Clinical Trial Design Considerations for the Development of Disease-Modifying Therapies for Type 1 Diabetes.

Abstract

Article highlights: Several disease-modifying therapies have reduced the progressive loss of β-cell function in type 1 diabetes, with efficacy observed across a wide array of underlying therapeutic mechanisms of action. These successes highlight opportunities to accelerate the development of novel and repurposed type 1 diabetes treatments; however, only one such therapy has received regulatory approval for clinical use and few phase 3 clinical trials have been performed. For bringing emerging therapies to people living with type 1 diabetes as quickly and efficiently as possible, overall regulatory strategy and clinical trial design, especially inclusion criteria and end point selection, are essential, and planning must include their careful consideration. The considerations discussed herein represent a feasible and promising road map for developers of disease-modifying therapies and establish that type 1 diabetes remains a promising area for continued investment.