Advancing Pediatric Drug Development

How to accelerate development of drugs for pediatric patients with type 2 diabetes?

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Briefly describe your research question :

Current pharmacological treatment options for youth-onset type 2 diabetes (T2D) are inadequate and limited to insulin and metformin. Unmet need is augmented by the fact that metformin monotherapy fails in many pediatric patients during the second year of treatment. The development of pharmacological treatments for pediatric use has been challenged by several factors, including the limited numbers of available pediatric patients for participation in all planned/ongoing studies. In addition, several of the completed studies have failed to meet their primary endpoint, most probably because of inadequate power, poor compliance, use of concurrent antidiabetic therapies, and inadequate washout periods. Model-informed drug development (MIDD) could help to prioritize development of drugs by identifying those predicted to be more efficacious and, to optimize the design of the selected studies thereby increasing the probability of success. Through MIDD, historical data and prior knowledge in adult and pediatric populations can be integrated and provide a starting point for future strategy of development of drugs for youth-onset T2D. Hence, it is proposed that historical data from TODAY and ADOPT study as well as data from sponsor driven studies are used to develop PK-PD models that differentiate between drug and system specific parameters. Quantification of differences in disease progression and manifestation will aid to the prediction of the long-term efficacy based on short-term data and, to the prediction of placebo and/or add-on monotherapy. This proposal is the result of multi-stakeholder interactions as reported by Barrett et al, 2017 ( and by Barrett et al, 2020 (




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Idea No. 121