FDA’s recent proposal to establish “a collaborative community” of stakeholders to support the agency in its decision-making process for diagnostics regulation could also serve as “a compelling new paradigm” for the cancer research community, Commissioner Scott Gottlieb said at a Friends of Cancer Research event Thursday (Sept. 13).

In August, FDA floated what it pegged as technical assistance to pending legislation that would cement the agency’s authority to regulate high-risk clinical laboratory diagnostics, including laboratory-developed tests and in-vitro diagnostics. FDA, in its technical assistance, proposed the establishment of “a collaborative community” of stakeholders –including patients, care partners, academics, health care professionals, payers, federal and state agencies, industry, and international regulatory bodies.

That community, according to the technical assistance document, would make recommendations to FDA on development and regulatory issues, such as: how to mitigate measures for in-vitro clinical tests; development and performance standards for in-vitro clinical tests; the scientific and clinical evidence needed to support new claims for tests; new technologies and methodologies for tests; stakeholder engagement; new approaches and solutions to multifaceted problems; and development of effective policies and processes.

Gottlieb hopes the concept will serve as a model for the cancer research community. “For example, FDA would hope to work on an on-going basis with leading oncologists, pathologists, and other outside experts in real time to develop standards for analytical validity for different types of tests,” he said Thursday. “We’d also seek to work with the right experts to evaluate emerging clinical evidence to support the clinical validity of newly identified variants.”

Building off the steps FDA has taken to regulate next-generation sequencing tests, the agency would also leverage public databases and expertise of academic medical centers, and work with researchers in oncology and other medical specialties to develop regulatory expectations for novel technologies.

Gottlieb also highlighted other FDA efforts to streamline medical device regulation, which he said could be used to accelerate cancer drug development. Specifically, the agency’s device center is working with the National Institute of Standards and Technology to advance reference materials that can evaluate the analytical performance of NGS tests and has introduced an approach that would allow reporting of additional oncogenes or gene variants of the same type without an additional submission to FDA.

Such advancements could help reduce patient screening time, accelerate cancer drug development and improve clinical outcomes, Gottlieb said. However, he added, the device program is still operating under an outdated regulatory framework that “isn’t well suited” to challenges presented by newer technologies.

“The decision to exercise enforcement discretion over laboratory developed tests was made in a different time and under different circumstances. We must ask whether that approach still makes sense?” the FDA chief said.

He continued: “If the opportunity exists to modernize our approach to diagnostics regulation through legislation, FDA is a willing partner in those efforts. We’ve already made clear we’ll embrace it and offer new ideas. We know there’s a lot of work to be done. And we know that stakeholder feedback is critical.”

In its August technical assistance document, FDA floated legislative language that would set up two risk categories for diagnostic tests: first-of-a-kind screening cross-labeled devices that would be subject to FDA premarket review, and low-risk devices that could voluntarily go through FDA’s precertification process.

FDA believes that a reformed, streamlined approach to diagnostics regulation could result in fewer than 10 percent of tests requiring individual premarket review, Gottlieb said Thursday. About 40 percent percent would be eligible for a precertification pathway and more than 50 percent would be exempt from premarket review, he added.

“In addition, we can foresee a market in which only about one-half of the test modifications that are currently subject to FDA premarket review would still be reviewed premarket,” the commissioner said. “So, the process we envision can be far more efficient and even more robust than our current efforts.” 

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