Promoting Advancements in Pediatric and Congenital Cardiology Amid Clinical Trial Challenges

Due to improvements in the diagnosis and treatment of pediatric heart disease across the lifespan, an increasing number of these patients are surviving well into adulthood. However, compared to our adult cardiology colleagues, pediatric cardiologists do not have nearly as much evidence to guide the management of their patients. The heterogeneity of diagnoses and the relatively small number of individuals affected by each condition pose challenges in achieving adequate study power. The broad spectrum of potential morbidities and unique pathophysiology associated with the different conditions makes identifying clinically meaningful and achievable study endpoints particularly complex. In addition, recruitment and retention of study participants is difficult due to the fragile nature of patients as well as the regionalization of cardiac care, which results in long distances from study sites for many patients and families. In the past few decades, researchers in the field of pediatric and congenital cardiology have established several multicenter registries, programs, learning networks, and other collaborative efforts to help address these issues. While not a comprehensive review, this paper, which arose in part from the 2023 CardioVascular Clinical Trialists (CVCT) forum, will describe some of the most significant and impactful pediatric cardiology clinical research efforts to date and will emphasize areas that require ongoing focused attention.