Katherine Cowan

Tackling treatment uncertainties together: the evolution of the James Lind Initiative, 2003–2013

At the end of 2003, the Journal of the Royal Society of Medicine published an article announcing the creation of the James Lind Initiative (JLI).1 The article began by recalling the way that James Lind, an 18th century Scottish naval surgeon, had confronted uncertainty about how to treat scurvy by doing a controlled trial. This had shown that oranges and lemons were a very effective cure for an often lethal disease.2,3 The 2003 article went on to suggest that therapeutic uncertainties today should also be confronted in research as an integral element of responsible healthcare, and that the JLI was being established to promote this principle. The current article describes the origins of the JLI and its work over the past 10 years.

Setting research priorities to improve the health of children and young people with neurodisability: a British Academy of Childhood Disability-James Lind Alliance Research Priority Setting Partnership

Objectives To engage young people, parent carers and clinicians in a systematic process to identify and prioritise research questions regarding ways to improve the health and well-being of children and young people with neurodisability. Design British Academy of Childhood Disability (BACD)-James Lind Alliance research priority setting partnership bringing together patients, carers and clinicians as equal stakeholders. Setting UK health service and community. Methods The BACD Strategic Research Group formed the partnership. A Steering Group was established; charity and professional partner organisations were recruited. Suggestions were gathered in an open survey and from research recommendations for statutory guidance. Items were aggregated to formulate indicative research questions and verified as uncertainties from research evidence. An interim survey was used to rank the questions to shortlist topics. A mixed group of stakeholders discussed the top 25 questions at the final priority setting workshop agreeing a final rank order and the top 10 research priorities. Participants Partner organisations were 13 charities and 8 professional societies. 369 people submitted suggestions (40% non-clinicians). 76 people participated in the interim prioritisation (26 parents, 1 young person, 10 charity representatives, 39 clinicians); 22 took part in the final workshop (3 young people, 7 parents, 3 charity representatives, 9 professionals). Results The top three research priorities related to (1) establishing the optimal frequency and intensity (dose) for mainstream therapies, (2) means for selecting and encouraging use of communication strategies and (3) ways to improve childrens attitudes towards disability. The top 10 included evaluating interventions to promote mobility, self-efficacy, mental health, continence, physical fitness, educational inclusion and reduce impacts of sleep disturbance. Conclusions The methodology provided a systematic and transparent process to identify research priorities that included stakeholders that have typically not contributed to setting the research agenda. The top 10 and other topics identified provide a resource for researchers and agencies that fund research

The Sight Loss and Vision Priority Setting Partnership (SLV-PSP): overview and results of the research prioritisation survey process.

Objectives The Sight Loss and Vision Priority Setting Partnership aimed to identify research priorities relating to sight loss and vision through consultation with patients, carers and clinicians. These priorities can be used to inform funding bodies’ decisions and enhance the case for additional research funding. Design Prospective survey with support from the James Lind Alliance. Setting UK-wide National Health Service (NHS) and non-NHS. Participants Patients, carers and eye health professionals. Academic researchers were excluded solely from the prioritisation process. The survey was disseminated by patient groups, professional bodies, at conferences and through the media, and was available for completion online, by phone, by post and by alternative formats (Braille and audio). Outcome measure People were asked to submit the questions about prevention, diagnosis and treatment of sight loss and eye conditions that they most wanted to see answered by research. Returned survey questions were reviewed by a data assessment group. Priorities were established across eye disease categories at final workshops. Results 2220 people responded generating 4461 submissions. Sixty-five per cent of respondents had sight loss and/or an eye condition. Following initial data analysis, 686 submissions remained which were circulated for interim prioritisation (excluding cataract and ocular cancer questions) to 446 patients/carers and 218 professionals. The remaining 346 questions were discussed at final prioritisation workshops to reach agreement of top questions per category. Conclusions The exercise engaged a diverse community of stakeholders generating a wide range of conditions and research questions. Top priority questions were established across 12 eye disease categories.

Top ten research priorities for spinal cord injury: the methodology and results of a British priority setting partnership

Study design:This is a mixed-method consensus development project.Objectives:The objective of this study was to identify a top ten list of priorities for future research into spinal cord injury (SCI).Setting:The British Spinal Cord Injury Priority Setting Partnership was established in 2013 and completed in 2014. Stakeholders included consumer organisations, healthcare professional societies and caregivers.Methods:This partnership involved the following four key stages: (i) gathering of research questions, (ii) checking of existing research evidence, (iii) interim prioritisation and (iv) a final consensus meeting to reach agreement on the top ten research priorities. Adult individuals with spinal cord dysfunction because of trauma or non-traumatic causes, including transverse myelitis, and individuals with a cauda equina syndrome (henceforth grouped and referred to as SCI) were invited to participate in this priority setting partnership.Results:We collected 784 questions from 403 survey respondents (290 individuals with SCI), which, after merging duplicate questions and checking systematic reviews for evidence, were reduced to 109 unique unanswered research questions. A total of 293 people (211 individuals with SCI) participated in the interim prioritisation process, leading to the identification of 25 priorities. At a final consensus meeting, a representative group of individuals with SCI, caregivers and health professionals agreed on their top ten research priorities.Conclusion:Following a comprehensive, rigorous and inclusive process, with participation from individuals with SCI, caregivers and health professionals, the SCI research agenda has been defined by people to whom it matters most and should inform the scope and future activities of funders and researchers for the years to come.Sponsorship:The NIHR Oxford Biomedical Research Centre provided core funding for this project.

Top ten research priorities for spinal cord injury

In the Edwin Smith papyrus—the oldest known surviving text about management of traumatic injuries— injuries to the spinal cord were considered to be “a medical condition that cannot be healed.’’ Although more than 3000 years later a cure for spinal cord injury has not yet been identifi ed, research into the diagnosis, prognosis, and treatment of spinal cord injury, and its wide range of secondary sequelae has intensifi ed in the past few decades. Nonetheless, to our knowledge, no systematic appraisal of patients’, carers’ and health-care professionals’ preferred research questions into spinal cord has been done to date. In collaboration with the James Lind Alliance, a UK National Institute for Health Research-supported organ isation that aims to identify gaps in scientifi c, medical, and psychosocial knowledge that matter most to patients, carers, and health professionals, we completed a multidisciplinary priority setting partnership that has defi ned a British research agenda for spinal cord injury for the next 5–10 years. There are as yet, no plans to monitor progress. Our priority setting process involved four key stages: (1) collecting research questions, (2) checking of existing research evidence through systematic searches of the literature by the project data manager, (3) interim prioritisation, and (4) a fi nal consensus meeting to reach agreement about the top ten research priorities. We invited individuals with spinal cord dysfunction due to trauma and non-traumatic causes, including trans verse myelitis and those with cauda equina syndrome (henceforth grouped and referred to as individuals with spinal cord injury), to participate in this priority setting partnership. Additionally, we encouraged caregivers and healthcare professionals with an interest in spinal cord injury to participate. In view of the highly specialist nature of spinal cord injury in children, no questions addressing paediatric injury were considered. We obtained 784 questions from 403 survey respondents (290 individuals with spinal cord injury) which, after merging of duplicate questions and checking of systematic reviews for evidence, were reduced to 109 unique unanswered research questions. A total of 293 people (211 individuals with spinal cord injury) participated in the interim prioritisation process, leading to the identifi cation of 25 shared top priorities. At a fi nal consensus meeting, a group of individuals with spinal cord injury, carers, and health professionals agreed their top ten priorities for future research (panel). Whilst the call for stem-cell research into spinal cord injury resonated with the people involved in this partnership, nine other research priorities show that spinal cord injury research should involve much more than presently meets the public eye. This research agenda for spinal cord injury has been defi ned by people to whom it matters most, and should now inform the scope and future activities of funders and researchers alike.

Setting Research Priorities for Kidney Cancer

Defining disease-specific research priorities in cancer can facilitate better allocation of limited resources. Involving patients and caregivers as well as expert clinicians in this process is of value. We undertook this approach for kidney cancer as an example. The Kidney Cancer Research Network of Canada sponsored a collaborative consensus-based priority-setting partnership that identified ten research priorities in the management of kidney cancer. These are discussed in the context of current initiatives and gaps in knowledge.

A paradigm change to inform fibromyalgia research priorities by engaging patients and health care professionals

ABSTRACT Background: Research objectives should be focused toward advancing knowledge that has meaningful impact on health. However, research agendas are mostly driven by the health care community, with limited input from patients. Aims: In this study, prioirities of uncertainties for the management of fibromyalgia (FM) that could propel future research were identified by a defined process using the James Lind Alliance Priority Setting Partnership (JLA-PSP) methodology. Methods: As a first step, a survey was distributed across Canada that engaged patients, caregivers, and health care professionals to provide narrative input to eight open-ended questions regarding FM care. Responses were thematically condensed and synthesized into an initial list of 43 uncertainties used to guide a comprehensive literature search. Questions already effectively addressed in the literature were excluded, leaving 25 uncertainties that were ranked during a one-day consensus workshop. Results: Three broad themes emerged: the value of personalized targeted treatment and subgrouping of patients; the efficacy of various self-management strategies and educational initiatives; and identification of the ideal health care setting to provide FM care. Opioids and cannabinoids were the only specific pharmacologic interventions ranked as needing further research. Conclusions: The prioritized questions highlight the importance of recognizing the heterogeneity of FM symptoms, the need for a personalized treatment approach, and a better understanding of the value of self-management strategies. This is the first study that uses an established and transparent methodology to engage all FM stakeholders to help inform researchers and funding bodies of clinically relevant research priorities.

Top 10 research priorities in head and neck cancer: Results of an Alberta priority setting partnership of patients, caregivers, family members, and clinicians

The epidemiology, etiology, and management of head and neck cancer are evolving. Understanding the perspectives and priorities of nonresearchers regarding treatment uncertainties is important to inform future research.

Dutch juvenile idiopathic arthritis patients, carers and clinicians create a research agenda together following the James Lind Alliance method: a study protocol

BackgroundResearch on Juvenile Idiopathic Arthritis (JIA) should support patients, caregivers/parents (carers) and clinicians to make important decisions in the consulting room and eventually to improve the lives of patients with JIA. Thus far these end-users of JIA-research have rarely been involved in the prioritisation of future research.Main bodyDutch organisations of patients, carers and clinicians will collaboratively develop a research agenda for JIA, following the James Lind Alliance (JLA) methodology. In a ‘Priority Setting Partnership’ (PSP), they will gradually establish a top 10 list of the most important unanswered research questions for JIA. In this process the input from clinicians, patients and their carers will be equally valued. Additionally, focus groups will be organised to involve young people with JIA. The involvement of all contributors will be monitored and evaluated. In this manner, the project will contribute to the growing body of literature on how to involve young people in agenda setting in a meaningful way.ConclusionA JIA research agenda established through the JLA method and thus co-created by patients, carers and clinicians will inform researchers and research funders about the most important research questions for JIA. This will lead to research that really matters.

Patient and Public Involvement in Identifying Dementia Research Priorities: Priorities for research on dementia

To engage persons with dementia, friends, family, caregivers, and health and social care providers to identify and prioritize their questions for research related to living with dementia and prevention, diagnosis, and treatment of dementia.