Abstract: INFO05-TH
Standard-of-Care and Novel Urine Biomarker Data: The PSTC/FNIH Biomarker Data Repository as a Foundation for Future European Medicines Agency Kidney Biomarker Qualification
Session Information
- Informational Posters - 1
October 22, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Acute Kidney Injury
- No subcategory defined
Authors
- Murray, Patrick T., University College Dublin, Dublin, Leinster, Ireland
- Weiss, Maqueline Alessandra, Tulane University, New Orleans, Louisiana, United States
- Monchi, Aymeric Cyrus Bruno, Sorbonne Universite, Paris, Île-de-France, France
- Roberts, Glenda V., Icahn School of Medicine at Mount Sinai, New York, New York, United States
- King, Nicholas M., Critical Path Institute, Tucson, Arizona, United States
- Peron, Katrina Jolene, Critical Path Institute, Tucson, Arizona, United States
- Poalillo, Andrew, Critical Path Institute, Tucson, Arizona, United States
- Sultana, Stefan, Critical Path Institute, Tucson, Arizona, United States
- Hoffmann, Steven C., Foundation for the National Institutes of Health, North Bethesda, Maryland, United States
- Friedman, Gary Steven, Critical Path Institute, Tucson, Arizona, United States
Description
In 2018, FDA qualified a panel of standard-of-care biomarkers (SBMs) and emerging or novel urine biomarkers (NBMs) for more sensitive detection of nephrotoxicant exposure and kidney injury in study cohorts. In May 2026, C-Path's Predictive Safety Testing Consortium (PSTC) & FNIH submitted a follow-on qualification package supporting use of paired SBM+NBM measurements for assessment of nephrotoxicant exposure in individual study participants.
The original qualification datasets primarily involved moderate-to-high nephrotoxic exposures. To support future FDA and European Medicines Agency (EMA) qualification efforts, the C-Path Biomarker Data Repository (BmDR) has (i) expanded to include completed pharma & non-pharma studies from North America, Europe, and Japan, and (ii) identified other academic consortia (KPMP, iC42 Tox Lab, SAFE-T/TransBioLine). More than 2,000 participants with paired SBM+NBM measurements are available for analysis of NBM performance across age groups, demographic subgroups, KDIGO eGFR strata, and diverse kidney injury populations, creating opportunities to evaluate additional biomarkers and novel contexts of use (COUs).
BmDR identified completed studies containing FDA-qualified SBMs+NBMs and emerging kidney biomarkers, including multi-omics, metabolomic, tissue, blood, and urine-based biomarkers. Using CDISC meta standards and robust statistical methods developed for the 2026 FDA qualification submission, BmDR & non-BmDR datasets can be evaluated for poolability and hierarchical stepdown analyses of smaller NBM subsets evaluating sensitivity and specificity (95% confidence intervals) across subsets of qualified NBMs and not-yet-qualified biomarkers.
The expanded BmDR provides a regulatory-grade framework for contextualizing the performance of emerging biomarkers with FDA-qualified SBM+NBM benchmarks. Pooling compatible datasets can improve estimation of biomarker sensitivity and specificity, address data gaps across patient populations, and support development of new biomarker COUs. This strategy leverages prior FDA qualification experience to accelerate future EMA (and FDA & PMDA) biomarker qualification plans to support development of precision medicine diagnostics and therapeutic decision-making tools for patients with kidney injury and kidney disease.
Funding
- Other U.S. Government Support (Food and Drug Administration)