How important might the Critical Path Institute be to the growth of Arizona’s bioscience sector? Important enough that C-Path has earned the largest investment to date from Science Foundation Arizona (SFAz): $9 million.
“C-Path is the first initiative of its kind and represents a major strategic move in positioning Arizona to be highly competitive and of interest to the global pharmaceutical community,” said William Harris, president and CEO of SFAz. “With C-Path’s success, the Tucson area has the potential to increase substantially its biomedical capacity with innovation, spin-off companies and a growing base of knowledge workers.”
Three of the world’s largest pharmaceutical companies, in fact, now have research operations in the Tucson area. Roche Holding AG, which purchased Ventana Medical Systems Inc. earlier this year, is the newest player on the scene, joining sanofi-aventis and Merck, which holds a significant interest in Tucson-based High Throughput Genomics.
What has drawn some of the world’s largest pharmaceutical companies to C-Path’s door is its reputation as an honest broker able to balance the interests of drug companies and the government agencies that regulate them, at a time when the drug-development process seems all but broken. According to C-Path, today only 5 percent of drug candidates that enter human trials reach the market, despite enormous development costs and years of research. C-Path want to elevate the success rate to 95 percent.
“Without breakthroughs, we face a medical tsunami of health-care costs posing immense economic and social threats,” said Raymond L. Woosley, C-Path’s president and CEO. “In the United States alone, the annual cost of caring for patients with just one disease, Alzheimer’s, is $150 billion, a staggering figure that is projected to reach $1 trillion, or 8 percent of today’s total U.S. economy.”
By encouraging collaboration and sharing of information among companies on product safety testing and other bottlenecks in the drug-development process, and by brokering better communication with regulators, the three year-old C-Path aims to make pharmaceutical development cheaper, faster, and safer.
Last year, for example, the U.S. Food and Drug Administration (FDA) issued a new recommendation that doctors consider ordering a genetic test before prescribing the widely used blood thinner warfarin, given its potential to cause serious side effects in some patients. That recommendation likely would not have been issued without C-Path’s involvement.
“As warfarin is a generic drug, there was no corporate entity willing to do the confirmatory science and request a revised drug label,” Dr. Woosley said in Nature Reviews Drug Discovery. “To help address this, we asked academic scientists to help provide the expertise and data needed to enable the FDA to revise the label and include information on the value of genetic tests when selecting initial doses of warfarin. There is still a lot left to be done, but our role was–and continues to be–as an independent convener, identifying consensus and pulling together the additional data required.”
Another of C-Path’s breakthroughs has been establishing with the FDA and its counterpart in Europe, the European Medicines Agency a new way for drug and diagnostics companies to submit scientific data on safety testing methods. That new pathway allowed C-Path’s Predictive Safety Testing Consortium, representing 18 major pharmaceutical companies, to submit to the regulators seven new biomarkers of kidney damage for use in safety testing.
Currently, C-Path is working with a number of diagnostics companies, the FDA, and the National Cancer Institute to design a better way for approval of new biomarkers for use as diagnostic tests in regulatory and clinical decision-making.
“During drug development of targeted therapies, sponsors need to be able to co-develop a drug and diagnostic test to predict how a patient will respond to the drug, as well as to facilitate determination of the drug’s efficacy,” Dr. Woosley said in Nature Reviews Drug Discovery. “We are also working with the FDA and diagnostics companies to create a new entity that would independently certify the performance characteristics of diagnostic tests, as no such body currently exists.”
As a step toward establishing such an entity, C-Path’s chief scientist, Jeffrey Cossman, is working with the diagnostics companies to design a clinical trial that would verify whether the epidermal growth factor receptor is a biomarker that can predict patient response to a targeted lung-cancer therapy.
The importance of maintaining a position of neutrality complicates C-Path’s efforts to secure funding for the research it performs and coordinates. If it accepted funding from the companies with which it works, regulators might have reason to doubt the validity of its scientific findings.
“The ultimate stakeholder therefore is the public and we have been very fortunate to find such support in Arizona,” Dr. Woosley said in Nature Reviews Drug Discovery. “The governor and state legislators recognize the importance of developing a ‘knowledge economy,’ including investment in education and biotechnology. The state and leading foundations also see that for technology to reach the public, and for it to have economic value to the state, it has to be approved by the FDA, and C-Path is recognized as an important component of this.”
For more information:
“An Audience with: Ray Woosley,” Nature Reviews Drug Discovery, 11/2008 (7, 884)
“C-Path lands $9 million grant for research,” Inside Tucson Business, 11/14/2008
C-Path news release, 11/12/2008