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Sept. 18 — The FDA is working with industry to advance precision medicine through FDA guidances and expanded oversight, an agency official Sept. 18 told a biotech conference.
E. David Litwack discussed what he called the least controversial topic, the Food and Drug Administration's approval of companion diagnostics, and most controversial, the FDA's plans for increased oversight of laboratory developed tests (LDTs).
Both, along with the FDA's regulatory strategies for next generation sequencing (NGS), are part of the modernization of regulation of genomic-based tests, he said.
Litwack, who is a policy adviser for the Office of In Vitro Diagnostics and Radiological Health in the FDA's Center for Devices and Radiological Health, delivered his remarks at a regulatory overview session of the 2015 BIO IP & Diagnostics Symposium in Alexandria, Va.
Precision medicine is an emerging approach for treating and preventing diseases that takes into account variability in genes, environment and lifestyle for each person. When implemented, precision medicine is expected to transform clinical care, Litwack said.
“The FDA's vision for President Obama's precision medicine initiative is to implement new regulatory policies to promote research and accelerate the translation of precision medicine technologies into treatments that benefit patients,” Litwack said.
“Our near-term goal is to create standards and shared resources that will enable the development of knowledge for research and patient decision making,” he said. “Our longer term goal: implement standards-based regulation of diagnostic tests that will ensure that the tests patients receive provide accurate, reproducible and meaningful results,” Litwack said.
Litwack said that a companion diagnostic is defined as a test essential for the safe and effective use of a corresponding therapeutic product.
The FDA's approach requires contemporaneous approval of the therapeutic and the test, he said.
“What's at work here is that the therapeutic's sponsor is driving this and so is responsible for assuring that a companion diagnostic device will be brought forward. The diagnostic sponsor, however, is responsible for the submission of the companion diagnostic for approval and for performance compliance,” he said. “The tricky part is that the therapeutic sponsor knows the drug regulations while the diagnostic sponsor knows device regulations. That's why it's important to have early discussions with the FDA.”
Two major lessons learned from the companion diagnostics experience, Litwack said, are that all development programs are different and that the FDA should use all possible regulatory mechanisms to create an approval pathway that works.
• early determination of companion diagnostics need is better for co-development, but the FDA can still handle variations;
• accelerated drug approval doesn't significantly change when companion diagnostics are needed; and
• drug and diagnostic sponsors should carefully define expectations for each other.
Litwack also noted that bridging the gap from an assay used in a clinical trial to in vitro diagnostics (IVD)—a test performed outside of the human body in a lab or other alternative environment—isn't easy, so it is important to save samples, consider co-variates and avoid bias.
Until it announced its plan to increase its oversight of LDTs, the FDA had exercised enforcement discretion over the tests. Clinical laboratories that perform diagnostic procedures such as LDTs are overseen by the Centers for Medicare & Medicaid Services (CMS) under the Clinical Laboratory Improvement Amendments (CLIA).
Litwack said that the FDA defines an LDT as an IVD that is intended for clinical use and is designed, manufactured and used within a single laboratory.
The goals of the FDA's proposed framework for increased regulatory oversight of LDTs, Litwack said, are to collect basic information on all LDTs through an optional notification process; use public processes such as advisory committees to obtain input on risk and priority for oversight; phase in enforcement of premarket review over nine years based on risk; and continue some enforcement discretion for specific categories determined by the FDA to be in the best interest of public health.
The FDA's LDT proposal generated immediate opposition from the American Clinical Laboratories Association, drew requests from organizations such as the American Medical Association to withdraw the plan and caused some groups to submit alternative regulatory oversight plans for LDTs.
Litwack summarized some of the written comments the FDA had received in response to its LDT plan.
“We heard that traditional LDTs [which use techniques and components marketed for clinical use that are interpreted directly by qualified health care providers] should be under complete enforcement discretion, and for LDTs for unmet needs that ending enforcement discretion once one test is cleared or approved could lead to market monopolies and access issues,” Litwack said.
He noted that some said that certain LDTs for public health purposes, such as newborn screening tests and public health surveillance tests, also should remain under continued enforcement discretion.
Litwack discussed the FDA-CMS task force on regulatory oversight of LDTs, “It's important to note that the FDA and the CMS have a long-standing relationship and understand the important role accrediting organizations [AOs] play in the implementation of the CLIA,” he said. “The FDA and CMS have met with all AOs to discuss their unique requirements for laboratories and how these requirements relate to LDTs.”
Most importantly, Litwack said, the task force has reviewed the intersection of FDA and CLIA regulations.
“The FDA quality system [QS] regulations and the CMS CLIA regulations do not specify how a lab should meet requirements, leaving room for labs to implement processes that meet their unique business operations and needs,” Litwack said.
“The FDA and CLIA regulations are not in conflict,” he concluded. “The task force confirmed that there are no activities required by CLIA that would put a laboratory in violation of the FDA QS regulations and vice versa.”
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