The effectiveness of parent training for children with autism spectrum disorder: a systematic review and meta-analyses.

BACKGROUND: Various parent training interventions have been shown to have some effect on the symptoms of children with autism. We carried out a systematic review and meta-analyses to assess effectiveness of parental training for children with autism on their symptoms and parental stress. METHODS: Four electronic databases, CINAHL, EMBASE, MEDLINE and PsycINFO were searched until March 2020 for relevant literature. Two reviewers independently screened bibliographies using an eligibility checklist and extracted data using a structured proforma. We have also carried out meta-analyses when data were available for pooling. RESULTS: Seventeen papers from 15 studies were included for data analysis. Fifteen papers showed a positive treatment effect when compared with the control group, although not always significant. Meta-analysis based on pooled data from only two studies in each respective intervention, showed small to moderate treatment effects for three interventions, DIR/Floortime, Pivotal Response and Parent focused training respectively. CONCLUSIONS: As in previous systematic reviews there was a mild to moderate treatment effects of three specific types of interventions respectively. However, it was difficult to draw any definitive conclusion about the effectiveness and generalisability of any intervention because of the wide variation in the interventions, control groups, outcome measures, small sample size, small number of studies in meta-analysis, overlap between the intervention and control procedures used in the included studies. There is an urgent need for experts in various international centres to jointly standardise a parent training intervention for children with autism and carry out a large scale RCT to assess its clinical and economic effectiveness. Research Registry Unique Identifying Number: reviewregistry915.

Care providers' and patients' attitudes toward using electronic-patient reported outcomes to support patients with traumatic brain injury: a qualitative study (PRiORiTy).

OBJECTIVES: To (a) identify residual symptoms and deficits resulting from a traumatic brain injury (TBI) and impact on patients' and their families' quality of life; (b) explore views and experience of care providers, researchers, patients, and carers of using PROMs; and (c) explore their attitudes toward reporting symptoms and impacts on an electronic platform. Methods: Qualitative semi-structured interviews with people with TBI and their carers; health-care professionals, researchers, and third sector staff members working with people with TBI. Results: Symptoms and long-term impacts of TBI included cognitive problems, difficulties functioning, anxiety, and depression. PROMs were seen as improving knowledge of residual symptoms and their impact post-TBI but not always accurately reflecting patients' residual problems. Challenges to completing PROMs were cognitive impairment and lack of insight into condition. Perceived advantages of an electronic platform included easy data collection; flexibility; improving workflow; and the ability to send/ receive feedback and reminders easily. Suggested features of an electronic platform included simple layout, lay language, short questions, few items on the screen, and capability to send/receive feedback and additional information. Conclusion: There is a demand for reporting symptoms and their impact electronically, providing the layout is kept simple and feedback from clinicians is provided.

Refining a model of collaborative care for people with a diagnosis of bipolar, schizophrenia or other psychoses in England: a qualitative formative evaluation.

BACKGROUND: Many people diagnosed with schizophrenia, bipolar or other psychoses in England receive the majority of their healthcare from primary care. Primary care practitioners may not be well equipped to meet their needs and there is often poor communication with secondary care. Collaborative care is a promising alternative model but has not been trialled specifically with this service user group in England. Collaborative care for other mental health conditions has not been widely implemented despite evidence of its effectiveness. We carried out a formative evaluation of the PARTNERS model of collaborative care, with the aim of establishing barriers and facilitators to delivery, identifying implementation support requirements and testing the initial programme theory. METHODS: The PARTNERS intervention was delivered on a small scale in three sites. Qualitative data was collected from primary and secondary care practitioners, service users and family carers, using semi-structured interviews, session recordings and tape-assisted recall. Deductive and inductive thematic analysis was carried out; themes were compared to the programme theory and used to inform an implementation support strategy. RESULTS: Key components of the intervention that were not consistently delivered as intended were: interaction with primary care teams, the use of coaching, and supervision. Barriers and facilitators identified were related to service commitment, care partner skills, supervisor understanding and service user motivation. An implementation support strategy was developed, with researcher facilitation of communication and supervision and additional training for practitioners. Some components of the intervention were not experienced as intended; this appeared to reflect difficulties with operationalising the intervention. Analysis of data relating to the intended outcomes of the intervention indicated that the mechanisms proposed in the programme theory had operated as expected. CONCLUSIONS: Additional implementation support is likely to be required for the PARTNERS model to be delivered; the effectiveness of such support may be affected by practitioner and service user readiness to change. There is also a need to test the programme theory more fully. These issues will be addressed in the process evaluation of our full trial. TRIAL REGISTRATION: ISRCTN95702682 , 26 October 2017.

Maternal filicide in a cohort of English Serious Case Reviews.

A national mixed-methods study of English Serious Case Reviews (SCRs) was carried out to better understand the characteristics and circumstances of maternally perpetrated filicides, to compare these with paternally perpetrated cases, and to identify learning points for mental health professionals. Published reports for all SCRs of children in England dying as a result of abuse or neglect from 2011 to 2014 were subject to qualitative analysis using a system of layered reading and inductive thematic analysis, along with descriptive and comparative quantitative analysis. There were 86 deaths directly attributable to child maltreatment within the immediate family. The mother was the suspected perpetrator in 20. Twelve of the mother perpetrators were victims of domestic violence, while 15 of the father perpetrators were known to be perpetrators of domestic violence. Those deaths resulting from impulsive violence or severe, persistent cruelty are almost exclusively perpetrated by males, while those with an apparent intent to kill the child are slightly more likely to be perpetrated by mothers. Four key themes were identified through the qualitative analysis: domestic violence, maternal mental illness, separation and maternal isolation, and the invisibility of the child. These findings highlight the important role of domestic violence and its interaction with maternal mental health. Professionals working with mothers with mental health problems need to adopt a supportive but professionally curious stance, to be alert to signs of escalating stress or worsening mental ill-health, and to provide supportive and accessible structures for at-risk families.

Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials - insights from an expert virtual roundtable.

Early phase clinical trials provide an initial evaluation of therapies' risks and benefits to patients, including safety and tolerability, which typically relies on reporting outcomes by investigator and laboratory assessments. Use of patient-reported outcomes (PROs) to inform risks (tolerability) and benefits (improvement in disease symptoms) is more common in later than early phase trials. We convened a two-day expert roundtable covering: (1) the necessity and feasibility of a universal PRO core conceptual model for early phase trials; (2) the practical integration of PROs in early phase trials to inform tolerability assessment, guide dose decisions, or as real-time safety alerts to enhance investigator-reported adverse events. Participants (n = 22) included: patient advocates, regulators, clinicians, statisticians, pharmaceutical representatives, and PRO methodologists working across diverse clinical areas. In this manuscript, we report major recommendations resulting from the roundtable discussions corresponding to each theme. Additionally, we highlight priority areas necessitating further investigation.

Extended brief interventions for weight management and obesity prevention in children: A rapid evidence review.

Uptake of child weight management (CWM) support is typically low, and services are not available in all areas. Extended brief interventions (EBIs) have been proposed as an affordable way to provide enhanced support, at a level between one-off brief advice and intensive CWM programs. This rapid systematic review sought to synthesize evidence on the efficacy of EBIs for weight management and obesity prevention in children (2-18 years). Embase and Web of Science were searched from January 2012 to January 2022. Nineteen studies, reporting on 17 separate EBIs, were included. The quality of studies was variable, and the EBIs were heterogeneous. The majority of EBIs (n = 14) were based on motivational interviewing. Five of the included studies reported significant improvements in parent or child determinants of health behavior change. However, robust measures of behavioral determinants were rarely used. No studies reported significant positive effects on child weight. No clear patterns in outcomes were identified. There is currently insufficient evidence for EBIs to be adopted as part of CWM services. To improve the evidence base, EBIs that are currently being implemented by local health services, should be evaluated to establish the most effective content, how it should be delivered, and by whom.

Retaining public health volunteers beyond COVID-19.

OBJECTIVES: The COVID-19 pandemic has led to a change in people's volunteering behaviours; participation has increased in informal volunteering (giving unpaid help to those who are not a relative) while decreasing in formal volunteering (unpaid help to groups or clubs). There is an interest from stakeholders who have experienced increased participation in maintaining the positive patterns of volunteering, aligning with National Health Service (NHS) objectives and realising benefits in a wider public health context. This research uses a local COVID-19 public health volunteering programme case study to explore the volunteer's journey and perspective using volunteers' reported experiences to consider the potential for volunteer retention and role expansion into other public health issues beyond the COVID-19 pandemic. METHODS: Recruitment was undertaken by Birmingham City Council Public Health Team via the COVID-19 Community Champions programme mailing list. Semi-structured focus group discussions, one-to-one interviews and email interviews were conducted with volunteers. Data were analysed through directed thematic analysis using an iteratively developed coding frame. RESULTS: Data were collected from three focus group discussions, four interviews, and one email interview involving a total of 16 participants. Six themes were identified: volunteer motivations and expectations; volunteer management; programme organisation; feeling valued; continued need for role, and interest in new responsibilities. CONCLUSION: Our findings indicate that the factors which are conducive to volunteer recruitment, retention and re-purposing were: maintaining the original terms of engaging with the volunteering opportunity (including retaining the original brief and remit), adjusting these through consultative processes with an emphasis on seeking permission from the volunteers already involved and ensuring a reliable and consistent management and support structure. While some of the learning is specific to the local volunteer programme in question and the context of the COVID-19 pandemic, there are lessons that can be generalised to other scenarios and settings.

Identifying patient-valued outcomes for use in early phase trials of ocular surface disease interventions.

BACKGROUND: Patient-reported outcomes (PROs) can be used to evaluate the impact of dry eye symptoms (DES) on daily life. Early-phase clinical trials provide an opportunity to evaluate PRO strategies. Existing measures identified through systematic review omitted important concepts that mattered to patients. The aim of our work was to develop a conceptual map of DES and assess the relative importance of identified concepts. METHODS: Web-based group concept mapping software was used to develop a conceptual map. This semi-quantitative mixed-methods approach consists of three stages 1) statement generation, 2) thematic sorting, 3) rating of statements for importance [1 (not important), 2 (important), 3 (very important)] and relevance [1 (not my experience), 2 (sometimes my experience); 3 (definitely my experience)]. Thirty-nine participants were recruited from two UK-based patient support groups (British Sjögren's Syndrome Association, PemFriends). Three withdrew, two for health reasons and one struggled with the web-based format. RESULTS: 125 statements and six thematic clusters were generated. The Environmental Impacts cluster scored highest for importance (2.45), followed by Pain and Discomfort (2.35), Eye Treatments (2.32), Daily Impact (2.07), Psychosocial Issues (1.78) and Miscellaneous (1.78). Mapping statements against existing PRO measures confirmed a number of important missing issues including the impact of 'UV levels' (2.50), hot dry weather (2.33), the temporal aspects of pain (2.64), and issues with night-driving (2.59). CONCLUSIONS: Group concept mapping identified important issues for people living with DES not currently captured by existing PROs, highlighting the need for additional PRO items to be considered for use in clinical trials.

Development of a core outcome set for use in adult primary glioma phase III interventional trials: A mixed methods study.

Background: Glioma interventional studies should collect data aligned with patient priorities, enabling treatment benefit assessment and informed decision-making. This requires effective data synthesis and meta-analyses, underpinned by consistent trial outcome measurement, analysis, and reporting. Development of a core outcome set (COS) may contribute to a solution. Methods: A 5-stage process was used to develop a COS for glioma trials from the UK perspective. Outcome lists were generated in stages 1: a trial registry review and systematic review of qualitative studies and 2: interviews with glioma patients and caregivers. In stage 3, the outcome lists were de-duplicated with accessible terminology, in stage 4 outcomes were rated via a 2-round Delphi process, and stage 5 comprised a consensus meeting to finalize the COS. Patient-reportable COS outcomes were identified. Results: In Delphi round 1, 96 participants rated 35 outcomes identified in stages 1 and 2, to which a further 10 were added. Participants (77/96) rated the resulting 45 outcomes in round 2. Of these, 22 outcomes met a priori threshold for inclusion in the COS. After further review, a COS consisting of 19 outcomes grouped into 7 outcome domains (survival, adverse events, activities of daily living, health-related quality of life, seizure activity, cognitive function, and physical function) was finalized by 13 participants at the consensus meeting. Conclusions: A COS for glioma trials was developed, comprising 7 outcome domains. Additional research will identify appropriate measurement tools and further validate this COS.

A toolkit for capturing a representative and equitable sample in health research.

Research participants often do not represent the general population. Systematic exclusion of particular groups from research limits the generalizability of research findings and perpetuates health inequalities. Groups considered underserved by research include those whose inclusion is lower than expected based on population estimates, those with a high healthcare burden but limited research participation opportunities and those whose healthcare engagement is less than others. The REP-EQUITY toolkit guides representative and equitable inclusion in research. The toolkit was developed through a methodological systematic review and synthesis and finalized in a consensus workshop with 24 participants. The REP-EQUITY toolkit describes seven steps for investigators to consider in facilitating representative and equitable sample selection. This includes clearly defining (1) the relevant underserved groups, (2) the aims relating to equity and representativeness, (3) the sample proportion of individuals with characteristics associated with being underserved by research, (4) the recruitment goals, (5) the strategies by which external factors will be managed, (6) the methods by which representation in the final sample will be evaluated and (7) the legacy of having used the toolkit. Using the REP-EQUITY toolkit could promote trust between communities and research institutions, increase diverse participation in research and improve the generalizability of health research. National Institute for Health and Care Research PROSPERO identifier: CRD42022355391.

Development and usability testing of an electronic patient-reported outcome (ePRO) solution for patients with inflammatory diseases in an Advanced Therapy Medicinal Product (ATMP) basket trial.

BACKGROUND: Electronic patient-reported outcome (ePRO) systems are increasingly used in clinical trials to provide evidence of efficacy and tolerability of treatment from the patient perspective. The aim of this study is twofold: (1) to describe how we developed an electronic platform for patients to report their symptoms, and (2) to develop and undertake usability testing of an ePRO solution for use in a study of cell therapy seeking to provide early evidence of efficacy and tolerability of treatment and test the feasibility of the system for use in later phase studies. METHODS: An ePRO system was designed to be used in a single arm, multi-centre, phase II basket trial investigating the safety and activity of the use of ORBCEL-C™ in the treatment of patients with inflammatory conditions. ORBCEL-C™ is an enriched Mesenchymal Stromal Cells product isolated from human umbilical cord tissue using CD362+ cell selection. Usability testing sessions were conducted using cognitive interviews and the 'Think Aloud' method with patient advisory group members and Research Nurses to assess the usability of the system. RESULTS: Nine patient partners and seven research nurses took part in one usability testing session. Measures of fatigue and health-related quality of life, the PRO-CTCAE™ and FACT-GP5 global tolerability question were included in the ePRO system. Alert notifications to the clinical team were triggered by PRO-CTCAE™ and FACT-GP5 scores. Patient participants liked the simplicity and responsiveness of the patient-facing app. Two patients were unable to complete the testing session, due to technical issues. Research Nurses suggested minor modifications to improve functionality and the layout of the clinician dashboard and the training materials. CONCLUSION: By testing the effectiveness, efficiency, and satisfaction of our novel ePRO system (PROmicsR), we learnt that most people with an inflammatory condition found it easy to report their symptoms using an app on their own device. Their experiences using the PROmicsR ePRO system within a trial environment will be further explored in our upcoming feasibility testing. Research nurses were also positive and found the clinical dashboard easy-to-use. Using ePROs in early phase trials is important in order to provide evidence of therapeutic responses and tolerability, increase the evidence based, and inform methodology development. TRIAL REGISTRATION: ISRCTN, ISRCTN80103507. Registered 01 April 2022, https://www.isrctn.com/ISRCTN80103507.

More and more patients tell clinicians how they feel by completing questionnaires electronically. Therefore, it is important to assess how easy it is for patients to do this. In this study, we describe how we developed an electronic platform for patients to report their symptoms and how we tested the usability of this platform with patient partners and research nurses. Once the electronic platform was developed, quality of life and symptoms questionnaires were programmed onto it. Alerts were sent to the clinical team if specific scores were obtained on the symptoms questionnaires. Although two patient partners were not able to finish the testing session because of technical issues, the ones who completed the session liked its simplicity and responsiveness. The research nurses also liked the system and only suggested minor modifications. Following this testing, we refined the electronic platform to test it further in a larger study which investigates the safety and use of a drug. We hope that thanks to this electronic platform, we will obtain useful information on the safety and efficacy of treatment.

Real-time remote outpatient consultations in secondary and tertiary care: A systematic review of inequalities in invitation and uptake.

BACKGROUND: Health policies in most high income countries increasingly recommend provision of routine outpatient care via remote (video and/or telephone) appointments, especially due to the pandemic. This is thought to improve access to care and promote efficiency within resource-constrained health services. There is limited evidence about the impact on existing inequalities in the invitation and uptake of health services when remote outpatient care is offered. AIM: To systematically review the evidence on the offer and/or uptake of real-time remote outpatient consultations in secondary and tertiary care, assessed according to key sociodemographic characteristics. METHODS: Seven electronic bibliographic databases were searched for studies reporting the proportion of patients with key characteristics (following PROGRESS Plus criteria) who were offered and/or accepted real-time remote outpatient consultation for any chronic condition. Comparison groups included usual care (face-to-face), another intervention, or offer/uptake within a comparable time period. Study processes were undertaken in duplicate. Data are reported narratively. RESULTS: Twenty-nine studies were included. Uptake of video consultations ranged from 5% to 78% and telephone consultations from 12% to 78%. Patients aged over 65, with lower educational attainment, on lower household incomes and without English as a first language were least likely to have a remote consultation. Females were generally more likely to have remote consultations than males. Non-white ethnicities were less likely to use remote consultations but where they did, were significantly more likely to choose telephone over video appointments (p<0.001). CONCLUSIONS: Offering remote consultations may perpetuate or exacerbate existing health inequalities in access to healthcare. More research is needed on current health disparities by sociodemographic characteristics and to explore what works well for different patient groups and why so that processes can be designed to ameliorate these health disparities. TRIAL REGISTRATION: PROSPERO registration no: CRD42021241791.

Outcome selection for tissue-agnostic drug trials for immune-mediated inflammatory diseases: a systematic review of core outcome sets and regulatory guidance.

BACKGROUND: Tissue-agnostic drug development provides a paradigm shift in precision medicine and requires innovative trial designs. However, outcome selection for such trials can prove challenging. The objectives of this review were to: (i) Identify and map core outcome sets (COS), across 11 immune-mediated inflammatory diseases (IMIDs) in order to facilitate the selection of relevant outcomes across the conditions for innovative trials of tissue-agnostic drug therapies. (ii) Compare outcomes or endpoints recommended by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) to identify and highlight similarities and differences. METHODS: The Core Outcome Measures in Effectiveness Trials (COMET), International Consortium for Health Outcomes Measurement (ICHOM), FDA and EMA databases were searched from inception to 28th December 2019. Two reviewers independently screened titles and abstracts of retrieved entries and conducted the subsequent full text screening. Hand searching of the reference lists and citation searching of the selected publications was conducted. The methodological quality of the included peer-reviewed articles was independently assessed by the reviewers based on the items of the COS-Standards for Development recommendations (COS-STAD) checklist. Core outcomes from the included publications were extracted and mapped across studies and conditions. Regulatory guidance from FDA and EMA, where available for clinical trials for the IMIDs, were obtained from their databases and recommendations on outcomes to measure directly compared. RESULTS: Forty-four COS publications were included in the final analysis. Outcomes such as disease activity, pain, fatigue, quality of life, physical function, work limitation/productivity, steroid use and biomarkers were recommended across majority of the conditions. There were significant similarities and differences in FDA and EMA recommendations. The only instance where either regulatory body directly referenced a COS was for jSLE-both referenced the Paediatric Rheumatology International Trials Organization (PRINTO) COS. CONCLUSIONS: The findings from this systematic review provide valuable information to inform outcome selection in tissue-agnostic trials for IMIDs. There is a need for increased collaboration between regulators and COS developers and inclusion of regulators as key stakeholders in COS development to enhance the quality of COS. TRIAL REGISTRATION: Not registered.

Feasibility of a new electronic patient-reported outcome (ePRO) system for an advanced therapy clinical trial in immune-mediated inflammatory disease (PROmics): protocol for a qualitative feasibility study.

INTRODUCTION: The use of electronic patient-reported outcome (ePRO) systems to capture PRO data in clinical trials is increasing; however, their feasibility, acceptability and utility in clinical trials of advanced therapy medicinal products (ATMPs) are not yet well understood. This protocol describes a qualitative study that aims to evaluate the feasibility and acceptability of ePRO data capture using a trial-specific ePRO system (the PROmics system) within an advanced therapy trial involving patients with immune-mediated inflammatory disease (rheumatoid arthritis, lupus, primary sclerosing cholangitis (PSC) and Crohn's disease). METHODS AND ANALYSIS: This protocol for a remote, qualitative, interview-based feasibility study is embedded within the POLARISE trial, a single-arm, phase II, multisite ATMP basket trial in the UK. 10-15 patients enrolled in the POLARISE trial and 10-15 research team members at the trial sites will be recruited. Participants will take part in semistructured interviews which will be transcribed verbatim and analysed thematically according to the framework method. Data collection and analysis will occur concurrently and iteratively. Researcher triangulation will be used to achieve a consensus-based analysis, enhancing rigour and trustworthiness. ETHICS AND DISSEMINATION: This study was approved by the London-West London and GTAC Research Ethics Committee (Ref: 21/LO/0475). Informed consent will be obtained from all participants prior to data collection. The study findings will be published in peer-review journals and disseminated via conference presentations and other media. Our patient and public involvement and engagement group and ATMP stakeholder networks will be consulted to maximise dissemination and impact. TRIAL REGISTRATION NUMBER: ISRCTN80103507.

Development of a core outcome set and identification of patient-reportable outcomes for primary brain tumour trials: protocol for the COBra study.

INTRODUCTION: Primary brain tumours, specifically gliomas, are a rare disease group. The disease and treatment negatively impacts on patients and those close to them. The high rates of physical and cognitive morbidity differ from other cancers causing reduced health-related quality of life. Glioma trials using outcomes that allow holistic analysis of treatment benefits and risks enable informed care decisions. Currently, outcome assessment in glioma trials is inconsistent, hindering evidence synthesis. A core outcome set (COS) - an agreed minimum set of outcomes to be measured and reported - may address this. International initiatives focus on defining core outcomes assessments across brain tumour types. This protocol describes the development of a COS involving UK stakeholders for use in glioma trials, applicable across glioma types, with provision to identify subsets as required. Due to stakeholder interest in data reported from the patient perspective, outcomes from the COS that can be patient-reported will be identified. METHODS AND ANALYSIS: Stage I: (1) trial registry review to identify outcomes collected in glioma trials and (2) systematic review of qualitative literature exploring glioma patient and key stakeholder research priorities. Stage II: semi-structured interviews with glioma patients and caregivers. Outcome lists will be generated from stages I and II. Stage III: study team will remove duplicate items from the outcome lists and ensure accessible terminology for inclusion in the Delphi survey. Stage IV: a two-round Delphi process whereby the outcomes will be rated by key stakeholders. Stage V: a consensus meeting where participants will finalise the COS. The study team will identify the COS outcomes that can be patient-reported. Further research is needed to match patient-reported outcomes to available measures. ETHICS AND DISSEMINATION: Ethical approval was obtained (REF SMREC 21/59, Cardiff University School of Medicine Research Ethics Committee). Study findings will be disseminated widely through conferences and journal publication. The final COS will be adopted and promoted by patient and carer groups and its use by funders encouraged. PROSPERO REGISTRATION NUMBER: CRD42021236979.

'Give Us The Tools!': development of knowledge transfer tools to support the involvement of patient partners in the development of clinical trial protocols with patient-reported outcomes (PROs), in accordance with SPIRIT-PRO Extension.

OBJECTIVES: (a) To adapt the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-patient-reported outcome (PRO) Extension guidance to a user-friendly format for patient partners and (b) to codesign a web-based tool to support the dissemination and uptake of the SPIRIT-PRO Extension by patient partners. DESIGN: A 1-day patient and public involvement session. PARTICIPANTS: Seven patient partners. METHODS: A patient partner produced an initial lay summary of the SPIRIT-PRO guideline and a glossary. We held a 1-day PPI session in November 2019 at the University of Birmingham. Five patient partners discussed the draft lay summary, agreed on the final wording, codesigned and agreed the final content for both tools. Two additional patient partners were involved in writing the manuscript. The study compiled with INVOLVE guidelines and was reported according to the Guidance for Reporting Involvement of Patients and the Public 2 checklist. RESULTS: Two user-friendly tools were developed to help patients and members of the public be involved in the codesign of clinical trials collecting PROs. The first tool presents a lay version of the SPIRIT-PRO Extension guidance. The second depicts the most relevant points, identified by the patient partners, of the guidance through an interactive flow diagram. CONCLUSIONS: These tools have the potential to support the involvement of patient partners in making informed contributions to the development of PRO aspects of clinical trial protocols, in accordance with the SPIRIT-PRO Extension guidelines. The involvement of patient partners ensured the tools focused on issues most relevant to them.

International perspectives on suboptimal patient-reported outcome trial design and reporting in cancer clinical trials: A qualitative study.

PURPOSE: Evidence suggests that the patient-reported outcome (PRO) content of cancer trial protocols is frequently inadequate and non-reporting of PRO findings is widespread. This qualitative study examined the factors influencing suboptimal PRO protocol content, implementation, and reporting, and use of PRO data during clinical interactions. METHODS: Semi-structured interviews were conducted with four stakeholder groups: (1) trialists and chief investigators; (2) people with lived experience of cancer; (3) international experts in PRO cancer trial design; (4) journal editors, funding panelists, and regulatory agencies. Data were analyzed using directed thematic analysis with an iterative coding frame. RESULTS: Forty-four interviews were undertaken. Several factors were identified that could influenced effective integration of PROs into trials and subsequent findings. Participants described (1) late inclusion of PROs in trial design; (2) PROs being considered a lower priority outcome compared to survival; (3) trialists' reluctance to collect or report PROs due to participant burden, missing data, and perceived reticence of journals to publish; (4) lack of staff training. Strategies to address these included training research personnel and improved communication with site staff and patients regarding the value of PROs. Examples of good practice were identified. CONCLUSION: Misconceptions relating to PRO methodology and its use may undermine their planning, collection, and reporting. There is a role for funding, regulatory, methodological, and journalistic institutions to address perceptions around the value of PROs, their position within the trial outcomes hierarchy, that PRO training and guidance is available, signposted, and readily accessible, with accompanying measures to ensure compliance with international best practice guidelines.

SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials.

Patient-reported outcomes (PROs) are used in clinical trials to provide valuable evidence on the impact of disease and treatment on patients' symptoms, function and quality of life. High-quality PRO data from trials can inform shared decision-making, regulatory and economic analyses and health policy. Recent evidence suggests the PRO content of past trial protocols was often incomplete or unclear, leading to research waste. To address this issue, international, consensus-based, PRO-specific guidelines were developed: the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT)-PRO Extension. The SPIRIT-PRO Extension is a 16-item checklist which aims to improve the content and quality of aspects of clinical trial protocols relating to PRO data collection to minimise research waste, and ultimately better inform patient-centred care. This SPIRIT-PRO explanation and elaboration (E&E) paper provides information to promote understanding and facilitate uptake of the recommended checklist items, including a comprehensive protocol template. For each SPIRIT-PRO item, we provide a detailed description, one or more examples from existing trial protocols and supporting empirical evidence of the item's importance. We recommend this paper and protocol template be used alongside the SPIRIT 2013 and SPIRIT-PRO Extension paper to optimise the transparent development and review of trial protocols with PROs.

A Factorial Survey Investigating the Effect of Disclosing Parental Intellectual Disability on Risk Assessments by Children's Social Workers in Child Safeguarding Scenarios.

Literature suggests that, as parents, people with intellectual disabilities experience disproportionately high rates of child removal compared to other groups. A factorial survey of 191 children's social workers investigated the effect of disclosing parental intellectual disability (ID) upon risk assessments in a range of hypothetical child safeguarding scenarios. The case scenarios depicted a range of child safeguarding situations and parents' ID status was randomly included as an additional item of information. The data were fitted into a generalised ordinal logistic regression model. Findings indicate that when presented with scenarios considered to be less risky, the parental ID disclosure contributed significantly to a higher risk assessment score. However, when presented with scenarios that were considered more risky, the additional parental ID disclosure did not significantly contribute to a higher score. These findings indicate that the risk associated with parental ID is not fixed but relative to the situation in which it is encountered. The research concludes that in cases of low risk, the effect of parental ID is identified as a support need, whereas the lesser contribution of the disclosure to assessments of higher risk cases may indicate that parental ID is overlooked.

Development of a core outcome set for use in community-based bipolar trials-A qualitative study and modified Delphi.

BACKGROUND: A core outcome set (COS) is a standardised collection of outcomes to be collected and reported in all trials within a research area. A COS can reduce reporting bias and facilitate evidence synthesis. This is currently unavailable for use in community-based bipolar trials. This research aimed to develop such a COS, with input from a full range of stakeholders. METHODS: A co-production approach was used throughout. A longlist of outcomes was derived from focus groups with people with a bipolar diagnosis and carers, interviews with healthcare professionals and a rapid review of outcomes listed in bipolar trials on the Cochrane database. An expert panel with personal and/or professional experience of bipolar participated in a modified Delphi process and the COS was finalised at a consensus meeting. RESULTS: Fifty participants rated the importance of each outcome. Sixty-six outcomes were included in Round 1 of the questionnaire; 13 outcomes were added by Round 1 participants and were rated in Round 2. Seventy-six percent of participants (n = 38) returned to Round 2 and 60 outcomes, including 4 outcomes added by participants in Round 1, received a rating of 7-9 by >70% and 1-3 by <25% of the sample. Fourteen participants finalised a COS containing 11 outcomes at the consensus meeting: personal recovery; connectedness; clinical recovery of bipolar symptoms; mental health and wellbeing; physical health; self-monitoring and management; medication effects; quality of life; service outcomes; experience of care; and use of coercion. CONCLUSIONS: This COS is recommended for use in community-based bipolar trials to ensure stakeholder-relevant outcomes, facilitate data synthesis, and transparent reporting. The COS includes guidance notes for each outcome to allow the identification of suitable measurement instruments. Further validation is recommended for use with a wide range of communities and to achieve standardised measurement.