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The U.S. Food and Drug Administration has issued a final guidance on the development, review, and approval or clearance of companion diagnostics used to detect some gene-based cancers. Along with that, consistent with the requirements of the FDA Safety and Innovation Act of 2012 (FDASIA), the agency also notified Congress of its intention to publish a proposed risk-based oversight framework for laboratory developed tests (LDTs), which are designed, manufactured, and used within a single laboratory, which include some genetic tests and tests that are used by health care professionals to guide medical treatment for their patients. (The FDA already oversees direct-to-consumer tests regardless of whether they are LDTs or traditional diagnostics.)

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"Ensuring that doctors and patients have access to safe, accurate, and reliable diagnostic tests to help guide treatment decisions is a priority for the FDA," FDA Commissioner Margaret A. Hamburg, MD, said in a news release. "Inaccurate test results could cause patients to seek unnecessary treatment or delay and sometimes forgo treatment altogether. [This] action demonstrates the agency's commitment to personalized medicine, which depends on accurate and reliable tests to get the right treatment to the right patient."


The new guidance for companion diagnostics is intended to help companies identify the need for such tests during the earliest stages of drug development and plan for the development of a drug and a companion test at the same time-the ultimate goal being to stimulate early collaborations resulting in faster access to new treatments for patients, the news release noted.


The agency has historically exercised enforcement discretion over laboratory-developed tests (generally not enforced applicable regulatory requirements), but these tests now may compete with FDA-approved tests without clinical studies to support their use.


"The FDA is seeking a better balanced approach for all diagnostics," Jeffrey Shuren, MD, Director of the FDA's Center for Devices and Radiological Health, said. "The agency's oversight would be based on a test's level of risk to patients, not on whether it is made by a conventional manufacturer or in a single laboratory, while still providing flexibility to encourage innovation that addresses unmet medical needs."


The agency intends to propose continuing to exercise enforcement discretion for low-risk LDTs, those for rare diseases, and, under certain circumstances, LDTs for which there is no FDA-approved or cleared test, according to the news release. The plan is also to publish a draft guidance outlining how laboratories can notify the FDA that they are currently manufacturing and using LDTs, how to provide information about their LDTs, and how they can comply with the medical device reporting requirements.


The FDA is required, based on a provision in FDASIA, to provide at least 60 days' notice to Congress before publishing for public comment any draft guidance on the regulation of LDTs. As such, the comment period will open at a later date when the draft guidances are published in the Federal Register, and the public is alerted to the start of the comment period. The agency noted that it also intends to hold a public meeting during the comment period to collect additional input.