Providing multimedia information to children and young people increases recruitment to trials: pre-planned meta-analysis of SWATs.

BACKGROUND: Randomised controlled trials are often beset by problems with poor recruitment and retention. Information to support decisions on trial participation is usually provided as printed participant information sheets (PIS), which are often long, technical, and unappealing. Multimedia information (MMI), including animations and videos, may be a valuable alternative or complement to a PIS. The Trials Engagement in Children and Adolescents (TRECA) study compared MMI to PIS to investigate the effects on participant recruitment, retention, and quality of decision-making. METHODS: We undertook six SWATs (Study Within A Trial) within a series of host trials recruiting children and young people. Potential participants in the host trials were randomly allocated to receive MMI-only, PIS-only, or combined MMI + PIS. We recorded the rates of recruitment and retention (varying between 6 and 26 weeks post-randomisation) in each host trial. Potential participants approached about each host trial were asked to complete a nine-item Decision-Making Questionnaire (DMQ) to indicate their evaluation of the information and their reasons for participation/non-participation. Odds ratios were calculated and combined in a meta-analysis. RESULTS: Data from 3/6 SWATs for which it was possible were combined in a meta-analysis (n = 1758). Potential participants allocated to MMI-only were more likely to be recruited to the host trial than those allocated to PIS-only (OR 1.54; 95% CI 1.05, 2.28; p = 0.03). Those allocated to combined MMI + PIS compared to PIS-only were no more likely to be recruited to the host trial (OR = 0.89; 95% CI 0.53, 1.50; p = 0.67). Providing MMI rather than PIS did not impact on DMQ scores. Once children and young people had been recruited to host trials, their trial retention rates did not differ according to intervention allocation. CONCLUSIONS: Providing MMI-only increased the trial recruitment rate compared to PIS-only but did not affect DMQ scores. Combined MMI + PIS instead of PIS had no effect on recruitment or retention. MMIs are a useful tool for trial recruitment in children and young people, and they could reduce trial recruitment periods.

An overview of interventions used across the life span to support transitions of those with a neurological condition: A systematic review of reviews.

OBJECTIVES: To systematically appraise published reviews on interventions used to support transitions for individuals with neurological conditions. DATA SOURCES: MEDLINE, CINAHL, The Allied and Complementary Medicine, AMED, PsycINFO, Cochrane database of systematic reviews and Web of Science were searched between 31st December 2010 and 15th September 2022. METHOD: The systematic review followed PRISMA guidelines. The quality and risk of bias were measured using A MeaSurement Tool to Assess systematic Reviews 2 and the Risk Of Bias In Systematic reviews' tool. All types of reviews which involved participants with neurological conditions were included. RESULTS: Seven reviews met the inclusion criteria. A total of 172 studies were included in the reviews. Effectiveness of transition interventions could not be calculated due to the lack of data. The findings suggested that the use of health applications may be beneficial by increasing self-management capabilities and disease knowledge. Education and clear communication between healthcare providers and recipients may also have positive impacts on quality of life. Risk of bias was found to be high in four of the reviews. Four reviews had low or critically low levels of evidence. CONCLUSIONS: There is a paucity of published evidence on interventions used to support the transitions of individuals with neurological conditions and the effect that these have on quality of life.

The economic burden experienced by carers of children who had a critical deterioration at a tertiary children's hospital in the United Kingdom (the DETECT study): an online survey.

BACKGROUND: Unplanned critical care admissions following in-hospital deterioration in children are expected to impose a significant burden for carers across a number of dimensions. One dimension relates to the financial and economic impact associated with the admission, from both direct out-of-pocket expenditures, as well as indirect costs, reflecting productivity losses. A robust assessment of these costs is key to understand the wider impact of interventions aiming to reduce in-patient deterioration. This work aims to determine the economic burden imposed on carers caring for hospitalised children that experience critical deterioration events. METHODS: Descriptive study with quantitative approach. Carers responded to an online survey between July 2020 and April 2021. The survey was developed by the research team and piloted before use. The sample comprised 71 carers of children admitted to a critical care unit following in-patient deterioration, at a tertiary children's hospital in the UK. The survey provides a characterisation of the carer's household and estimates of direct non-medical costs grouped in five different expenditure categories. Productivity losses can also be estimated based on the reported information. RESULTS: Most carers reported expenditures associated to the child's admission in the week preceding the survey completion. Two-thirds of working carers had missed at least one workday in the week prior to the survey completion. Moreover, eight in ten carers reported having had to travel from home to the hospital at least once a week. These expenditures, on average, amount to £164 per week, grouped in five categories (38% each to travelling costs and to food and drink costs, with accommodation, childcare, and parking representing 12%, 7% and 5%, respectively). Additionally, weekly productivity losses for working carers are estimated at £195. CONCLUSION: Unplanned critical care admissions for children impose a substantial financial burden for carers. Moreover, productivity losses imply a subsequent cost to society. Even though subsidised hospital parking and on-site accommodation at the hospital contribute to minimising such expenditure, the overall impact for carers remains high. Interventions aiming at reducing emergency critical care admissions, or their length, can be crucial to further contribute to the reduction of this burden. TRIAL REGISTRATION: Current Controlled Trials ISRCTN61279068, date of registration 07/06/2019, retrospectively registered.

A prospective, mixed-methods, before and after study to identify the evidence base for the core components of an effective Paediatric Early Warning System and the development of an implementation package containing those core recommendations for use in the UK: Paediatric early warning system - utilisation and mortality avoidance- the PUMA study protocol.

BACKGROUND: In hospital, staff need to routinely monitor patients to identify those who are seriously ill, so that they receive timely treatment to improve their condition. A Paediatric Early Warning System is a multi-faceted socio-technical system to detect deterioration in children, which may or may not include a track and trigger tool. It functions to monitor, detect and prompt an urgent response to signs of deterioration, with the aim of preventing morbidity and mortality. The purpose of this study is to develop an evidence-based improvement programme to optimise the effectiveness of Paediatric Early Warning Systems in different inpatient contexts, and to evaluate the feasibility and potential effectiveness of the programme in predicting deterioration and triggering timely interventions. METHODS: This study will be conducted in two district and two specialist children's hospitals. It deploys an Interrupted Time Series (ITS) design in conjunction with ethnographic cases studies with embedded process evaluation. Informed by Translational Mobilisation Theory and Normalisation Process Theory, the study is underpinned by a functions based approach to improvement. Workstream (1) will develop an evidence-based improvement programme to optimise Paediatric Early Warning System based on systematic reviews. Workstream (2) consists of observation and recording outcomes in current practice in the four sites, implementation of the improvement programme and concurrent process evaluation, and evaluation of the impact of the programme. Outcomes will be mortality and critical events, unplanned admission to Paediatric Intensive Care (PICU) or Paediatric High Dependency Unit (PHDU), cardiac arrest, respiratory arrest, medical emergencies requiring immediate assistance, reviews by PICU staff, and critical deterioration, with qualitative evidence of the impact of the intervention on Paediatric Early Warning System and learning from the implementation process. DISCUSSION: This paper presents the background, rationale and design for this mixed methods study. This will be the most comprehensive study of Paediatric Early Warning Systems and the first to deploy a functions-based approach to improvement in the UK with the aim to improve paediatric patient safety and reduce mortality. Our findings will inform recommendations about the safety processes for every hospital treating paediatric in-patients across the NHS. TRIAL REGISTRATION: Sponsor: Cardiff University, 30-36 Newport Road, Cardiff, CF24 0DE Sponsor ref.: SPON1362-14. Funder: National Institute for Health Research, Health Services & Delivery Research Programme (NIHR HS&DR) Funder reference: 12/178/17. Research Ethics Committee reference: 15/SW/0084 [13/04/2015]. PROSPERO reference: CRD42015015326 [23/01/2015]. ISRCTN: 94228292 https://doi.org/10.1186/ISRCTN94228292 [date of application 13/05/2015; date of registration: 18/08/2015]. Prospective registration prior to data collection and participant consent commencing in September 2014.

Parents' and young people's involvement in designing a trial of ventilator weaning.

Consulting with users is considered best practice and is highly recommended in designing new trials. As part of our feasibility work, we undertook a consultation exercise with parents, ex-patients and young people prior to designing a trial of protocol-based ventilator weaning. Our aims were to (1) ascertain views on the relevance and importance of the trial; (2) determine the important parent/patient outcome measures; and (3) ascertain views on informed consent in a cluster randomized controlled trial. We conducted audio-recorded face-to-face, telephone and focus group interviews with parents and young people. Data were content analysed to generate information to address our specific consultation objectives. The setting was the north-western region of England. A total of 16 participants were interviewed: 2 parents of paediatric intensive care unit (PICU) survivors; 1 PICU survivor; and 13 young people from the former Medicines for Children Research Network. The trial objectives were deemed important and relevant, and participants considered the most important outcome measure to be the length of time on ventilation. Parents and young people did not consider written informed consent to be a necessary requirement in the context of this trial, rather awareness of unit participation in the trial was important with the opportunity of opting out of data collection. This consultation provided useful, pragmatic insights to inform trial design. We encountered significant challenges in recruiting parents and young people for this consultation exercise, and novel recruitment methods need to be considered for future work in this field. Patient and public involvement is essential to ensure that future trials answer parent-relevant questions and have meaningful outcome measures, as well as involving parents and young people in the general development of health care services.

What does confidence mean to people who have had a stroke? A qualitative interview study.

OBJECTIVE: To explore the meaning of confidence to stroke patients after stroke in order to inform the development of a measurement tool. DESIGN: Qualitative interview study using interpretative phenomenological analysis (IPA). METHODS: Ten stroke survivors were purposively selected from those participating in a multi-centre randomised trial of outdoor mobility rehabilitation. Interviews about confidence were conducted in participants' homes, audio recorded and transcribed verbatim. RESULTS: Six themes emerged from the analysis. These were loss of identity, fear, social confidence, role confidence, mastering skill and attitudes and beliefs. Loss of identity was particularly evident in the early stages of stroke recovery. Fear was a barrier to regaining confidence and was associated with avoidance behaviours. Lack of social confidence was a common problem which appeared difficult to resolve. Life roles motivated participants to re-engage in daily life activities. Personal attitudes and beliefs, combined with the attitudes of significant others, contributed to personal feelings of competence. CONCLUSION: This study provides a coherent definition of the meaning of confidence through the experiences of stroke survivors. Being successful in gradually re-engaging in activities, including social activities and life roles helped to establish a positive self-belief. The influence of others, such as family and friends reinforce self-beliefs. Confidence and self-efficacy appear to be a similar construct. However, participants in this study also identified a relationship between confidence and self-esteem. The findings indicate that all six themes need to be included in a confidence measure to encompass the meaning of confidence as described by participants with stroke.

Exploring the perceptions and experiences of community rehabilitation for Long COVID from the perspectives of Scottish general practitioners' and people living with Long COVID: a qualitative study.

OBJECTIVES: To explore the experience of accessing Long COVID community rehabilitation from the perspectives of people with Long COVID and general practitioners (GPs). DESIGN: Qualitative descriptive study employing one-to-one semistructured virtual interviews analysed using the framework method. SETTING: Four National Health Service Scotland territorial health boards. PARTICIPANTS: 11 people with Long COVID (1 male, 10 female; aged 40-65 (mean 53) and 13 GPs (5 male, 8 female). RESULTS: Four key themes were identified: (1) The lived experience of Long COVID, describing the negative impact of Long COVID on participants' health and quality of life; (2) The challenges of an emergent and complex chronic condition, including uncertainties related to diagnosis and management; (3) Systemic challenges for Long COVID service delivery, including lack of clear pathways for access and referral, siloed services, limited resource and a perceived lack of holistic care, and (4) Perceptions and experiences of Long COVID and its management, including rehabilitation. In this theme, a lack of knowledge by GPs and people with Long COVID on the potential role of community rehabilitation for Long COVID was identified. Having prior knowledge of rehabilitation or being a healthcare professional appeared to facilitate access to community rehabilitation. Finally, people with Long COVID who had received rehabilitation had generally found it beneficial. CONCLUSIONS: There are several patient, GP and service-level barriers to accessing community rehabilitation for Long COVID. There is a need for greater understanding by the public, GPs and other potential referrers of the role of community rehabilitation professionals in the management of Long COVID. There is also a need for community rehabilitation services to be well promoted and accessible to the people with Long COVID for whom they may be appropriate. The findings of this study can be used by those (re)designing community rehabilitation services for people with Long COVID.

Investigating Scottish Long COVID community rehabilitation service models from the perspectives of people living with Long COVID and healthcare professionals: a qualitative descriptive study.

OBJECTIVES: This study aimed to explore the perceptions and experiences of barriers and facilitators to accessing Long COVID community rehabilitation. DESIGN: We used a qualitative descriptive design over two rounds of data collection with three participant groups: (1) people with experience of rehabilitation for Long COVID (PwLC); (2) National Health Service (NHS) staff delivering and/or managing community rehabilitation services (allied health professionals (AHPs)) and (3) NHS staff involved in strategic planning around Long COVID in their health board (Long COVID leads). SETTING: Four NHS Scotland territorial health boards. PARTICIPANTS: 51 interviews: eight Long COVID leads (11 interviews); 15 AHPs (25 interviews) and 15 PwLC (15 interviews). RESULTS: Three key themes were identified: (1) accessing care for PwLC, (2) understanding Long COVID and its management and (3) strengths and limitations of existing Long COVID rehabilitation services. CONCLUSIONS: Organisational delivery of Long COVID community rehabilitation is complex and presents multiple challenges. In addition, access to Long COVID community rehabilitation can be challenging. When accessed, these services are valued by PwLC but require adequate planning, publicity and resource. The findings presented here can be used by those developing and delivering services for people with Long COVID.

Quantifying upper limb tremor in people with multiple sclerosis using Fast Fourier Transform based analysis of wrist accelerometer signals.

INTRODUCTION: Tremor is a disabling symptom of Multiple Sclerosis (MS). The development of objective methods of tremor characterisation to assess intervention efficacy and disease progression is therefore important. The possibility of using a Fast Fourier Transform (FFT) method for tremor detection was explored. METHODS: Acceleration from a wrist-worn device was analysed using FFTs to identify and characterise tremor magnitude and frequency. Processing parameters were explored to provide insight into the optimal algorithm. Participants wore a wrist tri-axial accelerometer during 9 tasks. The FAHN clinical assessment of tremor was used as the reference standard. RESULTS: Five people with MS and tremor (57.6 ± 15.3 years, 3 F/2M) and ten disease-free controls (42.4 ± 10.9 years, 5 M/5F) took part. Using specific algorithm settings tremor identification was possible (peak frequency 3-15Hz; magnitude greater than 0.06 g; 2 s windows with 50% overlap; using 2 of 3 axes of acceleration), giving sensitivity 0.974 and specificity 0.971 (38 tremor occurrences out of 108 tasks, 1 false positive, 2 false negatives). Tremor had frequency 3.5-13.0 Hz and amplitude 0.07-2.60g. CONCLUSIONS: Upper limb tremor in people with MS can be detected using a FFT approach based on acceleration recorded at the wrist, demonstrating the possibility of using this minimally encumbering technique within clinical practice.

Can they stomach it? Parent and practitioner acceptability of a trial comparing gastric residual volume measurement versus no gastric residual volume in UK NNU and PICUs: a feasibility study.

BACKGROUND: Routine measurement of gastric residual volume (GRV) to guide feeding in neonatal and paediatric intensive care is widespread. However, this practice is not evidence based and may cause harm. As part of a feasibility study, we explored parent and practitioner views on the acceptability of a trial comparing GRV measurement or no GRV measurement. METHODS: A mixed-methods study involving interviews and focus groups with practitioners and interviews with parents with experience of tube feeding in neonatal and/or paediatric intensive care. A voting system recorded closed question responses during practitioner data collection, enabling the collection of quantitative and qualitative data. Data were analysed using thematic analysis and descriptive statistics. RESULTS: We interviewed 31 parents and nine practitioners and ran five practitioner focus groups (n=42). Participants described how the research question was logical, and the intervention would not be invasive and potential benefits of not withholding the child's feeds. However, both groups held concerns about the potential risk of not measuring GRV, including delayed diagnosis of infection and gut problems, increased risk of vomiting into lungs and causing discomfort or pain. Parent's views on GRV measurement and consent decision making were influenced by their views on the importance of feeding in the ICU, their child's prognosis and associated comorbidities or complications. CONCLUSIONS: The majority of parents and practitioners viewed the proposed trial as acceptable. Potential concerns and preferences were identified that will need careful consideration to inform the development of the proposed trial protocol and staff training.

Determining Optimal Outcome Measures in a Trial Investigating No Routine Gastric Residual Volume Measurement in Critically Ill Children.

BACKGROUND: Choosing trial outcome measures is important. When outcomes are not clinically relevant or important to parents/patients, trial evidence is less likely to be implemented into practice. This study aimed to determine optimal outcome measures for a trial of no routine gastric residual volume (GRV) measurement in critically ill children. METHODS: A mixed-methods approach was used: a focused literature review, parent and clinician interviews, a modified 2-round Delphi, and a stakeholder consensus meeting. RESULTS: The review generated 13 outcomes. Fourteen pediatric intensive care unit (PICU) parents proposed 3 additional outcomes; these 16 were then rated by 28 clinicians in Delphi round 1. Six further outcomes were proposed, and 22 outcomes were rated in the second round. No items were voted "consensus out." The 18 "no-consensus" items were voted in a face-to-face meeting by 30 participants. The final 12 outcome measures were time to reach energy targets, ventilator-associated pneumonia, vomiting, time enteral feeds withheld per 24 hours, necrotizing enterocolitis, length of invasive ventilation, PICU length of stay, mortality, change in weight and markers of feed intolerance (parenteral nutrition administered), feed formula altered, and change to postpyloric feeds all secondary to feed intolerance. CONCLUSION: We have identified 12 outcomes for a trial of no GRV measurement through a multistage process, seeking views of parents and clinicians.

Clinical trials in amyotrophic lateral sclerosis: a systematic review and perspective.

Amyotrophic lateral sclerosis is a progressive and devastating neurodegenerative disease. Despite decades of clinical trials, effective disease-modifying drugs remain scarce. To understand the challenges of trial design and delivery, we performed a systematic review of Phase II, Phase II/III and Phase III amyotrophic lateral sclerosis clinical drug trials on trial registries and PubMed between 2008 and 2019. We identified 125 trials, investigating 76 drugs and recruiting more than 15 000 people with amyotrophic lateral sclerosis. About 90% of trials used traditional fixed designs. The limitations in understanding of disease biology, outcome measures, resources and barriers to trial participation in a rapidly progressive, disabling and heterogenous disease hindered timely and definitive evaluation of drugs in two-arm trials. Innovative trial designs, especially adaptive platform trials may offer significant efficiency gains to this end. We propose a flexible and scalable multi-arm, multi-stage trial platform where opportunities to participate in a clinical trial can become the default for people with amyotrophic lateral sclerosis.

A national survey of community rehabilitation service provision for people with long Covid in Scotland.

Background: Over 50 million cases of COVID-19 have been confirmed globally as of November 2020. Evidence is rapidly emerging on the epidemiology of COVID-19, and its impact on individuals and potential burden on health services and society. Between 10-35% of people with COVID-19 may experience post-acute long Covid. This currently equates to between 8,129 and 28,453 people in Scotland. Some of these people will require rehabilitation to support their recovery. Currently, we do not know how to optimally configure community rehabilitation services for people with long Covid. Methods: This national survey aimed to provide a detailed description of current community rehabilitation provision for people with long Covid in Scotland. We developed, piloted, and conducted a national electronic survey of current community rehabilitation service provision for people presenting with long Covid symptomatology. Our sample were the Allied Health Professions Directors of all 14 territorial NHS Health Boards in Scotland. Fixed response and narrative data were analysed descriptively. Results: Responses were received from all respondents (14/14), enabling a national picture to be gained. Almost all Health Boards (13/14) currently deliver rehabilitation for people with long Covid within pre-existing services. Fatigue (11/14) and respiratory conditions (9/14) were the two most common presenting problems of patients. Most long Covid community rehabilitation services are delivered through a combination of face-to-face and digital contact (13/14). Conclusions: Community rehabilitation for people with long Covid is an emerging reality. This survey provides a national picture of current community rehabilitation for people with long Covid. We do not know how community rehabilitation can be optimally delivered for this population. This is vital as community rehabilitation services were already under pressure prior to the emergence of COVID-19. Further research is urgently required to investigate the implementation, outcomes and cost-effectiveness of differing models of community rehabilitation for this patient population.

Ensuring young voices are heard in core outcome set development: international workshops with 70 children and young people.

PLAIN ENGLISH SUMMARY: Researchers test treatments to ensure these work and are safe. They do this by studying the effects that treatments have on patients by measuring outcomes, such as pain and quality of life. Often research teams measure different outcomes even though each team is studying the same condition. This makes it hard to compare the findings from different studies and it can reduce the accuracy of the treatment advice available to patients. Increasingly, researchers are tackling this problem by developing 'core outcome sets'. These are lists of outcomes that all researchers working on a given condition should measure in their studies. It is important that patients have a voice in the development of core outcome sets and children and young people are no exception. But their voices have rarely been heard when core outcome sets are developed. Researchers are trying to address this problem and make sure that core outcome sets are developed in ways that are suitable for children and young people. As a first step, we held two international workshops with children and young people to listen to their views. They emphasised the importance of motivating young people to participate in developing core outcome sets, making them feel valued, and making the development process more interactive, enjoyable and convenient. We hope this commentary will encourage researchers to include children and young people when developing core outcome sets and to adapt their methods so these are suitable for young participants. Future research is important to examine whether these adaptations are effective. ABSTRACT: Background Different research teams looking at treatments for the same condition often select and measure inconsistent treatment outcomes. This makes it difficult to synthesise the results of different studies, leads to selective outcome reporting and impairs the quality of evidence about treatments. 'Core outcome sets' (COS) can help to address these problems. A COS is an agreed, minimum list of outcomes that researchers are encouraged to consistently measure and report in their studies. Including children and young people (CYP) as participants in the development of COS for paediatric conditions ensures that clinically meaningful outcomes are measured and reported. However, few published COS have included CYP as participants. COS developers have described difficulties in recruiting and retaining CYP and there is a lack of guidance on optimising COS methods for them. We aimed to explore CYP's views on the methods used to develop COS and identify ways to optimise these methods.Main body This commentary summarises discussions during two workshops with approximately 70 CYP (aged 10-18 years old) at the International Children's Advisory Network Research and Advocacy Summit, 2018. Delegates described what might motivate them to participate in a COS study, including feeling valued, understanding the need for COS and the importance of input from CYP in their development, and financial and other incentives (e.g. certificates of participation). For Delphi surveys, delegates suggested that lists of outcomes should be as brief as possible, and that scoring and feedback methods should be simplified. For consensus meetings, delegates advised preparing CYP in advance, supporting them during meetings (e.g. via mentors) and favoured arrangements whereby CYP could meet separately from parents and other stakeholders. Overall, they wanted COS methods that were convenient, enjoyable and engaging.Conclusion This commentary points to the limitations of the methods currently used to develop COS with CYP. It also points to ways to motivate CYP to participate in COS studies and to enhancements of methods to make participation more engaging for CYP. Pending much needed research on COS methods for CYP, the perspectives offered in the workshops should help teams developing COS in paediatrics and child health.

How to involve children and young people in what is, after all, their research.

Third in a series of four articles addressing ethical approaches and issues in undertaking clinical research with children and young people.

Supporting children and young people when making decisions about joining clinical trials: qualitative study to inform multimedia website development.

OBJECTIVES: To understand stakeholders' views regarding the content and design of paediatric clinical trial multimedia websites. To describe how this knowledge informed the development of the multimedia websites. DESIGN: Qualitative study comprising two rounds of interviews or focus groups, with thematic analysis of interview transcripts. PARTICIPANTS: Sixty-two people (21 children and young people with long-term health conditions, 24 parents and 17 professionals). SETTING: One UK children's hospital and one UK Young Persons' Advisory Group. RESULTS: When asked what was important in deciding whether to join a trial, children, young people and parents prioritised information about what participation would involve, what the trial was testing, potential benefits and risks of participation and knowing they could leave the trial if they later changed their minds. Young people and parents trusted trial teams to follow regulatory and quality requirements and therefore did not think such information was a priority for the websites, although logos of trusted organisations could lend credibility. Professionals largely concurred with these views. Children and young people advised on the importance of designing the multimedia website to ensure its appearance, tone and wording suited the intended audience and on using animated characters to facilitate children's engagement. CONCLUSIONS: Our study provides insights into the information that families value when deciding about healthcare trial participation. It provides guidance on the design of information resources to appeal to children and young people, while also being acceptable to parents and professionals who are often gatekeepers of children's access to information. Our findings will be of use to others developing similar multimedia websites. We report specific information needs and new visual preferences that are not usually addressed in printed trial information. Our work illustrates what qualitative research and participatory design practices can contribute to the development of information resources more generally. TRIAL REGISTRATION NUMBER: ISRCTN73136092; Pre-results.

Clinical audit research and evaluation of motor neuron disease (CARE-MND): a national electronic platform for prospective, longitudinal monitoring of MND in Scotland.

Objectives: Launched in 1989, the Scottish Motor Neuron Disease Register (SMNDR) has provided a resource for prospective clinical data collection. However, in 2015 we aimed to evolve a system to allow: i) A patient-centered approach to care based on recognized standards, ii) Harmonized data sharing between Scottish health professionals in "real-time", iii) Regular audit of care to facilitate timely improvements in service delivery, and iv) Patient participation in a diverse range of observational and interventional research studies including clinical trials. Methods: We developed a standardized national electronic data platform-Clinical Audit Research and Evaluation of MND (CARE-MND) which integrates clinical audit and research data fields. Data completion pre- and post-CARE-MND were compared, guided by recently published National Institute for Clinical Excellence (NICE) recommendations. Statistical difference in data capture between time periods was assessed using Z-test of proportions. Results: Data field completion for the historical 2011-2014 period ranged from 4 to 95%; median 50%. CARE-MND capture ranged from 32 to 98%; median 87%. 15/17 fields were significantly more complete post-CARE-MND (p < 0.001). All MND nurse/allied health specialists in Scotland use the CARE-MND platform. Management of MND in Scotland is now coordinated through a standardized template. Conclusions: Through CARE-MND, national audits of MND care and interventions have been possible, leading to protocols for harmonized service provision. Stratification of the MND population is facilitating participation in observational and interventional studies. CARE-MND can act as a template for other neurological disorders.

Involving children and young people in clinical research through the forum of a European Young Persons' Advisory Group: needs and challenges.

Children and young people are seen as fundamental to the design and delivery of clinical research as active and reflective participants. In Europe, involvement of children and young people in clinical research is promoted extensively in order to engage young people in research as partners and to give them a voice to raise their own issues or opinions and for their involvement in planning and decision making in addition to learning research skills. Children and young people can be trained in clinical research through participation in young person advisory groups (YPAGs). Members of YPAGs assist other children and young people to learn about clinical research and share their experience and point of view with researchers, thereby possibly influencing all phases of research including the development and prioritization of research questions, design and methods, recruitment plans, and strategies for results dissemination. In the long term, the expansion of YPAGs in Europe will serve as a driving force for refining pediatric clinical research. It will help in a better definition of research projects according to the patients' needs. Furthermore, direct engagement of children and young people in research will be favorable to both researchers and young people.