FDA Launches Pilot Program to Help Reduce Risks Associated With Using Laboratory Developed Tests to Identify Cancer Biomarkers

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The U.S. Food and Drug Administration (FDA) announced a new voluntary pilot program to help physicians select safe and effective cancer treatments, with guidance on in vitro companion diagnostic tests to determine which corresponding oncology drugs may provide the most benefit for their patients.

“We believe this guidance and the launch of the pilot program are important steps toward addressing safety risks posed by the use of poorly performing laboratory developed tests,” highlighted Jeff Shuren, MD, JD, Director of the Center for Devices and Radiological Health at the FDA. “The pilot aims to help by making transparent performance recommendations for diagnostic tests used to select certain oncology drug treatments,” he explained.

Current FDA Policy on Diagnostic Tests

Under the current FDA policy, an in vitro companion diagnostic test is one that provides information essential for the safe and effective use of a corresponding treatment. For instance, specific tests may be used to identify patients such as those with particular genetic mutations who may or may not benefit from certain cancer treatments.

In specific instances, tests offered as laboratory developed tests may be used for patient treatment decisions. Laboratory developed tests are diagnostics that have mostly been under an enforcement discretion approach—such that the FDA typically has not enforced applicable requirements with respect to most laboratory developed tests—and may not reviewed by the agency for safety or effectiveness. The current policy specifies that under certain limited circumstances where a general enforcement discretion approach has not been applied, such as during a declared public health emergency, the FDA may decide to approve a life-saving treatment requiring the use of an in vitro companion diagnostic test—even if the test has not yet received marketing authorization.

However, the agency has become increasingly concerned that some laboratory tests not authorized by the FDA may provide inaccurate and unreliable test results or perform poorly compared with FDA authorized tests. This may negatively impact treatment decisions.

Overview of the New Pilot Program

The new pilot program could help reduce the risks associated with using laboratory developed tests for oncology drug treatment decisions while the FDA continues to work on a broader approach and rules for these tests. Through the pilot program, the agency will request performance data from drug manufacturers for the tests used to enroll patients in clinical trials that support drug approval.

After assessing the data, the FDA expects to announce the minimum performance characteristics recommended for similar tests that may be used to select patients for treatment with the approved drug. Laboratories may use this information to guide their development of the tests and identify specific biomarkers used for selecting cancer treatments.


With greater transparency, the FDA aims to help facilitate better and more consistent performance of laboratory developed tests, resulting in better drug selection and improved care for patients with cancer.

“Today’s pilot program launch, and corresponding guidance publication, is a step forward in improving cancer care for patients,” emphasized Richard Pazdur, MD, Director of the Oncology Center of Excellence and Acting Director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research Offices and Divisions at the FDA. “This pilot program demonstrates the FDA’s commitment to collaborating with the pharmaceutical industry and other key stakeholders to address concerns and questions around the use of unauthorized diagnostics,” he concluded. 

The FDA anticipated that the initial phase of the pilot program will last up to 1 year—during which the FDA will evaluate no more than nine drug sponsors for possible acceptance into the pilot. The minimum recommended performance characteristics for in vitro companion diagnostic tests used with each approved drug product under the pilot, based on the clinical trial assays, will be made publicly available on the FDA’s website following drug approval.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.