The Polycystic Kidney Disease (PKD) Outcomes Consortium is a successful collaboration between Critical Path Institute (C-Path) and several leading academic medical centers, pharmaceutical companies, patient organizations, and international regulatory agencies. Its mission is to develop tools and processes, and promote research that will lead to the development and regulatory approval of new treatments for PKD, and improve the lives of all it affects.
The consortium has successfully qualified Total Kidney Volume as a prognostic biomarker with both the US Food and Drug Administration and the European Medicines Agency. It currently explores alternative endpoints, innovative trial designs, and other topics of general interest to the participants.
Autosomal Dominant PKD (ADPKD) is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide and for which there is currently no known cure or effective treatment. Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies.
The primary goals of the PKD Consortium are to develop CDISC data standards for PKD and to use clinical data from ADPKD patients collected over many years in patient registries and observational studies to support the FDA and EMA qualification of an imaging biomarker, Total Kidney Volume (TKV), for use in drug development trials.
Scientists will use the data collected to develop a disease progression model that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, hypertension, gross hematuria, kidney stones, urinary tract infections, development of end-stage renal disease, and mortality. These analyses will be used to support the regulatory qualification of TKV as an accepted measure for assessing the progression of ADPKD in clinical trials in which new therapies are tested.