In its efforts to establish Total Kidney Volume (TKV) as a prognostic biomarker for use in clinical trial enrollment, the Polycystic Kidney Disease Outcomes Consortium (PKDOC) reached two very significant milestones over the past year. On April 30th, 2013, two and a half years after the Consortium’s initiation, and a year of data standards development, the PKDOC submitted qualification briefing documents to both the US Food and Drug Administration (FDA), and the European Medicines Agency (EMA). On June 28th 2013, PKDOC representatives from academia, industry, and C-Path met with the FDA to review the qualification briefing package, followed by a teleconference on July 19th. In a similar process, PKDOC members travelled to London for a July 9th 2013 meeting with representatives of the EMA.
After several subsequent teleconferences, the PKDOC modified the research plan, ran additional analyses, and updated the Briefing Package. The final version was submitted concurrently to FDA and EMA on March 20th, 2014. These two regulatory submissions represent key milestones in the regulatory science efforts to collect, standardize, aggregate, and analyze decades of legacy PKD data. As a result of analyzing and modeling these data, PKDOC intends to qualify TKV as a more sensitive predictor of disease progression in the early stages of PKD when patients are more likely to respond to treatment.
The PKDOC is a successful collaboration between C-Path, the PKD Foundation, CDISC, and four leading academic medical centers (Tufts University, University of Colorado – Denver, Emory University, and Mayo Clinic). The Consortium is funded through a grant from the PKD Foundation and philanthropic donations. Additionally, representatives from the pharmaceutical industry and the U.S. Food and Drug Administration (FDA) participate in this ground-breaking collaboration