Trial Design
Biomarker
A biomarker is a measurable biological characteristic (molecular, histologic, radiographic, or physiologic) used in clinical trials to indicate normal biology, disease state, treatment response, or prognosis.
The FDA-NIH BEST (Biomarkers, EndpointS, and other Tools) Resource defines seven biomarker categories: susceptibility/risk, diagnostic, monitoring, prognostic, predictive, pharmacodynamic/response, and safety. Each has a distinct evidentiary bar for qualification.
Predictive biomarkers (e.g., HER2 for trastuzumab, EGFR for osimertinib) are the foundation of precision medicine and define eligibility in basket and umbrella trials.
References
Related terms
Basket Trial
A basket trial is a clinical trial that tests one investigational therapy across multiple disease types (typically cancers) that share a common molecular biomarker or genetic alteration.
Umbrella Trial
An umbrella trial is a clinical trial that evaluates multiple targeted therapies in patients with a single disease, stratified by molecular or genetic subtype, under one master protocol.
Endpoint (Clinical Trial)
A clinical trial endpoint is a defined, measurable outcome that determines whether the intervention being studied has the intended effect; it is the variable on which the trial's success or failure is statistically evaluated.
Companion Diagnostic
A companion diagnostic is an in vitro device or imaging tool that provides information essential for the safe and effective use of a corresponding therapeutic product, typically by identifying patients eligible for treatment.
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