All Times EDT
Keywords: Surrogate Endpoints, Biomarker, Validation
The regulatory pathway allows the FDA to approve a drug on the basis of its effect on an earlier endpoint that is deemed “reasonably likely” to predict clinical benefit, to address unmet medical needs for patients with serious or life threatening disease. One or more confirmatory trials conducted with due diligence may then be required to confirm the drug’s clinical benefit. The FDA accepts validated surrogate endpoints as evidence of benefit, but clinical trials are needed to show that surrogate endpoints can be relied upon to predict, or correlate with clinical benefit. The candidate biomarker (or surrogate endpoint) should be quantifiable, reproducible, and precise to be used as a surrogate than as a diagnostic tool. Evaluating candidate biomarkers in the exploratory phases of drug development and investigating surrogate endpoints in confirmatory trials require the establishment of a statistical and inferential framework. In this presentation, we will discuss the challenges in the evaluation and validation of the surrogate endpoints to the true clinical outcome of interest with different statistical approaches, and how the validation process might be arranged within the regulatory and practical constraints of the drug development process. We will demonstrate examples of validation study in oncology trials and discuss the potential candidate biomarkers for the hematologic trials in future studies.