Objective
We propose to address unanswered questions in type 1 diabetes screening by collaborating with regions and health systems to apply a previously built platform that estimates the costs and benefits of screening for type 1 diabetes. We plan to include new data sources from multiple countries and screening studies. The platform we built combines data to inform public health officials on investments in screening strategies specifically. But each region- or system-specific adaptation has different guidelines and requirements to pay for screening. We envision applying this platform in a way that allows for flexibility for public health officials to make better decisions that apply to their own populations.
Background Rationale
Identifying people at risk of developing type 1 diabetes can help avoid life-threatening complications. Without early identification the consequences are severe, including considerable cost and health consequences for patients and families. For example, an alarming 60% of all children diagnosed with type 1 diabetes in Colorado end up in the emergency room or hospital when they are diagnosed. We know now from multiple large-scale screening programs that identifying children at risk of type 1 diabetes, along with providing education and follow-up visits, reduces complications at onset significantly. However, screening and monitoring for type 1 diabetes risk can be expensive and time consuming.
A major obstacle to uptake of broad population-level screening is identifying screening strategies that provide the best value for money. Our team built a model platform that can be tailored to a range of populations and healthcare systems around the world. During the first two years of this JDRF-funded project, we presented initial findings at conferences, compared our model to clinical evidence, and have the first versions of multiple peer-reviewed publications in development. While we presented these initial findings at conferences, we found multiple people around the world requested assistance to apply the platform to their own screening strategies and health systems. For example, Australia has a pilot screening program and PLEDGE is a screening program in an integrated health system in the United States. We also have collaborators developing their own analyses that want to compare their findings to our platform using the same underlying data. These types of comparisons can improve transparency and trust for predictions on the benefits and costs of various screening strategies. Finally, the pipeline of type 1 diabetes interventions is growing and there is a need to compare newly approved therapies in real-world settings (e.g., teplizumab) to pipeline therapies projecting approval in the coming years.
Description of Project
This proposal is a renewal and continuation of a prior grant project through JDRF, titled “Clinical and Economic Optimization Platform for Type 1 Diabetes Screening.” This project aims to further develop and apply a comprehensive web-based platform to inform global public health adoption and on the costs and benefits of identifying people at risk of type 1 diabetes. Identifying people at risk of developing type 1 diabetes can help avoid life-threatening complications. A comprehensive platform requires more advanced software tools than what is available in standard software packages. To address the needs of public health decision makers, clinicians, and patients and families, our team had to build a model platform that can be tailored to a range of populations and healthcare systems around the world.
During the first two years of this JDRF-funded project, we presented initial findings at conferences, compared our model to published evidence, and already have the first versions of multiple peer-reviewed publications in development. While we presented these initial findings at conferences, we found multiple people around the world requested assistance to apply the platform to their own screening strategies and health systems. For example, Australia has a pilot screening program and PLEDGE is a screening program in an integrated health system in the United States. We also have collaborators developing their own analyses that want to compare their findings to our platform using the same underlying data. These types of comparisons can improve transparency and trust for predictions on the benefits and costs of various screening strategies. Finally, now that we have the first approved therapy for delay and prevention of type 1 diabetes, we can provide comparisons to new potential therapies that are enrolling patients in trials now. These comparisons will give insights in to the value of teplizumab in addition to the value of new therapies that will be approved in the future.
This study will bring together a group of global experts to combine our knowledge, new data sources, and new intervention data on screening in one user-friendly web platform. This platform will be applied in a way that will inform the value of various type 1 diabetes screening strategies and interventions around the world in addition to improving the platform with new data and insights from global experts.
Anticipated Outcome
The major outcomes from this study include new applications that can inform type 1 diabetes screening around the world. Specific applications include region- and system-specific adaptations facilitating collaboration with local teams to produce evidence most informative to public health officials and health system leaders. Separate but related applications include comparisons of real-world use of teplizumab versus early evidence on other type 1 diabetes products approved in the future as well as versus screening and monitoring alone to inform governments and insurers on the value and affordability of these new therapies. An important outcome is transparency and trust of the model findings. We propose to compare our model to other collaborators working in type 1 diabetes. This will help improve the model and ideally increase the uptake of findings. Finally, we plan to compare teplizumab to screening and monitoring alone and separately to other pipeline therapies. Importantly, these results may be too early for any strong conclusions on the cost-effectiveness of these therapies, however, these early insights can provide independent assessments of the potential for value to patients and broader society.
Relevance to T1D
There is direct relevance of this study to people with type 1 diabetes and those seeking to help reduce complications from type 1 diabetes. A publicly available platform can help public health officials, patients and families, and others understand all of the costs, risks, and benefits from their respective health systems that go into screening for pre-symptomatic type 1 diabetes.