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. Currently, PKDOC hosts monthly teleconferences to explore alternative endpoints, innovative trial designs, and other topics of general interest to the participants. Attendees include pharmaceutical companies, academic organizations, foundations, patient advocacy groups, and regulatory agencies from around the world. Anyone who is interested may participate.
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. PKD is marked by a long period of stable kidney function (as measured by the currently accepted endpoints in drug development) during which the kidneys expand enormously due to cyst growth. Traditional endpoints of renal function (serum creatinine levels) 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 before patients have incurred serious, irreversible damage and when they may be more likely to respond to new therapies.
The initial goals of the PKD Consortium were 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. These initial goals have been achieved.
Using the data collected, scientists were able to develop a disease progression model that evaluated 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 were 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.,
