Evaluation of the quality of patient involvement in a patient-led analysis of the lived experience of a rare disease.

BACKGROUND: 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. METHODS: 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. RESULTS: 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. CONCLUSION: 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.

WHY DID WE DO THIS RESEARCH?: 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. 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. 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.

A practical 'How-To' Guide to plain language summaries (PLS) of peer-reviewed scientific publications: results of a multi-stakeholder initiative utilizing co-creation methodology.

BACKGROUND: Peer-reviewed scientific publications and congress abstracts are typically written by scientists for specialist audiences; however, patients and other non-specialists are understandably interested in the potential implications of research and what they may mean for them. Plain language summaries (PLS)-summaries of scientific articles in easy-to-read language-are emerging as a valuable addition to traditional scientific publications. Co-creation of PLS with the intended audience is key to ensuring a successful outcome, but practical guidance on how to achieve this has been lacking. METHODS: Building on the Patient Engagement (PE) Quality Guidance previously developed by Patient Focused Medicines Development (PFMD), a multi-stakeholder working group (WG) of individuals with patient engagement experience and/or expertise in PLS was established to develop further activity-specific guidance. PLS guidance was developed through a stepwise approach that included several rounds of co-creation, public consultation (two rounds), internal review and a final external review. The iterative development process incorporated input from a wide variety of stakeholders (patient representatives, industry members, publishers, researchers, medical communications agencies, and public officials involved in research bodies). Feedback from each step was consolidated by the WG and used for refining the draft guidance. The final draft was then validated through external consultation. RESULTS: The WG comprised 14 stakeholders with relevant experience in PE and/or PLS. The WG developed a set of 15 ethical principles for PLS development. These include the necessity for objective reporting and the absence of any promotional intent, the need for balanced presentation, the importance of audience focus, the need to apply health literacy principles, and the importance of using inclusive and respectful language. The first public consultation yielded 29 responses comprising 478 comments or edits in the shared draft guidance. The second public consultation was an online survey of 14 questions which had 32 respondents. The final 'How-To' Guide reflects feedback received and provides a rational, stepwise breakdown of the development of PLS. CONCLUSIONS: The resulting 'How-To' Guide is a standalone, practical, ready-to-use tool to support multi-stakeholder co-creation of PLS.

We wanted to create practical guidance for people who are interested in developing plain language summaries of publications (PLS for short). PLS are summaries of scientific research published in journals or presented at conferences and are written in language that is easy to read and understand. We focused on how to involve patients in developing PLS, as they are often an important audience for these summaries. We brought together a group of people who had experience in PLS and patient involvement. As a working group, we wrote the first version of the 'How-To' Guide. Then we asked for feedback from others experienced in patient involvement and also from members of the general public. We got feedback on how we could improve what was in the guidance and also on how useful and user-friendly the guidance was. We used this feedback to create the final version of the 'How-To' Guide which is freely available online from https://pemsuite.org/How-to-Guides/WG5.pdf .

Hidden in plain sight? Identifying patient-authored publications.

BACKGROUND: Patient advocates are increasingly authoring peer-reviewed publications that could enhance patient care and understanding of the lived experience. Although patient authorship may be seen as an innovation in the peer-reviewed publication environment and some may not be aware of or accept patient authorship, we know patient-authored publications exist. However, identifying patient-authored publications is often challenging and time-consuming. MAIN BODY: In this commentary, we propose a definition for a patient author and patient-authored publications. We outline factors driving the increase in patient authorship, including patient interest, recognition of the value of including the patient voice and major funders recognising the importance of involving patient advocates in research. Evidence and experience-based guidance on patient authorship is emerging, and we highlight practical guidance for patient advocates on authoring peer-reviewed publications. To gain a better understanding of patient authorship, an efficient method is needed to identify patient-authored publications. A dataset on patient-authored publications could be used for a range of quantitative and qualitative research studies. The affiliation search function in PubMed can provide an easy, and reproducible way to identify a dataset of patient-authored publications in the international peer-reviewed literature, but only if patient authors include a standard metatag, (e.g. Patient Author) as one of their listed affiliations, combined with other affiliations as appropriate. From 2020 to 2021, there was a nine-fold increase in patient-authored publications in PubMed identified using the Patient Author tag. We recognize that terminology can be contentious and some authors may prefer alternative metatags. Further efforts are required to gain consensus on a suitable, standard metatag or set of metatags to use to show the true extent of patient authorship. CONCLUSION: Patient authorship is not only legitimate, but it also exemplifies the principles of diversity, equity and inclusion. Stakeholders in the publication community need to review their policies and procedures to identify and address barriers to patient authorship. Patient advocates, funders, researchers and publishers could all help to promote awareness and acceptance of patient authorship and the merits of using a standard metatag or set of metatags, so that patient-authored publications are no longer hidden in plain sight.

Some patients are leading or helping with medical research to improve understanding of their condition and patient care. To share research findings, patients can author articles published in scientific journals. These articles are reviewed by experts and are known as peer-reviewed publications. Patient authors can provide unique and valuable insights from their experience of living with a condition. Demonstrating that patients can be authors would be easier if there was a quick way to find patient-authored publications. In this article, we describe who a patient author is and what patient-authored publications are. We identify factors that may encourage patients to author research publications. We highlight the practical guidance available to help patient authors and those working with them. To help future research about patient authorship, we need a way to find patient-authored publications. One way is for patients to include a standard search term, such as 'Patient Author' in the affiliation section of their publication. Like all authors, patient authors can list more than one affiliation, such as their workplace if they wish. We used the 'Patient Author' search term to look at publications in PubMed, a free resource to access scientific publications. We found the number of patient-authored publications using the 'Patient Author' tag increased nine times from 2020 and 2021. We encourage patients, funders, researchers and publishers to use a standard metatag or an agreed set of metatags. This could make it easier to find and raise awareness of patient-authored publications.

Plain language summaries of publications of company-sponsored medical research: what key questions do we need to address?

OBJECTIVES: We aimed to gather multi-stakeholder insights on key issues relating to plain language summaries (PLS) of company-sponsored medical research to inform future industry recognized guidelines. METHODS: We identified diverse stakeholders based on expertise, familiarity with PLS, and geographical location. A Working Group (n = 11) with extensive expertise in PLS developed an initial list of 14 questions relating to PLS, which were shared with stakeholders. We used a modified Delphi approach to prioritize the 10 key questions that were then used to structure stakeholder discussions to collect evidence on the key challenges and opportunities to inform best practice for PLS. RESULTS: Overall, 29 stakeholders took part in the study, representing different professional sectors and geographies. There was strong alignment among stakeholders on the priority questions for PLS, with high response rates for both surveys (69% and 90%). Moderated online sessions were attended by 27/29 stakeholders and opportunities to improve PLS uptake were highlighted: developing industry-wide PLS guidelines would help define and maintain quality, including having a clear directive for when publications should have a PLS; further advocacy is needed by target audiences to ensure PLS become an established part of company-sponsored research publications; a searchable repository could facilitate discoverability and broad dissemination of PLS to multiple target audiences. CONCLUSIONS: Key issues identified by stakeholders provide broad insights into the real and perceived barriers relating to PLS uptake. Each emerging theme presents a possible action that could accelerate PLS uptake and facilitate sharing of new medical research with lay audiences.

What do you need to know?: Plain language summaries (PLS for short) explain medical research in a clear and understandable way. We found ways to improve how PLS are developed to help people understand medical research publications. See Additional file 1 for a graphic PLS. Why did we do this study?: Many people struggle to understand medical research because authors use complex words in research publications. Currently, PLS vary in style and format and most research publications do not have a PLS. What did we do?: We invited 29 experts from various organizations who develop, publish, read, or fund PLS to take part in this study. We asked them what issues they thought are preventing research publications from having a PLS, and how we could solve these issues. What did we find?: Most groups agreed on the top 10 issues we need to look at to improve PLS so that more publications have a PLS. There are several ways to improve how PLS are developed. Guidelines on which publications should have a PLS and what they should include are urgently needed. People who think PLS are important need to request them to help make sure they are regularly included in research publications. We also need clear ways to share PLS, so people with an interest in medical research can find and read them.

The Lived Experience of Myasthenia Gravis: A Patient-Led Analysis.

INTRODUCTION: A greater understanding of the reality of living with myasthenia gravis (MG) may improve management and outcomes for patients. However, there is little published data on the patient perspective of how MG impacts life. Our objective was to reveal the lived experience of MG from the patient perspective. METHODS: This analysis was led by an international Patient Council comprising nine individuals living with MG who serve as local/national patient advocates in seven countries (Europe and the United States). Insights into the lived experience of MG were consolidated from three sources (a qualitative research study of 54 people with MG or their carers from seven countries; a previous Patient Council meeting [September 2019]; and a literature review). Insights were prioritised by the Patient Council, discussed during a virtual workshop (August 2020) and articulated in a series of statements organised into domains. Overarching themes that describe the lived experience of MG were identified by the patient authors. RESULTS: From 114 patient insights and supporting quotes, the Patient Council defined 44 summary statements organised into nine domains. Five overarching themes were identified that describe the lived experience of MG. These themes include living with fluctuating and unpredictable symptoms; a constant state of adaptation, continual assessment and trade-offs in all aspects of life; treatment inertia, often resulting in under-treatment; a sense of disconnect with healthcare professionals; and feelings of anxiety, frustration, guilt, anger, loneliness and depression. CONCLUSION: This patient-driven analysis enriches our understanding of the reality of living with MG from the patient perspective.

Multicenter randomized controlled trial of the management of unresectable malignant mesothelioma proposed by the British Thoracic Society and the British Medical Research Council.

Malignant mesothelioma is almost invariably fatal. The incidence of the disease is rising rapidly in many countries, and there is no generally accepted standard treatment for patients with unresectable disease. According to current British Thoracic Society (BTS) guidelines, patients should be treated with active symptom control (ASC), involving (1) regular follow-up in a specialist clinic; (2) structured assessments of physical, psychological and social problems with appropriate action; (3) rapid involvement of additional specialists; and (4) parallel nursing support. Although many nonrandomized studies have reported tumor responses to anticancer chemotherapy, few have studied palliation and it is not known whether chemotherapy prolongs survival or provides clinically worthwhile palliation with acceptable toxicity when given in addition to ASC. We therefore plan to conduct a multicenter randomized controlled trial comparing (1) ASC alone, (2) ASC plus mitomycin vinblastine and cisplatin (MVP), and (3) ASC plus vinorelbine (N; Navelbine, Pierre Fabre Oncology, Winchester, UK). We chose these chemotherapy regimens because they have been shown in nonrandomized studies to provide good symptom control as recorded by patients. The outcome measures are overall survival, palliation of symptoms, performance status, analgesic usage, toxicity, quality of life, tumor response, and recurrence/progression-free survival. In a preliminary feasibility study, we are assessing the acceptability of the trial design to patients and the suitability of two standard quality-of-life instruments in mesothelioma. Data will help us to decide the final details of the large multicenter trial.