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New C-Path Group Would Smooth FDA-Industry Interaction Over Dx

The Critical Path Institute plans to launch a new organization in the coming months to assess the performance of diagnostics before they are submitted to the US Food and Drug Administration for evaluation, according to a C-Path Institute official.
The new non-governmental, non-profit organization, will provide a test evaluation service to companies, but will not have any enforcement power. Ultimately, with the launch of this new group, the C-Path Institute is hoping to smooth the regulatory interaction between diagnostic companies and the agency.
Jeffrey Cossman, chief scientific officer of the C-Path Institute, told Pharmacogenomics Reporter this week that the new group, to be called United States Diagnostics Standards, would function as a kind of underwriter for diagnostics firms.
“Companies already submit their [diagnostics] to data test sites for evaluation prior to FDA submission,” Cossman said. “This would take the place of a data test site.”
The organization, which would be funded by industry user fees, would initially operate under the C-Path Institute, but would eventually become an independent entity. “The idea is to remain neutral and assist companies through the regulatory process. The idea is not to be a hurdle,” Cossman said.
The launch of USDS is one aspect of C-Path Institute’s larger goal of helping FDA standardize the evaluation of diagnostic tests.
“There may be a role here for establishing an entity that could provide a means for standardizing the testing of diagnostics before they are submitted to the FDA,” Cossman said last month during a meeting of the HHS Secretary’s Advisory Committee on Genetics, Health, and Society. “We see many bottlenecks along the way.”
At the meeting, Cossman noted that under the current regulatory system the FDA often “surprises” the industry with new submission requirements, or tells companies to redo certain studies. Similarly, the industry also frequently submits data that the agency does not understand or will not accept.
“We want to look at ways to improve the efficiency of the requirement for the highest standard of approval at FDA, which is the [pre-market approval], and how companies can improve their efficiency in getting to that very high bar,” Cossman said at the meeting.
In certain cases, a USDS assessment would allow companies to test the feasibility of their assays on standard samples. This may allow more companies to submit their products as 510(k) predicate devices.

“Then you have a de facto regulatory system.”

“Ten companies may have an assay against, say, troponin or d-dimers, but they are not testing them against the same standard analyte sample. So the data that is coming in to FDA may not necessarily be comparable,” Cossman said. “So if you are looking for a ‘me-too’ device or a 510(k), we can't always prove that the test is equivalent because it hasn't been tested on the same clinical material.”
Diagnostic companies will pay USDS to assess their tests under a user-fee system similar to the FDA’s regulatory process. Following a USDS review however, the company will own the data and is free to use it for FDA submissions, internal data, marketing, or in conversations with insurers.
“For lab-developed tests such as genetics, which may not end up being submitted to the FDA as an in vitro diagnostic, it could be used to evaluate those as well so that providers, consumers, payers, and investors would know whether or not the genetic test or other laboratory-developed test performed as claimed,” Cossman said. USDS in some cases will also look at clinical utility of diagnostic tests.
According to Cossman, diagnostics companies easily pay hundreds of thousands of dollars to test sites to assess the performance of their assays. Since determining the clinical utility of tests often involves conducting large prospective trials, the costs for such assessments can be significantly more.
Finally, USDS assessments can also clear up some of the confusion in the industry over FDA’s regulation of certain algorithm-based laboratory-developed tests, so called in vitro diagnostic multivariate index assays [see PGx Reporter 02-14-2006]. 
“It is hoped that USDS will be able to help clear up confusion with IVDMIA and lab-developed tests by assisting in the evaluation of performance,” Cossman said.
At the SACGHS meeting several committee members expressed concern that acceptance of USDS by the payor community as an independent source for diagnostic analysis would inadvertently create another de facto regulatory body that could influence reimbursement decisions.
“In some sense, if you tie this to data that will be used by payers and other reimbursers, the people that control the purse strings … may say, ‘We are not going to reimburse any tests that haven't gone through this process,’” Marc Williams, director of the Intermountain Healthcare Clinical Genetics Institute and SACGHS member, said at the meeting. “Then you have a de facto regulatory system.”
USDS is being developed as part of a public-private partnership between the C-Path Institute and Ventana Medical Systems, which received a $2.1 million grant from Science Foundation Arizona to standardize FDA’s evaluation process for cancer therapies and their companion diagnostics [see PGx Reporter 10-17-2007]. 
Initially, C-Path had planned to help take Ventana’s Benchmark XT assay through the FDA regulatory process as the first test case under this public-private collaboration. The test is also being used in a National Cancer Institute study to assess its validity in determining which lung cancer patients will benefit when treated with the EGFR inhibitors Tarceva, sold by Roche, and Alimta, made by Eli Lilly.
However, after talking with the industry, C-Path expanded the scope of the collaboration to not just focus on establishing regulatory standards for cancer diagnostics, but for all diagnostics. According to Cossman, the feasibility of this independent entity in standardizing the evaluation of laboratory-developed tests will be tested in a pilot study, which is currently being planned. While Ventana’s test is still in the running to be the pilot project, the C-Path Institute has spoken with 25 diagnostics firms to determine the best test case to take forward first.
Additionally, the C-Path Institute plans to work with National Institute of Standards and Technology to help develop standards for regulating LDTs. “We can take advantage of their technical expertise,” he said.
The NIST issued a call recently asking for input on a number of subjects it has deemed “areas of critical national need,” including personalized medicine. The responses NIST receives will be used to develop new competitions for funding under its Technology Innovation Program.

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