Pediatric Obesity Pharmacotherapy: State of the Science, Research Gaps, and Opportunities

Pediatric obesity is a major public health problem, affecting nearly 20% of children and adolescents living in the United States. In 2023, the American Academy of Pediatrics released its first clinical practice guideline for the evaluation and management of child and adolescent obesity and recommended integrating health behavior and lifestyle interventions with pharmacological treatment when medically indicated. However, there is a limited evidence base to guide antiobesity medication treatment decisions in clinical practice and limited data on long-term safety during this critical period of growth and development in youth. Thus, in November of 2023, the National Institute of Diabetes and Digestive and Kidney Diseases sponsored a workshop to identify knowledge gaps and opportunities for research on the use of pharmacotherapy for obesity in children and adolescents. Leading scientific and clinical experts in obesity pathophysiology and treatment, pharmacotherapy, clinical trial design, and health equity and disparities, among others, identified gaps in clinical trial design, guidance for clinical use of medications in children and adolescents, additional treatment outcomes beyond body fat or weight, and improvement in care delivery. Adolescent patients and caregivers with lived experience of obesity and weight management were also invited to participate in a panel discussion, providing personal perspectives on living with obesity, clinical care considerations, and research needs. This article summarizes the workshop proceedings on the state of the science and identifies gaps and opportunities for future research to inform optimal and equitable medical management of children and adolescents with obesity.