UK research priority setting for childhood neurological conditions.

AIM: To identify research priorities regarding the effectiveness of interventions for children and young people (CYP) with childhood neurological conditions (CNCs). These include common conditions such as epilepsies and cerebral palsy, as well as many rare conditions. METHOD: The National Institute for Health and Care Research (NIHR) and the James Lind Alliance (JLA) champion and facilitate priority setting partnerships (PSPs) between patients, caregivers, and clinicians (stakeholders) to identify the most important unanswered questions for research (uncertainties). A NIHR-JLA and British Paediatric Neurology Association collaboration used the JLA PSP methodology. This consisted of two surveys to stakeholders: survey 1 (to identify uncertainties) and survey 2 (a prioritization survey). The final top 10 priorities were agreed by consensus in a stakeholder workshop. RESULTS: One hundred and thirty-two charities and partner organizations were invited to participate. In survey 1, 701 participants (70% non-clinicians, including CYP and parent and caregivers) submitted 1800 uncertainties from which 44 uncertainties were identified for prioritization in survey 2; from these, 1451 participants (83% non-clinicians) selected their top 10 priorities. An unweighted amalgamated score across participant roles was used to select 26. In the final workshop, 14 health care professionals, 11 parent and caregivers, and two CYP ranked the 26 questions to finalize the top 10 priorities. Ten top priority questions were identified regarding interventions to treat CYP with CNCs and their associated comorbidities, for example, sleep, emotional well-being, and distressing symptoms. INTERPRETATION: The results of this study will inform research into the effectiveness of interventions for children with neurological conditions.

The top 10 research priorities in psoriatic arthritis: a James Lind Alliance Priority Setting Partnership.

OBJECTIVES: To identify and prioritize the top 10 research questions for PsA. METHODS: The British Psoriatic Arthritis Consortium (BritPACT) formed a Priority Setting Partnership (PSP) comprising of people living with PsA, carers and clinicians, supported by the James Lind Alliance (JLA). This PSP followed the established three-stage JLA process: first, an online survey of people living with PsA, carers and clinicians to identify PsA questions, asking, 'What do you think are the most important unanswered questions in psoriatic arthritis research?' The questions were checked against existing evidence to establish 'true uncertainties' and grouped as 'indicative questions' reflecting the overarching themes. Then a second online survey ranked the 'true uncertainties' by importance. Finally, a workshop including people living with PsA and clinician stakeholders finalized the top 10 research priorities. RESULTS: The initial survey attracted 317 respondents (69% people living with PsA, 15% carers), with 988 questions. This generated 46 indicative questions. In the second survey, 422 respondents (78% people living with PsA, 4% carers) prioritized these. Eighteen questions were taken forward to the final online workshop. The top unanswered PsA research question was 'What is the best strategy for managing patients with psoriatic arthritis including non-drug and drug treatments?' Other top 10 priorities covered diagnosis, prognosis, outcome assessment, flares, comorbidities and other aspects of treatment (https://www.jla.nihr.ac.uk). CONCLUSION: The top 10 priorities will guide PsA research and enable PsA researchers and those who fund research to know the most important questions for people living with PsA.

Protocol for a Global Burns Research Priority Setting Partnership to agree the most important unanswered questions in international burns care.

INTRODUCTION: Burns affect 11 million people globally and can result in long-term disability with substantial associated healthcare costs. There is limited research funding to support trials to provide evidence for clinical decision-making. Research prioritisation ensures that research focuses on the topics most important to stakeholders, addressing issues of research waste and evidence gaps. The aim of this project is to agree the global top 10 research priorities important to international patients, carers and clinicians from all income status countries. METHODS AND ANALYSIS: The Global Burns Research Priority Setting Partnership will use James Lind Alliance methods to establish the top 10 research priorities in global burns care. An initial international online multilingual survey will collect candidate research priorities from stakeholders. To increase equity in participation, the survey will also be available via the social media app WhatsApp. Additionally, interviews will be conducted. Data will be analysed to identify and collate research questions and to verify that the priorities are true clinical uncertainties. This list will then be ranked by stakeholders in order of importance via a second online survey. Finally, a consensus meeting will identify the top 10 research priorities. ETHICS AND DISSEMINATION: The University of Bristol Medical School Faculty Ethical Committee has approved this project. Research into burn care should be prioritised to ensure that funding is focused where most needed. This should be undertaken internationally, to ensure inclusion of the views of professionals and patients from lower income countries, where the incidence of thermal burns is highest. The involvement of the James Lind Alliance will ensure that the methodology is robust and that the patient voice is heard. The final top 10 priorities will be disseminated to funders, governments and researchers internationally to inform future global burns research.

A Public Dialogue to Inform the Use of Wider Genomic Testing When Used as Part of Newborn Screening to Identify Cystic Fibrosis.

Cystic fibrosis (CF) has been included within the UK national newborn screening programme since 2007. The approach uses measures of immunoreactive trypsin (IRT) in dried blood spot samples obtained at day 5 of life. Samples which reveal IRT results >99.5th centile go on to be tested for a limited panel of CF mutations. While the programme works well and achieves a high level of sensitivity and specificity, it relies upon repeat testing in some cases and identifies probable carriers, both potentially provoking parental anxiety. In addition, the limited CF mutation panel may not fully reflect the ethnic diversity within the UK population. The use of wider genomic screening, made possible by next-generation sequencing to replace more limited panels, can be used to avoid these shortcomings. However, the way in which this approach is employed can either be designed to maximise specificity by limiting reporting to combinations of known pathogenic mutations or can maximise sensitivity by also reporting combinations of pathogenic mutations together with variants of uncertain significance. The latter approach also increases the number of Cystic Fibrosis Screen-Positive Inconclusive Diagnosis (CFSPID) designations reported, resulting in uncertainty for parents. To help consider the design of the programme, a dialogue was commissioned by the UK National Screening Committee (UKNSC) to elicit the views of members of the public without direct experience of CF, to determine if there was a preference for maximising the sensitivity or the specificity of CF screening. The participants initially expressed a clear preference to maximise sensitivity and avoid missing CF cases, but after time to reflect and consider the implications of their choice, a number changed their views so as to tolerate some missed cases if this resulted in greater certainty of outcome; this became the majority view. It is proposed that it may be a generalisable finding that the public, when facing whole-population screening programmes, may require significant time and information to inform and make their choices and may attach great importance to clarity and certainty of outcome in the screening process.

Research Priorities for Pediatric Intensive Care Nutrition Within the United Kingdom: A National Institute of Health Research James Lind Alliance Priority Setting Partnership.

OBJECTIVES: To determine research priorities in PICU nutrition, which represent the shared priorities of patients, parents, carers, and PICU healthcare professionals within the United Kingdom. DESIGN: A national multiphase priority setting methodology in partnership with the James Lind Alliance delivered over 16 months (June 2020-September 2021). Part 1: a national scoping survey asked respondents to submit their research uncertainties related to PICU nutrition. Part 2: summarizing and evidence-checking the submitted uncertainties. Part 3: interim prioritization survey. Part 4: consensus workshop. SETTING: PICU. PARTICIPANTS: Patients, parents, and carers of patients who had been admitted to PICU, and PICU healthcare professionals involved in the treatment of these patients within the United Kingdom. INTERVENTIONS: None. MEASUREMENTS AND MAIN RESULTS: A national scoping survey asked respondents to submit their research uncertainties related to PICU nutrition. In the first survey, 165 topic ideas were suggested (12% by parents/carers and 88% by PICU healthcare professionals). These were categorized into 57 summary questions. The existing evidence was searched to ensure that the proposed summary questions had not already been answered. Forty were judged to be true uncertainties following a systematic literature review. These 40 uncertainties were grouped into eight themes for the second interim survey, which asked respondents to prioritize their top research questions. One hundred and forty participants contributed to this second interim survey. A final shortlist of 25 questions was derived, with the top 18 questions taken to a multistakeholder workshop where a consensus was reached on the top 10 priorities. CONCLUSIONS: This research identified important research gaps in the management of patients in PICU. Areas that need to be addressed as a priority include energy requirements in ventilated neonates, nutritional supplementation of probiotics to manage and prevent sepsis, the impact of postintensive care syndrome on nutrition and growth, and when to commence parenteral (IV) nutrition. The challenge now is to refine and deliver answers to these research priorities.

Religion and organ donation: the views of UK faith leaders.

This article reports the findings from the one-to-one interviews with the main UK faith and belief leaders which were commissioned by the Organ Donation Taskforce as part of its evidence gathering. Interviews were arranged with the main faith and belief organisations within the UK. Interviews covered a range of issues related to organ donation. Although some faith groups had some reservations regarding organ donation, interviews with these leaders demonstrated that none of these faith groups have reached a consensus against organ donation. The interviewees stated that the majority opinion in their faith or belief group is to permit organ donation, with some actively supporting it. Interviewees were keen to stress that there is a broad spectrum of opinion on organ transplantation within each faith and belief group and that consequently it is difficult to speak on behalf of an entire group. One complication mentioned by interviewees is that as organ transplantation is a relatively new medical procedure, there is no explicit reference to it in many original religious texts. Consequently, positions on the receipt and donation of organs are based on interpretation. It was felt that a much greater level of engagement is needed, as organ donation is currently not a priority for many faith and belief groups.

'Opting-in or opting-out?'--the views of the UK's faith leaders in relation to organ donation.

INTRODUCTION: This article reports the findings from the interviews with the main United Kingdom faith and belief leaders which were commissioned by the Organ Donation Taskforce as part of its evidence gathering when exploring the potential impact of an opt-out system for organ donation. METHODOLOGY: 17 interviews were arranged with the main faith and belief organisations within the United Kingdom. Interviews covered a range of issues related to organ donation and opt-in and opt-out. RESULTS: The majority of faith and belief leaders are supportive of the opt-in system, and favour retaining it over the introduction of an opt-out system. Whilst the shortcomings of the current system are recognised, the majority felt that there is scope to make improvements without changing legislation. CONCLUSION: In order to achieve better engagement on organ donation with members of faith and belief groups in the future, it will be important to continue the dialogue that has begun with these interviews. It is important not only for the public to be aware of the position of faith and belief leaders, but also just as important for the medical profession and policy makers to recognise that all United Kingdom faith and belief leaders interviewed wish organ donation and transplantation to be a success.

Faith leaders united in their support for organ donation: findings from the UK Organ Donation Taskforce study.

SUMMARY: This article reports the findings from the one-to-one interviews with the main UK faith and belief leaders, which were commissioned by the Organ Donation Taskforce. Interviews were arranged with the main faith and belief organizations within the UK and covered a range of issues related to organ donation. No faith or belief groups were against organ donation in principle. The interviewees stated that the majority opinion in their faith or belief group is to permit organ donation, with some actively supporting it. Interviewees were keen to stress that there is a broad spectrum of opinions on organ transplantation within each faith and belief group, and that consequently it is difficult to speak on behalf of an entire group. One complication mentioned by interviewees is that as organ transplantation is a relatively new medical procedure, there is no explicit reference to it in many original religious texts. Consequently positions on the receipt and donation of organs are based on interpretation. It was felt that a much greater level of engagement is needed, as organ donation is currently not a priority for many faith and belief groups.