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FDA News Release

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

Pilot Geared Toward Sponsors of Certain Oncology Drug Products Used with Certain In Vitro Diagnostic Tests to Identify Patients for Certain Cancer Treatments

For Immediate Release:

Today, the U.S. Food and Drug Administration announced a new voluntary pilot program for certain oncology drug products used with certain corresponding in vitro diagnostic tests to help clinicians select appropriate cancer treatments for patients. 

“We believe this guidance and the launch of the pilot program are important steps towards addressing safety risks posed by the use of poorly performing laboratory developed tests,” said Jeff Shuren, M.D., J.D., director of the FDA’s Center for Devices and Radiological Health. “The pilot aims to help by making transparent performance recommendations for diagnostic tests used to select certain oncology drug treatments.”

Under 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. In oncology, for example, specific tests may be used to identify patients, such as those with a particular genetic mutation, who may or may not benefit from certain cancer treatments.

The current policy also provides that, in certain limited circumstances, the FDA may decide to approve a life-saving treatment that requires use of an in vitro companion diagnostic even if a corresponding in vitro companion diagnostic has not yet received marketing authorization. In these cases, tests offered as laboratory developed tests (LDTs) are being used for patient treatment decisions. LDTs are diagnostics that have generally been under an enforcement discretion approach (such that the FDA generally has not enforced applicable requirements with respect to most LDTs) and are not generally reviewed by the agency for safety or effectiveness. The FDA has not applied this general enforcement discretion approach in certain circumstances, such as during declared public health emergencies.

The agency has become increasingly concerned that some tests made by laboratories and not authorized by the FDA may not provide accurate and reliable test results or perform as well as FDA authorized tests. This may negatively impact treatment decisions. This pilot program is one step that may be helpful in reducing the risk of using LDTs for oncology drug treatment decisions while we continue to work on a broader approach for LDTs, including moving forward with rulemaking. 

Through the pilot program, the FDA will request, from drug manufacturers, performance information for the tests used to enroll patients into the clinical trials that support drug approval.  Based on an assessment of that information, the FDA will post to the FDA website 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 LDTs to identify specific biomarkers used for selecting cancer treatment. This transparency aims to help facilitate better and more consistent performance of these 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,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “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.” 

As discussed in the guidance, the initial phase of the pilot program is anticipated to last up to one 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 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.

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The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.


Jim McKinney

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