FDA Launches Pilot Program to Guide Accuracy of Diagnostic Lab Tests for Cancer Biomarkers

An in vitro companion diagnostic test “provides information essential for the safe and effective use of a corresponding treatment,” the FDA said (https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-reduce-risks-associated-using-laboratory-developed-tests-identify). In oncology, many of the tests are used to identify biomarkers that may predict a patient’s response to cancer treatment. More recent FDA cancer drug approvals are indicated for patients with certain genetic variants that have been identified through one of those companion tests, and FDA often announces the approval of the tests alongside the drug.

Until recently, diagnostic tests that were laboratory-developed had been under (https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-reduce-risks-associated-using-laboratory-developed-tests-identify) an enforcement discretion approach (i.e., FDA generally did not enforce applicable requirements) and the agency did not generally review them for safety or effectiveness. However, increasing evidence and public concern about LDTs’ accuracy and implications for treatment decisions led the FDA to finalize the new rule and create the pilot program.

Under the one-year pilot program, the FDA will ask up to nine drug manufacturers to provide performance information for the tests used to enroll patients into drug approval clinical trials. FDA will post on its website the minimum performance characteristics recommended for similar selection tests for the approved drug to help laboratories guide their development of LDTs to identify specific biomarkers used for identifying patient-appropriate cancer treatments. 

“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,” the FDA said (https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-reduce-risks-associated-using-laboratory-developed-tests-identify).

Drug manufacturers who wish to participate in the pilot program should submit a statement of interest (https://www.fda.gov/medical-devices/in-vitro-diagnostics/oncology-drug-products-used-certain-in-vitro-diagnostics-pilot-program) to their investigational new drug applications, new drug applications, or biologic license applications for consideration. 

“[The] pilot program launch, and corresponding guidance publication, is a step forward in improving cancer care for patients,” Richard Pazdur, MD, 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, said (https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-reduce-risks-associated-using-laboratory-developed-tests-identify). “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.”

Oncology nurses can explore information and resources for many of the biomarkers that LDTs identify in the ONS Biomarker Database (https://biomarkers.ons.org/biomarkers/). It covers 21 cancer types as well as tumor-agnostic biomarkers with content curated specifically for oncology nurses to use to support clinical decisions at the point of care.