Skip to content

MedPage Today – FDA Delays Guidance on Lab-Developed Tests

MedPage Today – FDA Delays Guidance on Lab-Developed Tests

The FDA backed off from a plan to issue final guidance for the regulation of laboratory developed tests (LDTs).

“We have been working to develop a new oversight policy for laboratory developed tests, one that balances patient protection with continued access and innovation, and realize just how important it is that we continue to work with stakeholders, our new Administration, and Congress to get our approach right. We plan to outline our view of an appropriate risk-based approach in the near future,” the agency said in a press statement.


The agency also emphasized the need for “accurate, reliable, and clinically valid tests to make good health care decisions — inaccurate or false test results can harm individual patients.”

Republicans in Congress, who believe such oversight unnecessary, applauded the decision. Patient advocates and research groups, concerned about the validity and accuracy of such tests, are hopeful a new administration will take up their cause.

LDTs are developed and used in a single laboratory, primarily to diagnose illness or to help determine the best treatment for individual patients. Historically, these tests have not been regulated by the FDA.


Instead, the Centers for Medicare and Medicaid Services (CMS), under the Clinical Laboratory Improvements Amendment (CLIA), has conducted reviews and inspections of the labs to ensure that adequate equipment is in place, and personnel are properly trained, to perform the tests.

The FDA has had the ability to regulate LDTs since 1976, explained Jeff Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health at a congressional briefing last year.

Initially these tests were simple and scarce and the agency did not see a reason to intervene. But over the past decade, the tests have become more numerous, more complex, and heavily marketed, he explained, so the agency began taking steps towards regulation.


In 2014, the FDA published a draft rule outlining a framework through which the agency could expand its jurisdiction over molecular tests to also include LDTs.

In 2015, the agency issued a report citing 20 examples where LDTs may have caused patient harm. Half of the case studies related to cancer — detecting the disease or the risk of disease or guiding treatment. Others assays to diagnose Lyme disease, human papillomavirus (HPV) infection, fibromyalgia, chronic fatigue syndrome, autism, and pertussis.

In one case, a defective non-invasive prenatal test, or “cell-free DNA test” used to detect fetal chromosomal abnormalities, was found to have contributed to decisions to abort healthy fetuses.

In late November, the FDA announced that it would wait for the new administration, and freeze the finalization of guidance that would have altered the regulation of LDTs, to the delight of some Republicans in the House and Senate.

“I am glad that the FDA has listened and will delay making final its current draft guidance that could have halted more than 60,000 lab-developed tests in their tracks, and will work with Congress and the new administration on next steps,” said Sen. Lamar Alexander (R-Tenn.) in a press statement.


In a previous statement, Alexander has stressed that subjecting a single test to FDA approval could cost academic centers from $30-$75 million.

“It was the right call,” said Rep. Fred Upton (R-Mich.), chairman of the House Energy and Commerce Committee, in a press statement. His committee has debated the issue of LDT regulation for the last 3 years and has said that adopting non-binding guidance is “not the best approach. Working together, we are forging significant consensus among a number of patient groups, labs, and manufacturers around a 21st century approach uniquely designed with all diagnostic tests in mind from the outset,” he said.

While critics of LDT regulation view it as duplicative, costly, and a barrier to innovation, others are not convinced.

“Current oversight of LDTs falls short of ensuring these tests produce accurate and meaningful results, according to a press statement from the American Cancer Society Cancer Action Network (ACS CAN).


Many tests now come to market without independent verification that their results were accurate or clinically valid, the group explained. Without final guidance, labs that perform tests to diagnose diseases where the test is critical to safety, health, or a treatment decision will not be required to validate a test’s accuracy and clinical claims with FDA.

In addition, they will not have to report adverse events impacting patient health.


ACS CAN urged the new administration to “prioritize the safety of patients and continue to work with stakeholders to develop and implement a framework for LDT oversight.”

Jeff Allen, PhD, president and CEO of Friends of Cancer Research, and an advocate for LDT regulation by the FDA, testified before a Senate Committee in October on the topic. He said he was not surprised by the decision to delay final guidance.

“It has been a challenging topic,” he told MedPage Today in a phone call, during which a public affairs representative was present. Allen emphasized that the issue had become quite polarizing in Congress, and noted that it could be wise to pursue legislation, “to at least codify the fact that FDA has the authority to regulate these products if they choose to do so.”

“These tests are are of growing importance, and of growing use in the future of personalized medicine. So I think we’ll see this debate continue,” he added.

An FDA representative said the agency could not speculate on “the potential, future policy or timing” of any new guidance.