Home 5 Clinical Diagnostic Insider 5 The Diagnostics Pipeline: FDA Launches Pilot for Cancer Companion Diagnostic LDTs

The Diagnostics Pipeline: FDA Launches Pilot for Cancer Companion Diagnostic LDTs

by | Jul 31, 2023 | Clinical Diagnostic Insider, Diagnostic Testing and Emerging Technologies, FDA-dtet

New program aims to provide greater transparency on the recommended performance characteristics of such tests.

On June 21, the FDA published a guidance document announcing the launch of a pilot program to regulate laboratory-developed tests (LDTs) used as companion diagnostics for oncology drug therapies.1 The goal of this voluntary program is to minimize the risks associated with using LDTs for cancer treatment selection by providing greater transparency on the recommended performance characteristics of such tests.

Current Regulation of Cancer Companion Diagnostic LDTs

In vitro companion diagnostic tests provide information that helps determine whether a corresponding treatment is likely to be safe and effective in a specific patient. In the oncology setting, tests are used to identify patients who may benefit from certain types of cancer treatments—for instance, those who have genetic mutations that the treatment targets.

Oncology drug developers must indicate whether their products require use of an in vitro companion diagnostic. In many cases, these diagnostics are approved alongside the new drug—but if no in vitro test has received FDA marketing authorization, the agency may still clear the oncology drug. This would allow use of an LDT for the limited purpose of determining whether a particular patient might benefit from the treatment. In keeping with the FDA’s oversight of LDTs, this agency practice is a policy, rather than a formal regulation.

In the press announcement accompanying the new guidance, the FDA said it has become “increasingly concerned” that some companion LDTs “may not provide accurate and reliable test results or perform as well as FDA authorized tests.”2 The agency has previously identified LDT issues such as insufficient evidence of clinical validity, poor or absent controls, and a lack of structure for adverse event reporting, all of which can negatively affect treatment decisions or outcomes.3, 4,5

The New Cancer Companion Diagnostic LDTs Pilot

The guidance, “Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program,” explains how the pilot will work. Specifically, the FDA will ask pharmaceutical manufacturers to furnish performance information for tests used to enroll patients in clinical trials. The agency will analyze the data and, if appropriate, extrapolate the clinical validity of tests used in patient selection “to other tests of the same type with similar analytical performance.” The resulting minimum performance characteristics for similar clinical tests will then be posted on a dedicated part of the FDA’s website so that laboratories can use the information when developing their own LDTs to identify biomarkers for cancer treatment selection.6

“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, MD, JD, 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.”2

Toward a Comprehensive LDT Solution

Although the pilot program will only include up to nine pharmaceutical companies, the implication that the FDA will not approve LDTs that fail to meet the resulting benchmarks for biomarker performance means that the guidance will significantly impact labs and other LDT developers of companion diagnostic tests.

However, the real takeaway from the pilot guidance is that the FDA has once again signaled its intention to address all LDTs: According to the agency’s press statement, “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.”2

Whether deliberate or unintentional, the likely end result of the FDA’s program will be to pressure Congress to pass the Verifying Accurate Leading-edge IVCT Development (VALID) Act or another bill to address LDT issues legislatively.7 Ironically, this dynamic may lead drug makers to balk at investing the time and resources necessary to enlist in the new FDA pilot, preferring instead to wait for a broader resolution, whether regulatory or legislative.


  1. https://public-inspection.federalregister.gov/2023-13134.pdf
  2. https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-reduce-risks-associated-using-laboratory-developed-tests-identify
  3. https://www.fda.gov/media/102367/download
  4. https://www.fda.gov/medical-devices/in-vitro-diagnostics/laboratory-developed-tests
  5. http://wayback.archive-it.org/7993/20171115144712/https:/www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Reports/UCM472777.pdf
  6. https://www.fda.gov/medical-devices/in-vitro-diagnostics/oncology-drug-products-used-certain-in-vitro-diagnostics-pilot-program
  7. https://www.g2intelligence.com/despite-mdufa-setback-valid-act-is-back-on-track-for-passage/


Here are the key laboratory testing and diagnostics products clearances announced since mid-June 2023:

New FDA Approvals & Emergency Use Authorizations (EUAs)

Manufacturer Product(s)
ARUP Laboratories Clearance for AAV5 DetectCDx™ electrochemiluminescence antibody assay for detection of antibodies in human plasma that bind to adeno-associated virus serotype 5 (AAV5) as companion diagnostic to identify patients eligible for treatment with gene therapy for severe hemophilia
Renalytix De Novo clearance for KidneyIntelX.dkd™ blood-based prognostic test for assessing risk of kidney function decline in those with diabetes and early-stage kidney disease
Roche 510(k) clearance for Elecsys® β-Amyloid (1-42) CSF II (AB42) and Elecsys® Total-Tau CSF (tTau) assays used together to provide ratio of tTau to Aβ42 proteins in diagnosing Alzheimer’s disease
Anumana Breakthrough device designation for AI-guided ECG algorithm for early identification of cardiac amyloidosis
Michigan State University laboratories, Department of Medicine Olin Student Health Center EUA for In-Dx SARS-CoV-2 RT-LAMP Assay for COVID-19 detection
Diagnostica Stago 510(k) clearance for automated STA R Max3® and STA Compact Max3® coagulation analyzers for clot-based, immunoturbidimetric, and colorimetric testing

New CE Marks & Global Certifications

Notable European CE certifications announced in June 2023:

New Approvals in Europe

Manufacturer Product
Creo Medical Speedboat Inject dissection/resection device for upper gastrointestinal use
Thorne HealthTech NanoDrop instrument for collecting capillary whole-blood samples
DiaSorin LIAISON® B·R·A·H·M·S MR-proADM™ immunodiagnostic assay to assess MR-pro-ADM biomarker released during inflammatory responses and infections

Other international clearances announced in June 2023:

Manufacturer Country Product(s)
Laboratory for Advanced Medicine & Health Group (Helio Genomics partner company) China Adaptation of Helio’s HelioLiver test to genetic makeup of Asia-Pacific population
LumiraDx UK LumiraDx SARS-CoV-2 Ag Ultra test
LumiraDx UK LumiraDx SARS-CoV-2 Ag & RSV test
Gencurix South Korea Droplex EGFR Mutation Test v2 for use with Bio-Rad’s Droplet Digital PCR instrument to identify EGFR mutations in non-small cell lung cancer
Neoteryx (subsidiary of Trajan Scientific and Medical) Australia Remote-use Mitra® blood microsampling instrument and Mitra® Blood Collection Kits for at-home blood sample collection
Roche Countries relying on World Health Organization prequalification Roche cobas® HPV test

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