The Urgent Need for Breakthrough Therapies and a World Without Type 1 Diabetes

Abstract

Despite significant progress, type 1 diabetes (T1D) still results in premature death, significant complications, and a substantial daily burden for those affected. T1D remains a lifelong condition that demands constant vigilance and resilience and has a significant social and economic impact. Individuals with T1D must walk a tightrope to minimize disease-related complications that result from insufficient insulin while also avoiding adverse effects from too much insulin. Achieving this balance is challenging, as diet, activity, medications, physiology, the environment, stress, and many other aspects of daily living all affect glucose levels, often differently from day to day. Persistent challenges of T1D go beyond maintaining glycemic control and include managing long-term complications and preventing potentially life-threating adverse reactions from insulin therapy, and the emotional and cognitive burdens that often lead to diabetes distress and burnout. The T1D community-researchers, sponsors, clinicians, those living with T1D, and advocates-must look beyond managing symptoms of T1D and aim for better treatments and to bring cures. Emerging therapies need clear and efficient regulatory pathways, and new solutions are needed to address ongoing regulatory challenges. The perspectives of people with T1D must be front and center in research and regulatory decision-making. Through the collective efforts of the T1D community, the urgent needs of those with T1D can be met, and T1D can be made a thing of the past.

Keywords: Diabetes burnout; Diabetes distress; Drug development; Glycemic management; Hypoglycemia; Regulatory; T1D; Type 1 diabetes; Unmet needs.

Fig. 1

Decline of beta cell function across stages of T1D. Progression of type 1 diabetes (T1D) resulting from autoimmune attack on pancreatic beta cells. Stage 1 is the presence of two or more islet autoantibodies with normoglycemia; stage 2 is the presence of two or more autoantibodies with dysglycemia; stage 3 involves the presence of two or more autoantibodies, dysglycemia, and the onset of symptomatic disease. Exogenous insulin is required after the onset of stage 3 T1D when remaining beta cell function is no longer able to meet the body’s insulin requirements. Established T1D, often with complete insulin deficiency, is commonly called stage 4 T1D [9]

Fig. 2

Important clinical outcomes and effects of type 1 diabetes

Fig. 3

How type 1 diabetes impacts patients. Semi-structured qualitative interviews identified six domains of life commonly reported to be highly impacted by type 1 diabetes (T1D): well-being (e.g., stress), eating (e.g., carbohydrate counting), productivity (e.g., decreased concentration at work), sleeping (e.g., frequent overnight alarms), activity (e.g., feeling weak), and relationships (e.g., sense of stigma)