Kidney diseases are, fortunately, relatively uncommon in the pediatric population. However, when they occur the physical, mental, and financial burdens on patients and their families are dramatic and life altering. There is large unmet need for safer and more effective treatments for the full range of pediatric kidneys diseases affecting infants, children, and adolescents. However, because of the rare nature of kidney disease in childhood and the ethical concerns raised by clinical research in pediatrics, it has proven difficult to develop novel therapies in this age group in a timely and efficient manner.

In July 2023, in response to the urgent need to assess the efficacy of SGLT2 inhibitors as renoprotective therapy in pediatric chronic kidney disease (pCKD), a workshop sponsored by NephCure and the Kidney Health Initiative (KHI) was convened in Washington DC. Attendees at the workshop included pediatric nephrologists, basic scientists, representatives of the Food and Drug Administration (FDA), industry sponsors, patients, and patient advocacy groups. The objective was to address the scientific, logistic, and regulatory issues that needed to be resolved in order for a randomized clinical trial of SGLT2 inhibitors in pCKD to be implemented. As a result of this workshop, Boehringer-Ingelheim agreed to design a trial to answer this pressing clinical problem. This workshop serves as a paradigm for a concerted effort needed by the pediatric nephrology community to define a clinical priority and organize the relevant stakeholders required to conduct the intended clinical trial. The critical first step in this process was identifying a consensus clinical research priority in pediatric nephrology. The National Institute of Diabetes and Digestive and Kidney Diseases would additionally benefit from understanding consensus clinical research priorities.