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Ventana’s Submission for Lung Cancer Test May be Model for Future Dx/Rx Approvals

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A public/private partnership between the Critical Path Institute and Ventana Medical Systems may be the first step in designing a business model to effectively develop drug/diagnostic combination products.
 
Last week, the C-Path Institute announced that it will use a $2.1 million Arizona state grant to help Ventana take a lung cancer diagnostic through the US Food and Drug Administration’s regulatory submissions process. The effort, part of a larger National Cancer Institute clinical trial to evaluate a variety of lung cancer assays, aims to standardize a regulatory pathway for companion diagnostics and therapies.
 
The FDA is slated to release a guidance document on Rx/Dx co-development by year end. With this two-year collaboration, the agency is attempting to develop performance standards it can use to guide the evaluation of future Rx/Dx co-submissions, according to a C-Path Institute official.
 
The FDA is hoping the project can be “a model that paves the way for future submissions for other types of targeted therapy diagnostics,” Jeff Cossman, the C-Path Institute’s chief scientific officer, told Pharmacogenomics Reporter this week.
 
“Currently, there is no proven development pathway for FDA approval of the necessary companion diagnostic tests and their associated targeted therapies,” Ventana said in a statement. “The goal of this collaboration is to establish the performance standards that would serve as the model for future FDA co-submissions of these companion diagnostic tests and their targeted drug therapies.”
 
FDA officials view Rx/Dx co-development as the Holy Grail of personalized medicine. Slow physician adoption of new genetic tests for older drugs like warfarin and certain cancer treatments illustrate the difficulty of introducing new technologies in established markets.
 
At a conference on personalized medicine at Harvard Medical School in Boston last week, Felix Frueh, head of the FDA’s Interdisciplinary Pharmacogenomics Research Group, noted that there is no business model encouraging the development of Rx/Dx products, which are critical to the advancement of personalized medicine.
 

“The idea is not to make an extra hurdle. The idea is to make it easier for [test developers] to get their tests validated, to have them validated in the same way that other companies’ tests are validated. So, when FDA sees the data, they see a familiar process.”

On the industry side, Rx/Dx co-development is hampered by misaligned development timelines for drugs and diagnostic products. While drug companies are increasingly using PGx strategies to learn more about their investigational products earlier in clinical development, pharma has often criticized device manufactures for not adequately establishing the clinical validity of their tests. This one aspect, pharma officials say, is a major barrier to advancing personalized medicine [see PGx Reporter 05-02-2007].
 
By bringing industry and government together with the C-Path Institute, an independent third party, this collaboration may be the first step toward addressing some of the barriers to Rx/Dx co-development.
 
In addition to helping Ventana take its test through FDA, the C-Path Institute is also in early discussions with the agency about developing a laboratory in Arizona that will help companies validate new diagnostics with standardized samples prior to submitting them to the agency for clearance.
 
The agency “wants to learn more [about] how these kinds of tests are validated and how the science is run on them,” Cossman said. “The FDA will work with [the C-Path Institute] because we’re neutral, with no financial incentive. We also have expertise in molecular diagnostics.”
 
“The ultimate goal of the project is to guide the choice of targeted therapy so that patients receive the most effective treatments," C-Path Institute CEO Raymond Woosley said in a statement.
 
C-Path is a publicly funded non-profit based in Tucson, Ariz., that aims to help the FDA implement its Critical Path Initiative. The $2.1 million grant for the Ventana project was awarded by Science Foundation Arizona, which operates with state, industry, and private backing, and funds public-private economic development projects around technology.
 
The Test
 
The diagnostic that Ventana will submit for FDA clearance with the guidance of the institute is the Benchmark XT, an automated tissue testing platform that assesses protein expression by immunohistochemistry and gene amplification by in situ hybridization.
 
According to Cossman, Ventana will use the platform to study EGFR in the C-Path project.
 
“In developing its tests, Ventana goes through a series of laboratory verification and validation steps. What we will do is partner with them and review that process, [and] see how they collect and report that information,” Cossman explained. Throughout this process, the C-Path Institute will meet with FDA’s Office of In Vitro Diagnostics on a regular basis to report on Ventana’s submissions progress and discuss regulatory requirements.
 
“The point is not to assist Ventana specifically but rather to help the process in general. It is hoped this information will help FDA in its mission to develop a broader regulatory pathway for diagnostics of targeted therapy,” Cossman said.
 
Cossman said the collaboration with Ventana is also part of a larger effort at the NCI investigating the validity of various gene-based lung cancer tests. In a 1,200-patient prospective trial using Tarceva and pemetrexed, the NCI will study the utility of diagnostics to predict response to the two drugs.
                                                         
The Critical Path Institute is charged with putting together a list of tests for this study. So far, the institute has spoken to about 20 test developers and is in the process of prioritizing which tests to recommend for the study, Cossman said.
 
Learning Collaboration
 
While the collaboration appears to be an effort to standardize Rx/Dx submission procedures, the FDA has no plans to formally release a guidance document or a ruling at the end of the process.
 
“The collaboration will clearly be of interest to FDA and the lessons learned will be considered as we continue to address the many complex regulatory, scientific, and administrative issues in this area,” Office of In Vitro Diagnostics Director Steven Gutman told Pharmacogenomics Reporter in an e-mail this week. “There is no effort to directly tie this work to any particular rule or guidance and the manner in which this information is used is less formal.”
 
As a component of the project funded by Science Foundation Arizona, the C-Path Institute has “informally” introduced the idea of developing a testing laboratory in Arizona that will analyze new diagnostic tests for sponsors against standardized samples prior to their submission for FDA clearance, Cossman said. The institute is hoping the agency will like the idea of such a service as “it would standardize the way diagnostic assays are validated,” he said.
 
Ultimately, taking advantage of this lab, which Cossman described as a “sort of underwriter’s facility for diagnostic laboratory tests,” might improve a test’s chances for FDA clearance. However, it would not necessarily be a mandatory aspect of the Rx/Dx regulatory submissions process.
 
“The idea is not to make an extra hurdle. The idea is to make it easier for [test developers] to get their tests validated, to have them validated in the same way that other companies’ tests are validated. So, when FDA sees the data, they see a familiar process,” Cossman said. In this way, the “FDA has the best chance of understanding [the submission data], which increases [the companies’] chances” that their tests will be cleared.
 
Eyeing Rx/Dx Development
 
For its part, Ventana recognizes the value of Rx/Dx products and appears eager to enter this development space.
 
In a statement announcing financial results for the period ending June 30, the company noted that “the companion diagnostics market is growing rapidly, as leading pharmaceutical companies focus on targeted therapies for breast, lung, colon, and prostate cancer.
 
“Matching therapy with diagnostics is highly attractive to major industry players as it can significantly reduce drug development time and cost, and facilitate regulatory approval,” Ventana CEO Christopher Gleeson said in a statement.
 
Ventana is currently partnering with nine pharmaceutical companies on 22 projects, Gleeson said, noting that the company “is uniquely positioned to capture a significant share of the companion diagnostics market.”
 
The company recently has been adamantly thwarting the acquisition overtures of Roche, which has twice extended a tender offer to acquire Ventana, noting that the company will help attract Rx/Dx partnerships [see PGx Reporter 09-26-2007, 06-27-2007].

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