Differences in clinical trial and FDA approval datasets: implications for clinical and policy decision-making for noninvasive colorectal cancer screening tests.

Abstract

Objective: The comparative effectiveness of clinical services may be inferred from concurrent studies in the same population, but such data are scarce. The U.S. Food and Drug Administration (FDA) creates a Summary of Safety and Effectiveness Data (SSED) to standardize population and performance characteristics for approved drugs and medical devices with the same or similar indicated use. Based on FDA approval requirements, SSED may differ from published clinical trial results. Previous studies reported the clinical implications of regulatory-associated data standardization for FDA-approved drugs, but to our knowledge, this has not been examined in medical devices.

Methods: We evaluated the performance metrics of clinical trial data and FDA-approved datasets for noninvasive colorectal cancer screening tests including blood- and stool-based tests. Using a previously validated CRC screening microsimulation model in the US, we compared the lifetime impact of performance differences between the FDA and clinical trial data.

Results: We found notable source-specific differences in reported performances for blood- and stool-based tests that significantly impacted modeled outcomes for CRC screening benefits, burden, and efficiency.

Conclusion: These novel findings highlight the importance and impact of using standardized FDA data as the reference standard for comparative effectiveness studies, guideline recommendations, and other practice-informing activities.