Title: Intensive behavioural interventions for young children with autism: A systematic review and cost-effectiveness analysis
Funder: Department of Health and Social Care

Abstract: Design: Systematic review and individual participant data (IPD) meta-analysis of effectiveness, decision model of cost-effectiveness, budget impact analysis and value of information (VOI) analysis. Background: Autism spectrum disorder (ASD) is a lifelong neurodevelopmental disorder, characterised and diagnosed by early onset di?culties in social communication and restricted repetitive patterns of behaviour activities and interests (1). The high prevalence and serious long term outcomes, as well as the costs of providing high-quality health, educational and community services for this population (2, 3) and their families makes ASD a considerable health concern. The most widespread and advocated of such therapies are those based on applied behaviour analysis (ABA). Despite a number of previous systematic reviews in this area, it remains unclear whether these interventions are effective and cost-effective in the short and long-term. Aims of the research: The aims of the research are to evaluate the clinical and cost-effectiveness of intensive ABA-based interventions for young children with ASD based on current evidence; to establish the financial consequences of introducing the interventions in the UK considering the specific budget impact upon national health services, local authority and social care; and to estimate the value of undertaking additional research in this area. Design and methods: The question of whether intensive ABA-based interventions are effective for young children with ASD will be addressed by a systematic review and individual participant data meta-analysis (IPD-MA). The results of IPD-MA will inform a decision model to evaluate the cost-effectiveness of the interventions in the UK setting, as will a series of additional systematic reviews of cost-effectiveness, utilities and epidemiological studies as required by the model. Expert elicitation will also be used to address areas of uncertainty and populate the model. IPD-MA of all relevant controlled studies will evaluate clinical effectiveness and explore whether clinical effectiveness varies between particular types of children. Where we are unable to obtain IPD from study authors, we will carry out an aggregate data meta-analysis to compare with the IPD-MA results. A new decision-analytic model will be developed to assess the cost-effectiveness of intensive ABA-based interventions for young children with ASD. This model will be used to evaluate the cost per Quality Adjusted Life Year (QALY) of early intensive ABA-based interventions and quantify the main uncertainties facing decision makers. This analysis will include a VOI analysis to quantify the value of undertaking further research to resolve these uncertainties. A budget impact model will also be developed to estimate the financial consequences of introducing the intensive ABA-based interventions for young children in the UK. Dissemination: Findings of the systematic reviews, cost-effectiveness, VOI analysis and budget impact analysis will be reported in an HTA monograph. We will publish the results of clinical effectiveness and economic evaluation as open access articles in peer-review journals, and as a widely accessible summary report directed at patients and a more general audience. The outputs will be of value to clinicians, parent and cares’ of children with autism, guideline authors, policy makers and commissioners of services for children with autism.

Lay abstract: This study aims to find out whether or not intensive early interventions based on applied behaviour analysis (ABA) can help young children with autism, and whether it represents good value for money. Autism is a complex life-long condition that impacts on development in different areas including intellectual, communication, social, emotional, and adaptive skills for daily living. The proportion of children diagnosed with autism has increased considerably over the last two decades, and it now affects approximately 3 in 200 children born in the UK. People with autism often experience difficulties in life and can also experience poor mental health. Autism, while not a life threatening condition in itself, is also associated with premature death. The high prevalence, serious long-term outcomes, and the costs of providing high-quality health, educational and community services for the people with autism and their families makes it a considerable concern. There have been some studies completed with young children with autism showing that ABA-based treatments can help. These intense, comprehensive approaches when used early in the child’s development target a broad range of skills and behaviours. However, it remains unclear whether these interventions are effective in the short and long-term. Our proposed work will consist of two related studies: the first will look at examining how well ABA-based treatments work for young children with autism, and the second will look whether ABA-based treatments are likely to represent value for money. In the first study we will identify all known studies that have looked into ABA-based interventions for young children with autism. We will combine the results of these studies together to find out how effective ABA-based interventions are in improving important outcomes such as cognitive, adaptive or social abilities, communication/language skills, symptom severity, quality of life, educational attainment, or daily functioning and decision making capacity. As well as looking at the average effects found by studies, our work will analyse data from individual children, to help us explore whether effectiveness varies between particular groups of children. In the second study we will look at the costs and benefits of implementing ABA-based interventions for young children with autism. We will do this by developing an economic model to reflect short term, midterm and long term costs and benefits of ABA-based interventions. The work will be carried out from public sector point of view. This work will also look at the consequences of implementing ABA-based interventions on different government budgets such as the NHS or local authority, as well as the value of conducting further research in this area. We will organise three half day advisory group meetings which will include at least two people with autism and two parents/carers. These meetings will be aimed at helping the research team better understand autism’s impact on individuals and carers, and ensuring that our work is clear and transparent to all stakeholders. We will publish research findings in peer-review journals and as a widely accessible summary report directed at patients and a more general audience. The outputs will be of value to clinicians, parents and carers of children with autism, guideline authors, policy makers and commissioners of services for children with autism.

Research activity codes: 6.6 PSYCHOLOGICAL AND BEHAVIOURAL;8.2 HEALTH AND WELFARE ECONOMICS
Health categories: MENTAL HEALTH