FDA Launches Pilot Program Using Cancer Biomarkers to Mitigate Risk

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FDA’s new voluntary pilot program is designed to help clinicians select appropriate cancer treatments for patients.

On June 20, 2023, FDA announced a new voluntary pilot program for certain oncology drug products used with certain corresponding in-vitro diagnostic tests. The goal of this program is to help clinicians select appropriate cancer treatments for patients.

Under current guidelines, an in-vitro companion diagnostic test is one that provides information needed for the safe and effective use of a corresponding treatment. In certain limited circumstances, FDA may decide to approve a life-saving treatment that requires an in-vitro companion diagnostic even if a corresponding in-vitro companion diagnostic has not yet received marketing authorization. In these situations, laboratory developed tests (LDTs) are being used for patient treatment decisions, but these are not generally reviewed by the agency for safety or effectiveness.

The new pilot program hopes to address concerns that LDTs do not provide accurate or reliable test results, according to an agency press release. FDA will request drug manufacturers to provide performance information for the tests used to enroll patients into the clinical trials that support drug approval. Following assessment of that information, FDA will publish minimum performance characteristics recommended for similar tests that may be used to select patients for treatment with the approved drug. In doing this, laboratories may use this information to guide LDT development to identify specific biomarkers used for selecting cancer treatment. According to the release, this transparency aims to help facilitate better and more consistent test performance, ultimately resulting in better drug selection and improved care for cancer 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, director, Center for Devices and Radiological Health, FDA, in the release. “The pilot aims to help by making transparent performance recommendations for diagnostic tests used to select certain oncology drug treatments.”


The initial phase of the pilot program is intended to last up to one year. 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 website following drug approval.

Source: FDA