KHI Current Project

Drug Development in Children with Kidney Diseases


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 aims 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.


A key deliverable for this signature project is the Kidney Pediatric Accelerator Trial Clearing House (Kidney-PATCH) to optimize planning for pediatric kidney disease clinical trials.


A workgroup has been assembled to draft a manuscript, over a 12 month timeframe, which is projected to take inventory of the current challenges and propose consensus based recommendations for improvements, including but not limited to:

  1. Recognize the legal remit and regulatory framework for pediatric study plans in US and Europe. Outline the current avenues of collaboration and communication between the regulatory agencies in US and Europe regarding pediatric plans
  2. Explore avenues for harmonization of study designs (including endpoints) and timelines for pediatric plans across US and European regulatory agencies.
  3. Develop a mechanism to prioritize drugs and drug classes for trials in children with kidney diseases through multi-stakeholder guidance and input, considering the following prioritization factors:
    • Unmet patient needs (e.g., new drug class, new mechanism of action)
    • Finite research resources in terms of number of patients affected and sites, in context of numerous pediatric commitments
    • Regulatory landscape for studies in children with kidney diseases
    • Timelines and priority metrics
  4. Optimize planning of pediatric drug trials by
    • Enabling feasibility assessment in terms of the available patient populations through data sharing and access to kidney diseases pediatric registries
    • Consultation on design and protocols by a panel of pediatric nephrologists, trialists, patient representatives early in the design process
    • Enhance the likelihood of successful trial execution through assessment of the capacity of various pediatric renal clinical trial organizations
  5. Identify key considerations for a balanced assessment of perceived benefits and risks (including toxicity), through a collaboration with experts (including toxicologists, clinical trialists from other pediatric disciplines that deal with toxicology risk vs. benefit, patients and caregivers). The prioritization process in Goal 3 should be informed by the patient/caregiver perspective in terms of this balance.


Completion goal of December 2020

Children with kidney diseases are a vulnerable population in need of therapeutic innovation. The United States and the European Union have permanent legislation in place mandating plans for pediatric development as part of an overall product development strategy.(1,2) EU and FDA requirement are not necessarily aligned. The overall number of children aged 0-19 on dialysis is about 2000 in the US with an equally small number estimated in the preceding stages of kidney diseases.(3)

Only a fraction of children with kidney diseases may be eligible for a particular study and there is active competition for different trials. There are marked clinical differences across age groups and the numbers dwindle with decreasing ages. This highlights the need to prioritize those programs that may be deemed most necessary and impactful by all stakeholders to facilitate successful study completion in this population of limited size. Furthermore, study design and execution must carefully consider specific technical and scientific issues related to diseases, treatments, age and ethics unique to children with kidney diseases.(4) Safety concerns are often heightened due to greater uncertainties stemming from less directly applicable information on pharmacodynamics and toxicity for younger age groups.

This project aims to foster drug development in children with kidney diseases by commissioning a workgroup to take inventory of current challenges, share insights and lessons learnt, and develop consensus based recommendations for overcoming barriers. The workgroup membership will take advantage of the diversity of constituent members within KHI and include representatives for patients, healthcare providers, researchers, professional organizations, industry partners, and regulators.

Role Name Organization
Co-ChairStuart Goldstein, MDCincinnati Children's Hospital Medical Center
Co-Chair, Board of Directors LiaisonKatrin Uhlig, MD, MSKeryx Biopharmaceuticals, Inc.
MemberAnn Danderand, MDOtsuka America Pharmaceuticals, Inc.
MemberPamela DuquetteJohns Hopkins University, KHI Patient and Family Partnership Counicl
MemberElizabeth Fox, MD, MSChildren's Hospital of Philadelphia
MemberDebbie Gipson, MDUniversity of Michigan
MemberEgger Gunter, DVMEuropean Medicines Agency
MemberJan Iles, MD, MPHAmgen, Inc.
MemberMona Khurana, MDDivision of Pediatric and Maternal Health, FDA
MemberTeresa Vu Lewis, PharmD, BCPSThe University of Oklahoma Health Sciences Center, College of Pharmacy
MemberAmy Mason, MD, MSBayer AG
North American Pediatric Renal Trials and Collaborative Studies LiaisonAlicia Neu, MDJohns Hopkins University
MemberRon Portman, MD, FAAP, FASNNovartis Pharmaceutical Corporation
MemberMarco Prunotto, PhDRoche Pharmaceuticals
MemberJesse Roach, MDCenter for Medicare and Medicaid Services
MemberMichelle Rheault, MDUniversity of Minnesota
MemberH. William Schnaper, MDNorthwestern University
MemberSandi See Tai, MDPfizer, Inc.
MemberAliza Thompson, MD, MSUS Food and Drug Administration
Staff LiaisonMeaghan AllainKidney Health Initiative


Selected References:
  1. Covered by the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA)
  2. Covered by Regulation (EC) No. 1901/2006 and Amending Regulation EC No 1902/2006
  3. 2014 USRDS ESRD Database (accessed 12/13/2015)
  4. Clinical research in pediatric nephrology: challenges, and strategies to address them. Foster BJ, Warady BA. J Nephrol. 2009 Nov-Dec;22 (6):685-93.