The Food and Drug Administration (FDA) has come under fire in recent years from the pharmaceutical and medical device lobbies for regulatory overreach. But that has not stopped the agency from inserting itself into the laboratory sector and the scores, if not hundreds, of unique tests it develops each year. On July 31, the FDA notified Congress that it would take steps to regulate some forms of laboratory-developed tests (LTDs). Its primary focus for now will be on companion diagnostics, which typically focus on determining whether or not specific drugs will benefit or hurt a patient’s treatment. Such tests have become more prominent in the field of oncology in recent years, although they have also been used to determine the efficacy of more commonly used drugs such as statins and blood thinners. While the FDA has had some discretionary authority over LDT kits since the mid-1970s, the agency has expressed concern that more of the assays contain components not legally marketed for clinical use while playing an expanding role in medical decisionmaking—all without any scrutiny as to their clinical utility. Cognizant of the sensitivity to the issue, the agency said it would take the better part of a decade to […]
The Food and Drug Administration (FDA) has come under fire in recent years from the pharmaceutical and medical device lobbies for regulatory overreach. But that has not stopped the agency from inserting itself into the laboratory sector and the scores, if not hundreds, of unique tests it develops each year.
On July 31, the FDA notified Congress that it would take steps to regulate some forms of laboratory-developed tests (LTDs). Its primary focus for now will be on companion diagnostics, which typically focus on determining whether or not specific drugs will benefit or hurt a patient’s treatment. Such tests have become more prominent in the field of oncology in recent years, although they have also been used to determine the efficacy of more commonly used drugs such as statins and blood thinners.
While the FDA has had some discretionary authority over LDT kits since the mid-1970s, the agency has expressed concern that more of the assays contain components not legally marketed for clinical use while playing an expanding role in medical decisionmaking—all without any scrutiny as to their clinical utility.
Cognizant of the sensitivity to the issue, the agency said it would take the better part of a decade to phase in the regulations. Moreover, it would also focus its scrutiny primarily on tests that have a high risk of affecting patient care. It would place tests into three classes, with only class III tests undergoing the highest level of scrutiny, including premarket review and a requirement that labs report any adverse events within 30 days of receiving notice they have occurred. Tests for rare diseases or with no other approved alternative would be exempt.
“The agency’s oversight would be based on a test’s level of risk to patients . . . while still providing flexibility to encourage innovation that addresses unmet medical needs,” said Jeffrey Shuren, M.D., director of the FDA’s Center for Devices and Radiological Health.
The reaction to the FDA’s move has been mixed.
The American Clinical Laboratory Association (ACLA) asked the FDA to exercise caution in how it regulated lab tests, noting that more regulation could stifle innovation.
“Laboratories have been regulated for decades by the Centers for Medicare and Medicaid Services under the Clinical Laboratory Improvement Amendments (CLIA) and by state law,” said ACLA President Alan Mertz. “Under the CLIA framework, a thorough and detailed regulatory process, we’ve seen an explosion of innovation in laboratory diagnostics that has allowed labs to diagnose and measure disease with an accuracy and precision never before possible.”
Amanda Murphy and Brian Weinstein, analysts with William Blair & Co., observed that “increased regulation is never a good thing” because it delays product introductions and stifles innovation. But they added that the FDA “appears to be taking a reasonable approach.”
Heather Creran, an Atlanta-based health care consultant with a focus on laboratories, observed that “better standards need to be applied to testing so that patients receive consistently accurate results, regardless of where the test is performed. In addition, patients are demanding and probably soon will and should be allowed to order many diagnostic tests on their own, without having to go through a doctor, and to that end, the results they receive need to be reliable.”
However, Creran added that “it is a mistake to layer another level of bureaucracy on top of the existing CMS and CLIA regulations. [The Department of Health and Human Services] needs to take a step back and consider how to reform the entire process from diagnostic test clearance, to laboratory oversight and reimbursement. If diagnostic tests are going to require an approval process, like pharmaceuticals and devices, then the reimbursement model must change as well. Currently, FDA-cleared tests cost multiple times more than that of the same non-FDA cleared test, but the reimbursement from Medicare and other third-party payers is the same.”
Foundation Medicine, a Massachusetts-based laboratory that has developed a number of molecular tests related to oncology, expressed confidence that the FDA’s regulation would not impede ongoing product development.
“Foundation Medicine developed [its FoundationOne assays] on the basis that tests for patients with advanced cancer should be fully comprehensive, thoroughly validated and backed by peer-reviewed publications,” the company said in a statement. “The stakes are extremely high for this patient population, and testing options must meet high quality standards. We will continue our work with FDA and ensure our approach meets its standards of excellence in this field.”
The FDA said it would release draft regulations later this year. They will be subject to a public comment period.
Takeaway: The decision by the Food and Drug Administration to regulate laboratory-developed tests could have a far-ranging impact on personalized medicine and the development of new assays.
