This article has been updated with additional information from Memorial Sloan Kettering Cancer Center.
NEW YORK (GenomeWeb) – The US Food and Drug Administration said today that it has authorized Memorial Sloan Kettering Cancer Center's MSK-IMPACT next-generation sequencing tumor profiling assay as an in vitro diagnostic test. In addition, the agency said that it has accredited the New York State Department of Health (NYSDOH) as an FDA third-party reviewer of IVDs, including similar tumor profiling tests.
FDA is also establishing a Class II regulatory pathway for the review of other NGS-based tumor profiling tests for use in cancer patients. This designation makes these tests eligible for the 510(k) clearance process, either by applying to the FDA directly or through an accredited third-party reviewer like NYSDOH.
MSK-IMPACT analyzes 468 genes in matched solid tumor/normal samples for mutations and looks for other molecular changes. Its results may inform a patient's cancer treatment, though they are not conclusive or prescriptive for labeled use of specific drugs. It is the first laboratory-developed tumor profiling test authorized by the FDA. According to the agency, the test can identify a higher number of cancer-specific mutations, or biomarkers, than any test it previously reviewed.
"This milestone authorization is a testament to MSK's expertise in the rapidly expanding field of genome-driven oncology and will set the precedent for future approvals of this type," said José Baselga, MSK's physician-in-chief, in a statement. To date, more than 20,000 patients with advanced cancer have had their tumors sequenced with MSK-IMPACT.
The agency said that its action on MSK-IMPACT "advances a policy framework that paves the way for the efficient review and availability of other NGS-based cancer profiling tools."
As part of this, FDA recently accredited the NY State Department of Health as a third-party reviewer of IVDs. Going forward, labs whose NGS-based tumor profiling tests have been approved by the NYDOH do not need to submit a separate 510(k) application to the FDA but may request that their NY state application, along with the state's review memorandum and recommendation, be forwarded to the FDA for possible 510(k) clearance.
FDA said that it might accredit other third parties to conduct such reviews and to make clearance recommendations to the agency.
"The goal of allowing NGS-based tumor profiling tests to undergo review by accredited third parties is to reduce the burden on test developers and streamline the regulatory assessment of these types of innovative products," said FDA Commissioner Scott Gottlieb in a statement. "As the field advances, we are modernizing the FDA's approach to the efficient authorization of laboratory tests from developers that voluntarily seek 501(k) clearance," he said.
"This is another example of where the FDA is working to find creative and flexible approaches to regulation that spurs development and efficient delivery of innovative technology," Gottlieb said. "We'll continue to look for opportunities to create regulatory efficiencies where possible to drive broader access to tools that improve American health, while maintaining the safety and efficacy standards that patients should expect from their FDA-reviewed products."
Jeff Shuren, director of the FDA's Center for Devices and Radiological Health, said that the FDA recognized the significance of a patient's tumor mutations for the planning of their care and their outcome, and that FDA worked closely with NYSDOH and Memorial Sloan Kettering to assess the MSK-IMPACT test. "This collaboration is an excellent example of how the FDA can partner with the medical and development communities to review innovative tests as quickly as possible," he said.
FDA reviewed the MSK-IMPACT test through the de novo premarket review pathway, which is designed for low- to moderate-risk devices that are novel and for which there is no legally marketed, or predicate, device.
An assessment of the test's analytical performance found that it is more than 99 percent accurate and detects mutations at a frequency of approximately 5 percent. Also, the test detected microsatellite instability with 92 percent concordance to traditional methods in 175 cases across multiple cancer types.
NYSDOH had previously approved the laboratory-developed assay for use as a clinical test in the state of New York. MSK submitted a de novo application for MSK-IMPACT to the FDA, which included and extended the information it had submitted to NY state in order to inform and expedite FDA authorization.