Life with type 1 diabetes, includes myriad considerations beyond individual treatment regimens. In the Saturday, June 22, session Navigating the Early Phases of Pediatric Type 1 Diabetes, panelists discussed the patient perspective on navigating clinical trials as well as efforts to model the cost effectiveness of monitoring and prevention strategies.

The session can be viewed on-demand by registered meeting participants on the virtual meeting platform. If you haven’t registered for the 84th Scientific Sessions, register today to access the valuable meeting content through Aug. 26.

Amanda Twigg, MD, University of California, San Francisco, described her and her daughter’s journey with type 1 diabetes. Dr. Twigg was diagnosed with type 1 diabetes at the age of 20. When she had children, she decided to be proactive in finding out their risk for the disease. Screening revealed her daughter had several risk factors that made type 1 diabetes inevitable.

“This was one of the most crushing days of my life,” she said. “I had worked so hard to stay healthy during my pregnancies.”

The screening also strengthened Dr. Twigg’s resolve to be proactive on behalf of her daughter.

“As somebody who’s experienced diabetes and understands what it’s like to live day in and day out with a chronic disease, my instincts as a parent meant I really wanted to delay this as long as possible for my child,” she said.

She described their experience participating in clinical prevention trials, which often meant long hospital stays, days off of school, enduring a PICC line, and finding safe ways to celebrate holidays when they had to remain isolated.

Deciding to participate in clinical trials wasn’t easy, Dr. Twigg explained. They weighed several pros and cons, including potential side effects, efficacy, and the impact on their lives.

“We knew what the default course would look like of ending up on insulin. We didn’t want the treatment to be worse than the disease, especially because she wasn’t sick yet,” she said. “We were also worried about the future loss of a missed opportunity. Teplizumab may offer a delay in onset, but would there be something on the horizon that could offer a cure?”

Her daughter recently finished the PETITE-T1D trial, a prevention study of teplizumab in children. Despite the difficulties, Dr. Twigg remains optimistic.

“Each day for my kids without diabetes is a win,” she concluded.

R. Brett McQueen, PhD, University of Colorado Anschutz Medical Campus, described the economic-related factors involved in the detection and treatment of early-stage type 1 diabetes.

Currently, type 1 diabetes screening and monitoring is not cost effective when considering only the impact on reducing diabetes-related ketoacidosis (DKA) at onset, but including other factors, such as glycemic control, can increase cost effectiveness.

Dr. McQueen described a model his group is developing to correlate intermediate-term outcomes, such as changes in A1C to long-term impact, such as survival and quality of life. The model uses machine learning to analyze thousands of screening strategies to input factors such as genetic risk score, family history, and autoantibodies and solve for outputs such as cost and reduction in DKA.

“We’re trying to build a useful model that can help public health decision-makers understand the trade-offs,” he said. “It’s always subject to inputs and data sources. We will continue to update the model as more effectiveness evidence comes out and we see the real-world use of monitoring.”

He hopes that the model can provide insight on the optimum balance of screening and monitoring protocols and cost. They’ve found that various type 1 diabetes detection strategies can be cost effective if long-term benefits are demonstrated. In addition, volume-based discounts and bundles with other types of screening programs will likely provide additional cost savings.

Dr. McQueen is now working to build an online version of their model to inform implementation and budget impact scenarios around the world and enable health systems to tailor the model to the specific protocols they are able to implement.

Additional speakers in the session included Anette-Gabriele Siegler, MD, Helmholtz Center Munich and Technical University, who discussed pediatric type 1 diabetes screening programs, and Andrea Steck, MD, Barbara Davis Center for Diabetes, who discussed continuous glucose monitoring and early stage type 1 diabetes.