Overcoming Barriers to Drug Development in Children with CKD.

Like all sick children, children with chronic kidney disease (CKD) need access to safe and effective medicines that have been formulated and examined specifically for them. Despite legislation in the United States (US) and the European Union (EU) that either mandates or incentivizes programs for children, conducting trials to advance the treatment of children continues to prove to be a challenge for drug developers. This is also the case for drug development in children with CKD, where trials face challenges in recruitment and completion, and where there remains a substantial time lag between initial approval of a medicinal product for use in adults and completion of studies that result in the addition of pediatric-specific labeling for the same indication. The Kidney Health Initiative commissioned a workgroup of diverse stakeholders (https://khi.asn-online.org/projects/project.aspx?ID=61), including participants from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), to think carefully through the challenges in drug development for children with CKD, and how to overcome them. This paper provides an overview of the regulatory frameworks in the US and EU which govern pediatric drug development, the current landscape of drug development and approval for children with CKD, the challenges in conduct and execution of these drug trials, and the progress that has been made to facilitate drug development for children with CKD.