In 2020, RWE4Decisions, a multi-stakeholder initiative commissioned by the Belgian payer, published stakeholder actions to support the generation, analysis, and interpretation of real-world evidence (RWE) to inform the decision making of health technology assessment (HTA) bodies/payers for highly innovative medicines in the European Union (EU). Since 2020, changes in the decision-making environment and advancements in RWE have created an impetus to update stakeholder actions for the EU and Canada.

RWE4Decisions’ experts led focus groups with individual stakeholder groups (HTA bodies/payers, pharmaceutical industry, clinicians, patients, registry holders, and data analytical experts). Each focus group crafted new actions for their stakeholder, then the actions were discussed and revised in a multi-stakeholder meeting, a public webinar, and a public consultation. Themes across actions and meetings were identified.

Detailed new actions for each stakeholder group are presented. Key themes identified are the need to address interorganizational fragmentation regarding secondary data use and methodologies to build robust RWE. HTA bodies/payers need to develop a common vision about the potential use of RWE. The role of the whole clinical team as primary data collectors is critical. Opportunities for scientific advice across the life cycle of a medicine are essential, and the implementation of RWE guidance related to HTA is paramount. Progress requires specific, operational actions and a collective effort by a variety of stakeholders.

Carrying out these actions will facilitate the development of methodological best practices for generating RWE to inform HTA of highly innovative medicines and build trust between stakeholders in the use of RWE.

HOPE (National Institute for Health and Care Research Global Health Research Group on Homelessness and Mental Health in Africa) aims to develop and evaluate interventions that address the unmet needs of people who are homeless and have severe mental illness (SMI) living in three African countries in ways that are rights-based, contextually grounded, scalable and sustainable.

We will work in the capital city (Addis Ababa) in Ethiopia, a regional city (Tamale) in Ghana, and the capital city (Nairobi) and a rural county (Makueni) in Kenya to understand different approaches to intervention needed across varied settings.

We will be guided by the MRC/NIHR framework on complex interventions and implementation frameworks and emphasise co-production. Formative work will include synthesis of global evidence (systematic review, including grey literature, and a Delphi consensus exercise) on interventions and approaches to homelessness and SMI. We will map contexts; conduct focused ethnography to understand lived experiences of homelessness and SMI; carry out a cross-sectional survey of people who are homeless (n = 750 Ghana/Ethiopia; n = 350 Kenya) to estimate prevalence of SMI and identify prioritised needs; and conduct in-depth interviews and focus group discussions with key stakeholders to understand experiences, challenges and opportunities for intervention. This global and local evidence will feed into Theory of Change (ToC) workshops with stakeholders to establish agreement about valued primary outcomes, map pathways to impact and inform selection and implementation of interventions. Intervention packages to address prioritised needs will be co-produced, piloted and optimised for feasibility and acceptability using participatory action research. We will use rights-based approaches and focus on community-based care to ensure sustainability. Realist approaches will be employed to analyse how contextual variation affects mechanisms and outcomes to inform methods for a subsequent evaluation of larger scale implementation. Extensive capacity-strengthening activities will focus on equipping early career researchers and peer researchers. People with lived experience of SMI and policymakers are an integral part of the research team. Community engagement is supported by working closely with multisectoral Community Advisory Groups.

HOPE will develop evidence to support action to respond to the needs and preferences of people experiencing homelessness and SMI in diverse settings in Africa. We are creating a new partnership of researchers, policymakers, community members and people with lived experience of SMI and homelessness to enable African-led solutions. Key outputs will include contextually relevant practice and policy guidance that supports achievement of inclusive development.

Epidemiological data offer conflicting views of the natural course of binge-eating disorder (BED), with large retrospective studies suggesting a protracted course and small prospective studies suggesting a briefer duration. We thus examined changes in BED diagnostic status in a prospective, community-based study that was larger and more representative with respect to sex, age of onset, and body mass index (BMI) than prior multi-year prospective studies.

Probands and relatives with current DSM-IV BED (n = 156) from a family study of BED (‘baseline’) were selected for follow-up at 2.5 and 5 years. Probands were required to have BMI > 25 (women) or >27 (men). Diagnostic interviews and questionnaires were administered at all timepoints.

Of participants with follow-up data (n = 137), 78.1% were female, and 11.7% and 88.3% reported identifying as Black and White, respectively. At baseline, their mean age was 47.2 years, and mean BMI was 36.1. At 2.5 (and 5) years, 61.3% (45.7%), 23.4% (32.6%), and 15.3% (21.7%) of assessed participants exhibited full, sub-threshold, and no BED, respectively. No participants displayed anorexia or bulimia nervosa at follow-up timepoints. Median time to remission (i.e. no BED) exceeded 60 months, and median time to relapse (i.e. sub-threshold or full BED) after remission was 30 months. Two classes of machine learning methods did not consistently outperform random guessing at predicting time to remission from baseline demographic and clinical variables.

Among community-based adults with higher BMI, BED improves with time, but full remission often takes many years, and relapse is common.

Spinal muscular atrophy (SMA) is a leading genetic cause of infant death and represents a significant burden of care. An improved understanding of the epidemiology of SMA in Canada may help inform strategies to improve the standard of care for individuals living with SMA.

We employed a multisource approach to estimate the minimal incidence and prevalence of 5q SMA and to gain greater insight into recent clinical practices and treatment trends for the Canadian SMA population. Data sources included the Canadian Paediatric Surveillance Program (CPSP), Canadian Neuromuscular Disease Registry (CNDR), and molecular genetics laboratories in Canada.

The estimated annual minimum incidence of 5q SMA was 4.38, 3.44, and 7.99 cases per 100,000 live births in 2020 and 2021, based on CPSP, CNDR, and molecular genetics laboratories data, respectively, representing approximately 1 in 21,472 births (range 12,516–29,070) in Canada. SMA prevalence was estimated to be 0.85 per 100,000 persons aged 0–79 years. Delay in diagnosis exists across all SMA subtypes. Most common presenting symptoms were delayed milestones, hypotonia, and muscle weakness. Nusinersen was the most common disease-modifying treatment received. Most patients utilized multidisciplinary clinics for management of SMA.

This study provides data on the annual minimum incidence of pediatric 5q SMA in Canada. Recent therapeutic advances and newborn screening have the potential to drastically alter the natural history of SMA. Findings underline the importance of ongoing surveillance of the epidemiology and long-term health outcomes of SMA in the Canadian population.

To evaluate the impact of a menu box delivery service tailored to the long-day care (LDC) setting on improving menu compliance with recommendations, children’s diet quality and dietary intake while in care.

A cluster randomised controlled trial in LDC centres randomly assigned to an intervention (menu box delivery) or comparison (menu planning training) group. The primary outcome was child food provision and dietary intake. Secondary outcomes include menu compliance and process evaluation, including acceptability, fidelity and menu cost (per child, per day).

South Australian LDC centres.

Eight LDC centres (n 224 children) provided data.

No differences were observed in serves/d between intervention and comparison centres, for provision (intervention, 0·9 inter-quartile range (IQR) 0·7–1·2; comparison, 0·8 IQR 0·5–1·3) or consumption (intervention, 0·5 IQR 0·2–0·8; comparison, 0·5 IQR 0·3–0·9) of vegetables. Child food provision and dietary intake were similar across both groups for all food groups (P < 0·05). At follow-up, all intervention centres met menu planning guidelines for vegetables, whereas only one comparison centre met guidelines. Intervention centre directors found the menu box delivery more acceptable than cooks. Cost of the intervention was AUD$2·34 greater than comparison centres (intervention, AUD$4·62 (95 % CI ($4·58, $4·67)); comparison, AUD$2·28 (95 % CI ($2·27, $2·30)) per child, per day).

Menu compliance can be improved via a menu delivery service, delivering equivalent impacts on child food provision and dietary intake compared with an online training programme. Further exploration of cooks acceptability and cost is essential before scaling up to implementation.

The prevalence and patterns of autism spectrum disorder (ASD) symptoms/traits and the associations of ASD with psychiatric and substance use disorders has not been documented in non-clinical students in Sub-Saharan Africa, and Kenya in particular.

To document the risk level of ASD and its traits in a Kenyan student population (high school, college and university) using the Autism-Spectrum Quotient (AQ); and to determine the associations between ASD and other psychiatric and substance use disorders.

This was a cross-sectional study among students (n = 9626). We used instruments with sufficient psychometric properties and good discriminative validity to collect data. A cut-off score of ≥32 on the AQ was used to identify those at high risk of ASD. We conducted the following statistical tests: (a) basic descriptive statistics; (b) chi-squared tests and Fisher's exact tests to analyse associations between categorical variables and ASD; (c) independent t-tests to examine two-group comparisons with ASD; (d) one-way analysis of variance to make comparisons between categorical variables with three or more groups and ASD; (e) statistically significant (P < 0.05) variables fitted into an ordinal logistic regression model to identify determinants of ASD; (f) Pearson's correlation and reliability analysis.

Of the total sample, 54 (0.56%) were at high risk of ASD. Sociodemographic differences were found in the mean scores for the various traits, and statistically significant (P < 0.05) associations we found between ASD and various psychiatric and substance use disorders.

Risk of ASD, gender characteristics and associations with psychiatric and substance use disorders are similar in this Kenyan sample to those found in Western settings in non-clinical populations.