthenia Gravis Patient Council in September 2019 and August 2020 (see Law et al.  for full details of the patient council members). Jenny Hepburn (Envision Pharma Group) assisted with the thematic analysis of the insights, funded by UCB Pharma.
This manuscript is based on work that has been presented as a poster at The International Society for Medical Publication Professionals Annual Meeting 2022 and the abstract was published in Current Medical Research and Opinion .
This analysis was funded by UCB Pharma (Brussels, Belgium).
Ethics approval and consent to participate
Neither the global qualitative study nor the patient council–led analysis was an investigation of clinical outcomes with any intervention. The global qualitative study adhered to local market research guidelines in the countries included and was conducted in accordance with EphMRA Code of Conduct. Therefore, neither ethics committee approval nor clinical trial registration was required. The patients who participated in the qualitative research study were fully informed of the purpose of research, provided consent to participate and agreed for their data to be published.
Consent for publication
Members of the patient council consented to the publication of their insights and analysis.
DL and JO are employees and shareholders of Envision Pharma Group. KD is a member of the patient council. KS and VP are employees of UCB Pharma. DL, KD and KS were participants in the authorship experience survey described in this article.
Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
About this article
Cite this article
Lobban, D., Oliver, J., Davio, K. et al. Evaluation of the quality of patient involvement in a patient-led analysis of the lived experience of a rare disease. Res Involv Engagem 9, 35 (2023). https://doi.org/10.1186/s40900-023-00445-2