FDA Lab-Developed Test Oversight Could Spur Gene Test Advances

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By Jeannie Baumann

Sept. 22 — The FDA's efforts to reform regulatory structures—including its proposal to regulate laboratory-developed tests (LDTs)—could help spur innovation of new genetic testing technologies, the agency's commissioner said.

“I taught in business school, and standard teaching is that regulation and innovation are polar opposites, and they’re in conflict,” Robert M. Califf said during a Sept. 21 keynote address. However, “If we get this right, regulation will actually improve innovation,” he said.

As commissioner of the Food and Drug Administration, Califf delivered one of three keynote addresses at the American Medical Informatics Association'sannual policy event.

“Informatics is actually a gateway to bring the two together, to facilitate innovation and regulation at the same time,” he said.

Over the past decade, Califf said, there's been a digital confluence that has led to new opportunities to harness new forms of data, including wearable technologies that transmit health data and wider adoption of electronic health records.

“Ten years ago not many people had EHRs,” he said. “They’re pretty crappy right now, but everybody’s got one.”

Describing how regulation can improve innovation, the commissioner cited the advent of next-generation sequencing (NGS) tests, or high-throughput genetic screening tests that can examine millions of DNA variants at a time. These tests are expected to be critical for advancing the White House Precision Medicine Initiative, and the FDA issued two draft guidance documents in July that together propose how the agency wants to regulate these tests. The agency's current procedures for evaluating diagnostic tests are directed toward more traditional screening tests that measure a limited number of substances associated with a disease or condition, such as blood glucose or cholesterol levels.

“If we applied traditional regulation to [NGS tests], it’ll be 100 years from now before we even have a fraction of what we need to know about what all these measurements mean,” Califf said. “And yet the measurements are going to be in the electronic health record because costs are coming down to the order of $300 a person” for such tests, he said.

Next-Gen, Lab-Developed Tests

Most NGS tests fall under LDTs, defined as tests that are designed, manufactured and used in a single laboratory. Califf's remarks came just one day after Sen. Lamar Alexander (R-Tenn.), a chairman of the Senate health committee, blasted the agency's plan to regulate LDTs (10 LSLR 19, 9/30/16).

Released almost two years ago as draft guidance, the laboratory industry also opposes the FDA's plan to regulate LDTs, which the Centers for Medicare & Medicaid Services currently regulates. But the device industry generally supports the proposal, calling it an effort to improve oversight.

Califf told the AMIA meeting attendees that he's on a campaign to win the support of the academic medical centers on the LDT issue. The Association of American Medical Colleges issued a position statement in August expressing concerns that “the FDA’s regulation of LDTs as proposed would interfere with delivering innovative, cutting-edge medical care, negatively impact patients, or mire the development of critical new tests in a costly and laborious process.”

Califf said, “We’ve got to get academic medical centers to say it’s okay for us to be regulated because people deserve to have accurate tests when they go from one place to another. And we’re meeting tremendous resistance to this because people think FDA’s going to come in and just bring everything to a halt. But if we look at what’s happening with pipelines and PrecisionFDA, this is the way we’re thinking about it and I think we’re going to speed it up.”

PrecisionFDA is an online platform that allows scientists to test, pilot and evaluate new bioinformatics approaches using NGS processing.

New Ways to Evaluate Devices

Califf also indicated he would like to see changes in how regulators evaluate medical devices.

“Relying on spontaneous adverse event reporting to figure out what’s going on with the medical devices is just ridiculous,” he said. “We’ll never do away with that because it has great value for particular things, and we’re always going to need it. But we should be looking at numerators and denominators to understand what the risks are so that we can match them up with the potential benefits and also understand whether the signals are real.”

“What I'd like to do is go to a national system where active surveillance is the norm, leveraging claims and electronic health record data embedded in the health systems and sharing it with the ecosystems throughout the entire community.”

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bna.com

To contact the editor responsible for this story: Randy Kubetin at rkubetin@bna.com

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