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FDA unveils a streamlined path for the authorization of tumor profiling tests alongside its latest product action
Newly authorized test detects genetic cancer mutations in 468 unique genes
For Immediate Release
November 15, 2017
Summary
FDA unveils streamlined path for the authorization of tumor profiling tests alongside its latest product action
Release
The U.S. Food and Drug Administration today authorized Memorial Sloan Kettering Cancer Center’s (MSK) IMPACT (Integrated Mutation Profiling of Actionable Cancer Targets) tumor profiling test (assay), an in vitro diagnostic test that can identify a higher number of genetic mutations (biomarkers) that may be found in various cancers than any test previously reviewed by the agency. The IMPACT test uses next-generation sequencing (NGS) to rapidly identify the presence of mutations in 468 unique genes, as well as other molecular changes in the genomic makeup of a person’s tumor. Cancer profile tests are gaining wider acceptance. By identifying what genetic mutations are present in a particular tumor, the test results can provide patients and health care professionals with useful insight that may help inform how best to treat the cancer.
Today’s action advances a policy framework that paves the way for the efficient review and availability of other NGS-based cancer profiling tools. The FDA is also announcing the recent accreditation of the New York State Department of Health (NYSDOH) as an FDA third-party reviewer of in vitro diagnostics, including tests similar to the IMPACT test. Moving forward, laboratories whose NGS-based tumor profiling tests have been approved by NYSDOH do not need to submit a separate 510(k) application to the FDA. Instead, developers may choose to request that their NYSDOH application, as well as the state’s review memorandum and recommendation be forwarded to the FDA for possible 510(k) clearance. Other accredited, third-party FDA reviewers also may become eligible to conduct such reviews and 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. As this field advances, we are modernizing the FDA’s approach to the efficient authorization of laboratory tests from developers that voluntarily seek 510(k) clearance,” said FDA Commissioner Scott Gottlieb, M.D. “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. 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.”
According to the National Cancer Institute at the National Institutes of Health, approximately 38.5 percent of American men and women will be diagnosed with a form of cancer at some point during their lifetime. Unlike many cancer diagnostics that are designed to detect one cancer biomarker for use with a single drug, the IMPACT test works by comparing tumor tissue to a “normal” sample of tissue or cells from the same patient to detect genetic alterations that might help guide treatment options. While the test is intended to provide information on cancer biomarkers, its results are not conclusive for choosing a corresponding treatment.
“NGS technologies can examine hundreds, if not millions, of DNA variants at a time; and we are only at the beginning of realizing the true potential for these devices to assist patients and their health care providers in learning about the genetic underpinnings of their disease,” said Jeffrey Shuren, M.D., director of the FDA’s Center for Devices and Radiological Health. “Recognizing the significant effect information about an individual’s biomarkers can have on their care planning and outcomes, the FDA worked closely with NYSDOH and MSK to help ensure that the IMPACT test is accurate, reliable and clinically meaningful. 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.”
The IMPACT test was reviewed by the FDA through the de novo premarket review pathway, a regulatory pathway for some low- to moderate-risk devices that are novel and for which there is no legally marketed device (predicate device). Its ability to detect genetic mutations (analytical performance) was evaluated for precision, accuracy and limit of detection. Results indicated that the assay is highly accurate (greater than 99 percent) and capable of detecting a mutation at a frequency of approximately 5 percent (range of 2-5 percent). Additionally, detection of certain molecular changes (microsatellite instability) using the IMPACT test was concordant more than 92 percent of the time across multiple cancer types in 175 cases, when compared to traditional methods of detection.
Specific to the IMPACT test’s authorization, the NYSDOH previously conducted its own review and approved it for use on samples coming from patients in the state of New York. However, MSK had not previously submitted the test for the FDA’s review because it is a laboratory-developed test, for which the agency has generally not enforced premarket review and other applicable requirements. MSK submitted a de novo application for the IMPACT test to the FDA, including and extending the information submitted for NYSDOH’s prior review, to inform and expedite today’s FDA authorization.
Along with this authorization, the FDA is also establishing a Class II regulatory pathway for the review of other NGS-based tumor profiling tests for use in patients diagnosed with cancer. Class II designation allows these types of tests to be eligible to use the FDA’s 510(k) clearance process, either by submitting the application to the FDA directly or through an accredited third-party reviewer, like NYSDOH.
The FDA granted marketing authorization for the IMPACT tumor profiling assay to Memorial Sloan Kettering Cancer Center.
The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585347.htm?utm_campaign=11152017_PR_FDA%20unveils%20streamlined%20tumor%20profiling&utm_medium=email&utm_source=Eloqua
They didn't have tests for these mutations not that long ago
December 01, 2017
FDA Announces Approval, CMS Proposes Coverage of First Breakthrough-designated Test to Detect Extensive Number of Cancer Biomarkers
FDA approves test to detect mutations in 324 genes, two genomic signatures. Second to be approved with proposed coverage under FDA/CMS Parallel Review Program.
FDA News Release
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The U.S. Food and Drug Administration today approved the FoundationOne CDx (F1CDx), the first breakthrough-designated, next generation sequencing (NGS)-based in vitro diagnostic (IVD) test that can detect genetic mutations in 324 genes and two genomic signatures in any solid tumor type. The Centers for Medicare & Medicaid Services (CMS) at the same time proposed coverage of the F1CDx. The test is the second IVD to be approved and covered after overlapping review by the FDA and CMS under the Parallel Review Program, which facilitates earlier access to innovative medical technologies for Medicare beneficiaries.
“By leveraging two policy efforts aimed at expediting access to promising new technologies, we’ve been able to bring patients faster access to a breakthrough diagnostic that can help doctors tailor cancer treatments to improve medical outcomes and potentially reduce health care costs,” said FDA Commissioner Scott Gottlieb, M.D. “The FDA’s Breakthrough Device Program and Parallel Review with CMS allowed the sponsor to win approval for this novel diagnostic and secure an immediate proposed Medicare coverage determination within six months of the FDA receiving the product application.”
Compared to other companion diagnostics previously approved by the FDA that match one test to one drug, the F1CDx is a more extensive test that provides information on a number of different genetic mutations that may help in the clinical management of patients with cancer. Additionally, based on individual test results, the new diagnostic can identify which patients with any of five tumor types may benefit from 15 different FDA-approved targeted treatment options. Its results provide patients and health care professionals access to all of this information in one test report, avoiding duplicative biopsies.
“The F1CDx can help cancer patients and their health care professionals make more informed care decisions without the often invasive process of extracting tumor samples multiple times to determine eligibility for a single treatment or enrollment in a clinical trial,” said Jeffrey Shuren, M.D., director of the FDA’s Center for Devices and Radiological Health (CDRH). “With the run of one test, patients and health care professionals can now evaluate several appropriate disease management options.”
Today, CMS also issued a proposed national coverage determination of the F1CDx and other similar NGS IVDs for Medicare beneficiaries with advanced cancer (i.e., recurrent, metastatic or advanced stage IV cancer), who have not been previously tested using the same NGS technology and continue to seek further cancer therapy. The proposed national coverage determination provides coverage of NGS IVD tests to assist patients and their treating physicians in making informed cancer treatment decisions that improve health outcomes. Use of a test as a diagnostic also includes the ability to help patients and their treating physicians determine candidacy for cancer clinical trials.
“Through parallel review and collaboration, we speed access to innovative diagnostics, so that doctors are better able to deliver the best quality care to their patients and patients have access to these state-of-the-art tests,” said Seema Verma, Administrator of CMS. “Our proposal establishes clear expectations, while at the same time delivering better outcomes for the people we serve.”
This determination was made under the FDA-CMS Parallel Review Program, where the agencies concurrently review medical devices to help reduce the time between the FDA’s approval of a device and Medicare coverage. This voluntary program is open to certain premarket approval applications for devices with new technologies and to medical devices that fall within the scope of a Part A or Part B Medicare-benefit category and have not been subject to a national coverage determination.
The F1CDx detects gene mutations that may be found in any solid tumor and this information can be used by physicians according to professional guidelines to manage cancer patients. Moreover, it can be used as a companion diagnostic to identify patients with specific mutations who may benefit from certain FDA-approved treatments for non-small cell lung cancer, melanoma, breast cancer, colorectal cancer or ovarian cancer. Importantly, the F1CDx can detect genetic mutations that are indicated for multiple FDA-approved treatments, which extends beyond the previous “one test for one drug” model.
The device works by sequencing DNA from a patient’s tumor sample to determine the presence of gene mutations and alterations. It also detects certain molecular changes (microsatellite instability and tumor mutation burden). Clinical performance of the test was established through a least burdensome means by comparing the F1CDx to previously FDA-approved companion diagnostic tests that are currently used to determine patient eligibility for certain treatments. Results indicated that the test’s ability to detect select mutation types (substitutions and short insertions and deletions) representative of the entire 324 gene panel is accurate approximately 94.6 percent of the time.
The F1CDx had not been previously submitted for the FDA’s review because it is a laboratory-developed test, for which the agency has generally not enforced premarket review and other applicable requirements. However, at the test developer’s request, the FDA worked closely with them to help enter it into the agency’s newly established Breakthrough Device Program. Because of the test’s potential to consolidate multiple companion diagnostic claims for patients and health care providers in a single test, the F1CDx was granted Breakthrough Devicedesignation. Under the Breakthrough Device Program, the FDA provides intensive interaction and guidance to the company on efficient device development, which expedites evidence generation and the agency’s review of devices that provide for more effective treatment or diagnosis for life-threatening or irreversibly debilitating diseases for which no approved or cleared treatment exists or that offer significant advantages over the existing standard of care.
The FDA also reviewed the F1CDx application using a coordinated, cross-agency approach; the clinical review was conducted by FDA’s CDRH with support from FDA's Oncology Center of Excellence, while all other aspects of review and the final product approval determination was conducted by the FDA’s CDRH.
The FDA granted approval for the F1CDx test to Foundation Medicine, Inc.
