FDA Announces Increased Oversight of Laboratory Developed Tests

August 4, 2014

Last week, the US Food and Drug Administration (FDA) announced two steps it will take to ensure the safety and utility of certain diagnostic tests.

"Margaret Hamburg official portrait" by Food and Drug Administration - High-res version of image found here:http://www.fda.gov/ucm/groups/fdagov-public/documents/image/ucm164991.jpg. Licensed under Public domain viaWikimedia Commons.

Last week, the US Food and Drug Administration (FDA) announced two steps it will take to ensure the safety and utility of certain diagnostic tests. First, the agency is issuing final guidance on the development, review, and approval or clearance of companion diagnostics.1Second, it has signaled to Congress its intention to publish a proposed risk-based oversight framework for laboratory developed tests (LDTs)2, i.e., tests that are designed, manufactured, and used within a single laboratory.

The final guidance issued on companion diagnostics includes a requirement that these tests be approved together with new targeted drugs. The goal of this mandate, according to the announcement, is to help companies identify the need for these tests at the earliest stages of drug development, which will ultimately result in faster access to new treatments for patients with serious diseases.

The FDA notified Congress regarding its intention to regulate LDTs to comply with the Food and Drug Administration Safety Act of 2012, which stipulates that any new draft guidelines or regulations may not be published sooner than 60 days after Congress has been alerted. In the notification, the agency indicated that oversight would be introduced over several years, beginning with the most high-risk LDTs.

The goal of these two actions is to address concerns about safety, accuracy, and reliability of tests that can have serious consequences for patients.

“Inaccurate test results could cause patients to seek unnecessary treatment or delay and sometimes forgo treatment altogether. Today’s action demonstrates the agency’s commitment to personalized medicine, which depends on accurate and reliable tests to get the right treatment to the right patient,”3noted FDA Commissioner Margaret A. Hamburg, MD.

Historically, LDTs were simple, low risk, available to small numbers of patients, and often for rare conditions. For these reasons, the FDA has generally exercised enforcement discretion by not regulating these tests.

In the nearly 40 years since that policy was decided, however, the role of LDTs in the healthcare field has evolved. They are more commonly used to diagnose serious diseases such as Alzheimer’s disease and certain types of cancer.

Modern LDTs, which include nearly all genetic tests, are also often more complex and used in greater numbers of patients than in the past. Some LDTs directly compete with FDA-approved tests created by manufacturers.

The lack of oversight for LDTs “is concerning,” writes Jeffrey Shuren, MD, JD, director of the FDA’s Center for Devices and Radiological Health. “Without appropriate safeguards, neither patients nor their health care providers can be assured that these tests are safe and effective. This is particularly troubling when an FDA-approved test is available, because it puts patients at unnecessary and avoidable risk. It also stifles innovation by creating disincentives for conventional manufacturers to invest in developing new, medically important tests.”4

At present, laboratories conducting LDTs are regulated by the US Centers for Medicare and Medicaid Services under the Clinical Laboratory Improvement Amendments (CLIA).

In a statement in response to the FDA’s announcement, the American Clinical Laboratories Association “urged the agency to exercise caution, and expressed concern that another layer of regulation could stifle diagnostic innovation and ultimately jeopardize patient access to timely and effective treatments.”5


  1. Food and Drug Administration. In Vitro Companion Diagnostic Devices - Guidance for Industry and Food and Drug Administration Staff. http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM262327.pdf. Accessed August 1, 2014.
  2. Food and Drug Administration. Notification to Congress — FDA’s Laboratory Developed Tests Framework. http://www.fda.gov/downloads/MedicalDevices/ProductsandMedicalProcedures/InVitroDiagnostics/UCM407409.pdf. Accessed August 1, 2014.
  3. Food and Drug Administration. FDA takes steps to help ensure the reliability of certain diagnostic tests. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm407321.htm. Accessed August 1, 2014.
  4. FDA Voice. Curbing Risk, Not Medical Innovation, in Personalized Medicine. http://blogs.fda.gov/fdavoice/index.php/2014/07/curbing-risk-not-medical-innovation-in-personalized-medicine/. Accessed August 1, 2014.
  5. American Clinical Laboratory Association. ACLA Expresses Concern with FDA Guidance on Laboratory Developed Tests (LDTs). http://www.acla.com/acla-expresses-concern-with-fda-guidance-on-laboratory-developed-tests-ldts/. Accessed August 2, 2014.