The US Food and Drug Administration (FDA) has issued a final guidance on the development, review, and approval or clearance of companion diagnostics, which are tests used to identify patients that would benefit or not from specific therapies. These tests are commonly used to detect certain types of gene-based cancers.
Companion diagnostics are medical devices that help doctors decide which treatments to offer patients and which dosage to give, tailored specifically to the patient. Because the companion diagnostic test is designed to be paired with a specific drug, the development of both products requires close collaboration between experts in both FDA’s device center, which evaluates the test to determine whether it may be cleared or approved, and FDA’s drug center, which evaluates the drug to determine whether it may be approved.
The companion diagnostics guidance, In Vitro Companion Diagnostic Devices: Guidance for Industry and Food and Drug Administration Staff, is intended to help companies identify the need for these tests during the earliest stages of drug development and to plan for the development of a drug and a companion test at the same time. The ultimate goal of the final guidance is to stimulate early collaborations that will result in faster access to promising new treatments for patients living with serious and life-threatening diseases. This guidance finalizes and takes into consideration public comment on the draft guidance issued in 2011.
In a separate development, the FDA has 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. They include some genetic tests and tests that are used by healthcare 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.
While the FDA has historically exercised enforcement discretion over LDTs (generally not enforced applicable regulatory requirements), these tests may compete with FDA-approved tests without clinical studies to support their use. The LDT notification to Congress provides the anticipated details of the draft guidance through which the agency would propose to establish an LDT oversight framework, including pre-market review for higher-risk LDTs, such as those that have the same intended use as FDA-approved or cleared companion diagnostics currently on the market. The draft guidance would also propose to phase in enforcement of pre-market review for other high risk and moderate risk LDTs over time. The agency intends to propose continuing to exercise enforcement discretion for low-risk LDTs, LDTs for rare diseases and, under certain circumstances, LDTs for which there is no FDA-approved or cleared test.
The FDA also intends 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.
A provision in Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) requires the FDA to provide at least 60 days’ notice to Congress before the agency publishes 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, according to the FDA. The agency also intends to hold a public meeting during the comment period to collect additional input.
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