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Activity Number:
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237
- Statistical Challenges in Evaluating Molecular Diagnostic Assays Geared Toward Companion Diagnostics Devices
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Type:
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Topic-Contributed
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Date/Time:
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Wednesday, August 11, 2021 : 10:00 AM to 11:50 AM
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Sponsor:
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Section on Medical Devices and Diagnostics
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Abstract #317351
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Title:
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Challenges in the Bridging Study to Validate Companion Diagnostics for Rare Predictive Biomarkers
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Author(s):
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Dandan Xu*
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Companies:
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US Food and Drug Administration
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Keywords:
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predictive biomarkers;
bridging study;
companion diagnostics;
rare biomarkers
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Abstract:
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A predictive biomarker is a biomarker that predicts the effect of a treatment. Companion diagnostic (CDx) which measures the biomarker provides information that is essential for the safe and effective use of a corresponding therapeutic product. When a clinical trial assay (CTA) other than CDx is used to enroll patients into a clinical trial, a bridging study is commonly used to validate CDx by retesting samples using CDx. The bridging study assesses the agreement between CTA and CDx and evaluates the clinical efficacy for biomarker-positive population defined by CDx. For rare biomarkers such as NTRK gene fusions, bridging studies could be challenging and sometimes infeasible. The limited number of biomarker-positive patients and missing data present challenges. The negative percent agreement between CTA and CDx is especially important for rare biomarkers in determining the feasibility and success of a bridging study and therefore introduces additional challenges. We illustrate the challenges in the study design and data analysis of a bridging study and provide some recommendations.
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Authors who are presenting talks have a * after their name.
