How to approve multi-cancer detection tests?

November 29, 2023, the FDA convened an Advisory Committee Meeting on multi-cancer detection (MCD) tests. The panel addressed the design of these tests, the consideration of the clinical studies, and key study outcomes relevant to evaluating their probable benefits and risks. The The committee’s discussion and recommendations are set to guide the FDA's future regulatory decision-making for these innovative tests. https://www.fda.gov/advisory-committees/advisory-committee-calendar/november-29-2023-molecular-and-clinical-genetics-panel-medical-devices-advisory-committee-meeting

?? Here is the summary of the one day long discussion:

Topic I: Clinical Study Design

• The Panel recommends randomized controlled studies for MCD test validation, comparing them against existing cancer screening methods.
• Opinions vary on primary endpoints for clinical trials; some suggest cancer-specific mortality.
• Stage detection and disease state at detection are critical for supporting early detection claims.
• Assay performance evaluation is crucial, emphasizing positive predictive value, sensitivity, specificity.- Trials should include diverse populations, with pre-specified follow-up diagnostic pathways and clear statistical plans.

Topic II: Use of Tissue of Origin (TOO)

• MCD tests should include tissue-of-origin component for targeted diagnostic follow-up, reducing risks like radiation exposure.
• Concerns about access to diagnostic tools and limitations of current imaging technologies were raised.- Prespecified, clear follow-up paths in trials are essential, considering health equity and rural access issues.
• Negative MCD test results should still follow standard cancer screenings; trials should consider cancer type differences.

Topic III: Benefit/Risk

• Benefits and risks of false positive MCD tests should be evaluated per cancer type.
• Education and counseling are key to manage expectations and risks of false results and overdiagnosis.
• High specificity (>99%) may be needed depending on cancer prevalence in the target population.
• Follow-up for positive results should be prompt and guided by TOO component, complemented with educational resources.

Additional Considerations:

Health equity in diagnostic timelines, interval testing based on trial data, and cautious use of real-world data/evidence, suggesting a patient registry for standardized data collection.

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