PKD Foundation Meets with the FDA
A key part of our Accelerating Treatments to Patients initiative is the PKD Outcomes Consortium (PKDOC). This is a significant collaboration between the Foundation, Critical Path Institute, members of the pharmaceutical industry, PKD researchers and clinicians, and the Food and Drug Administration (FDA). The goal of PKDOC is to facilitate clinical trial development for PKD therapies by establishing regulatory endpoints (or biomarkers) to measure early disease progression. Current traditional endpoints only show changes late in the course of the disease when kidneys are approximately five times normal size and normal tissue has been destroyed by hundreds of cysts and fibrotic tissue. At this stage, it may be too late for treatment.
Most recently, on Nov. 9, an in-person meeting with the FDA took place to review the PKDOC project data summary and analysis plan. The PKDOC steering committee thought that excellent progress was made at this meeting. The PKD Foundation was represented by Chief Scientific Officer Jill Panetta, Ph.D.; Scientific Advisory Committee member and PKDOC clinical lead Ron Perrone, M.D.; and Lorrie Rome, R&D Director.
Further discussions are planned in the near future to refine the analysis plan for submission in anticipation of another in-person meeting with the FDA early in 2013. This will be followed by the formal submission of the biomarker qualification package.