KHI Workgroup Opportunity
Drug Development in Children with Kidney Diseases
Overview
Drug and device developers face major obstacles in conducting trials to advance diagnosis and treatment of children with kidney diseases, also known as Chronic Kidney Disease (CKD). The rarity of pediatric patients with kidney diseases and their broad dispersion across the globe contributes to these obstacles. Drug development in children with kidney diseases must consider issues unique to this vulnerable population of limited size. Legislation by the United States and the European Union mandates plans for pediatric development as part of an overall product development strategy. This highlights the need to prioritize those programs that may be deemed most necessary and impactful and to optimize designs to facilitate successful completion. This project aimed to bring together relevant stakeholders for patients, healthcare providers, researchers, professional organizations, industry partners, and regulators, to develop recommendations to foster drug development for children with kidney diseases.
Deliverables
Kidney Pediatric Accelerator Trial Clearing House (Kidney-PATCH) Pilot Program
The Kidney-PATCH Pilot Program addressed these issues and facilitated broad coordination of clinical studies among networks. It enabled a feasibility assessment in terms of the available patient populations through data sharing and access to CKD pediatric registries; facilitated assessment of the capacity of various pediatric kidney clinical trial organizations; and assisted with identification of expertise that can provide consultation on study planning.