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Kidney Week

Abstract: FR-PO1150

Establishing an Organisational Entity to Support Development of Therapeutics in Children with CKD: A KHI Initiative

Session Information

  • Pediatric Nephrology - I
    October 26, 2018 | Location: Exhibit Hall, San Diego Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Pediatric Nephrology

  • 1600 Pediatric Nephrology

Authors

  • Rheault, Michelle N., University of Minnesota, Minneapolis, Minnesota, United States
  • Schnaper, H. William, Feinberg School of Medicine, Chicago, Illinois, United States
  • Duquette, Pam, Nephcure Kidney International, Baltimore, Maryland, United States
  • Gipson, Debbie S., University of Michigan Mott Children's Hospital, Ann Arbor, Michigan, United States
  • Lewis, Teresa, The University of Oklahoma Health Sciences, Oklahoma City, Oklahoma, United States
  • Neu, Alicia, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States
  • Schaefer, Franz S., University of Heidelberg, Heidelberg, BW, Germany
  • West, Melissa, American Society of Nephrology, Washington, District of Columbia, United States
  • Vande walle, Johan, UZGent, Gent, Belgium
  • Goldstein, Stuart, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
  • Uhlig, Katrin, Keryx Biopharmaceuticals, Brookline, Massachusetts, United States
Background

A Kidney Health Initiative (KHI) workgroup on ‘Overcoming Barriers to Drug Development in Children with CKD’ identified the need for more concerted and rational planning and conduct of clinical trials in children with CKD to address EMA and FDA regulatory requirement.

Methods

An Implementation Committee was charged to establish an organizational entity that will help optimize planning of therapeutic trials in children with CKD. Its charter addresses aims, guiding principles, composition of a standing Leadership Committee, committee operations, and funding.

Results

The aims of the new entity are to a) Enable feasibility assessment of available patient populations through data sharing and access to CKD pediatric registries b) Facilitate assessment of the capacity of various pediatric kidney clinical trial organizations c) Assist with identification of expertise that can provide consultation on study planning. To manage potential conflicts of interest, this entity needs to adhere to guiding principles regarding transparency, information sharing, international participation and collaboration, and financial independence, while respecting the rights and intellectual property of principal investigators, sponsors, and trial networks, and ensuring the confidentiality of proprietary information and protected patient information. The Leadership Committee will be comprised of representatives from pediatric collaborative trial networks, patient/family support groups and subject matter experts without industry sponsors. Operationally, industry sponsors will be able to submit queries and requests that will be reviewed, triaged, and addressed by the Leadership Committee. The Implementation Committee will draft a finance model, sustainability plan, and identify a permanent administrative home with a commitment to transparency, equity, and economy. In the interim, KHI will continue to support this entity.

Conclusion

This new entity will seek collaboration with other stakeholders to better support therapeutic development in children with CKD.