Objective
The overarching aim of this proposal is to address key knowledge gaps on the benefits, harms, feasibility, acceptability and cost-effectiveness of population-wide type 1 diabetes (T1D) screening in children. This knowledge is essential to inform public health policy decisions on the implementation of a government-funded, national T1D screening program for all Australian children.
Objectives:
1. Effectiveness: To quantify and compare the early benefits and harms of screening for T1D to usual care (no screening) through a randomised controlled study (first phase only in this proposal).
2. Implementation: to evaluate stakeholder perspectives (children, parents, health professional and schools) on the feasibility and acceptability of screening, including parental anxiety and perceived consequences of screening.
3. Epidemiology: To assess the Australian age and ethnicity-specific prevalence of early-stageT1D autoimmunity in children to identify the optimal ages and intervals for T1D antibody screening
4. Community Needs: to understand broad and diverse community preferences and experiences with T1D screening to inform the optimal screening model, implementation strategies and community messaging.
Background Rationale
T1D is an incurable autoimmune condition affecting over 127,000 Australians (8 new cases/day). Children are often diagnosed late, with at least 1 in 3 Australian children (up to 80% in regional areas) requiring emergency medical care for life-threatening diabetic ketoacidosis (DKA). This is traumatic for families and has lifelong implications for cognitive impairment, future DKA risk and long-term glycaemic control, increasing the risk of severe complications in early adulthood.
Two newly recognised ‘silent’ stages of T1D (Stage 1 and 2) can be readily identified through simple screening. This provides the opportunity for early diagnosis, which is essential for preventing diabetic ketoacidosis (DKA), reducing trauma at diagnosis and improving long-term health. Indeed, screening has been consistently linked with markedly reduced DKA risk (<5%).
Australia is well positioned to implement screening programs and there is clear interest and growing support for a national population-wide screening program for T1D. The 2024 Parliamentary Inquiry into Diabetes recommended the Australian Government explore a national screening program, featuring our Type 1 Diabetes National Screening Pilot8. Our Breakthrough T1D funded-research program, registered over 6,700 children for T1D screening in the world’s only head-to-head comparison of major screening models. Our study revealed that screening was feasible, acceptable and could be cost-effective as a public health initiative in Australia.
For a population screening program to be formally considered in Australia, an application must be submitted to the Medical Services Advisory Committee (MSAC) – an independent body appointed by the Federal Health Minister to advise on public health funding. This includes a health technology assessment (HTA) of the available evidence – typically high level evidence provided by randomised controlled trials (RCTs) – on the safety, clinical efficacy and cost-effectiveness. Furthermore, applications must include evidence on how screening should be implemented, particularly in the Australian healthcare system and the diverse needs of our rich multicultural population. A draft application, prepared as part of our previous Australian Type 1 Diabetes National Screening Pilot research program, revealed there were no RCTs on T1D screening, and limited Australian-specific epidemiological and implementation data on early-stage T1D. We therefore propose a clinical research program addressing key evidence gaps on the benefits, harms, feasibility, acceptability and cost-effectiveness of population-wide T1D screening in children. Collectively, this proposal is the next major advancement in the field and is poised to have substantial impact on policy and practice, both in Australia and globally.
Description of Project
Screening for presymptomatic type 1 diabetes (T1D) enables early diagnosis and the opportunity for prevention of life-threatening diabetic ketoacidosis (DKA) and trauma for families at diagnosis, and improves long-term health outcomes. As 90% of children with T1D have no family history, general population screening the only way to identify the vast majority of cases early. International programs show DKA rates of <5%, and screening also supports prevention efforts by identifying participants for clinical trials.
Our overarching vision is that general population screening for T1D will be implemented in Australia to decrease the burden of DKA and its multiple sequelae, creating the window of opportunity to prevent the condition altogether.
Australia is well placed to deliver such programs, with growing support including recommendations from the 2024 Parliamentary Inquiry into Diabetes and strong uptake in our Breakthrough T1D-funded National Screening Pilot, which registered over 6,700 children in the world’s first head-to-head comparison of screening models. Our findings showed screening is feasible, acceptable, and potentially cost-effective in Australia. However, a formal submission to the Australian Government’s Medical Services Advisory Committee (MSAC). Our draft application highlighted the lack of RCTs and limited Australian data on early-stage T1D and implementation approaches in our multicultural health system. We have therefore proposed a clinical research program addressing key evidence gaps on the benefits, harms, feasibility, acceptability and cost-effectiveness of population-wide T1D screening in children.
Specifically, we propose the first phase of the world’s first randomised controlled trial of T1D screening to deliver essential effectiveness, implementation and epidemiology evidence. This will be supported by comprehensive assessment of community needs and preferences for screening program design, including priority populations.
Anticipated Outcome
This proposal will generate high level evidence on the feasibility, acceptability, efficacy and cost-effectiveness of paediatric T1D screening, produced in collaboration with key stakeholders across the field. It builds on our existing successful community-focused and policy-informed research program. At the conclusion of the three year grant period, we will have delivered the necessary evidence to inform a national screening program and will have cemented a strong, integrated network from screening through to clinical care for early stage T1D in Australia.
This proposal is a novel and creative research program, providing the first randomised controlled trial of T1D screening, in a realistic and real-world setting yet with rapid and comprehensive assessment. We have strategically adopted a national, health systems-orientated approach that is sustainable and scalable to population-level. Furthermore, whilst the research is locally focused it contributes to the global effort towards general population screening.
Specifically, this program of research will deliver:
•the first phase of high level evidence quantifying and establishing causality between T1D screening, harms and immediate and long-term health benefits, which could be used in applications for government-funded population screening programs in Australia and around the world
• Strong community engagement with Australian families in the general community and priority populations, with deep understanding of their experiences, needs and preferences and tangible, culturally and linguistically-specific community-facing resources
• Evidence of the incidence, prevalence and natural history of early-stage T1D in the Australian paediatric population by age, ancestry and geography
• Scaled and refined T1D screening processes (e.g. screening assays, standardised reporting, confirmation testing etc.)
• Widespread T1D screening education and advocacy platform for families and health professionals
• The first significant dataset on early-stage T1D in the general population in Australia, which can be used for advocacy and further research, as well as readily harmonised with international registries and trials for comparison and to provide statistical power for subgroup analyses
• Accelerated early-stage T1D prevention therapies and research in Australia by generating a pool of children with early-stage T1D who may wish to receive disease modifying therapies, as clinically available and/or participate in clinical trials and/or clinical research studies (e.g. prevention clinical trials and qualitative studies to improve screening communication and clinical care)
Collectively, this is a pivotal next step in achieving our overarching vision for general population screening for T1D to be implemented into routine healthcare across Australia, with the principal aim of reducing the burden of DKA and its multiple sequelae and creating the window of opportunity to prevent/delay the need for complex and burdensome insulin therapy and associated complications.
Relevance to T1D
Type 1 diabetes (T1D) is a serious autoimmune condition that often begins in childhood and requires lifelong insulin therapy. Many children are diagnosed too late, with 1 in 3 needing emergency care due to life-threatening diabetic ketoacidosis (DKA). However, we now know that type 1 diabetes starts silently months or even years before symptoms appear. This creates a powerful opportunity to detect the condition early—before serious illness occurs.
Our research focuses on introducing general population screening to find children in the early stages of T1D. Early diagnosis can prevent emergency hospitalisations, reduce the trauma for families, and open the door to therapies that may delay, or ultimately prevent, the need for insulin altogether. With most children who develop T1D having no family history, screening everyone is the only way to catch it early.