Join KHI

Workgroup

  • Khurana, Mona – Division of Pediatrics and Maternal Health, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland
  •  Egger, Gunter F.– Paediatric Medicines Office, Scientific Evidence Generation Department, Human Division, European Medicines Agency (EMA), The Netherlands
  •  Yao, Lynne – Division of Pediatrics and Maternal Health, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland
  • Thompson, Aliza -Division of Cardiology and Nephrology, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland
  •  Pallidis, Chrissi – Paediatric Medicines Office, Scientific Evidence Generation Department, Human Division, European Medicines Agency (EMA), The Netherlands
  • Goldstein, Stuart L.– Cincinnati Children’s Medical Center, Cincinnati, Ohio
  • Laskin, Benjamin L.– The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
  • Tuchman, Shamir –  Division of Pediatrics and Maternal Health, Office of New Drugs, Center for Drug Evaluation and Research, Food and Drug
  • Malley, Meaghan A.– Kidney Health Initiative, American Society of Nephrology, Washington, DC 
  • Uhlig, Katrin – Division of Nephrology, Tufts Medical Center, Boston, Massachusetts and Agios Pharmaceuticals, Cambridge, Massachusetts
     

Drug Development in Children with Kidney Diseases

Projects
Portfolio:
Drugs
Barrier Type:
Clinical Trial Endpoints

Drug and device developers face major obstacles in conducting trials to advance diagnosis and treatment of children with kidney diseases, including 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.

Publications & Resources


Overcoming Barriers to Drug Development in Children with CKD : Clinical Journal of the American Society of Nephrology

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.

View More Projects

Xenotransplantation: Patient FAQ

  • Biologics, Patient Preferences

Identifying Clinical Research Priorities in Pediatric Nephrology

  • Clinical Trial Infrastructure, Devices, Drugs

Clinical Trials Innovation in Cardiac Surgery–Associated AKI

  • Clinical Trial Endpoints, Drugs

Developing a Roadmap to Accelerate the Expansion of AKI Biomarkers for Drug Development

  • Drugs, Education and Resources

Surrogate Endpoints in FSGS

  • Clinical Trial Endpoints, Drugs

Pediatric Clinical Trials: Making Them Family-Centric

  • Clinical Trial Infrastructure, Drugs
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