ABSTRACT
The National Institutes of Health defines surrogate endpoint as ‘a biomarker intended to substitute for a clinical endpoint’. The problem is that statistical and clinical burden of proof remains high, since surrogate endpoints are not always true indicators or nor do they capture the full treatment effect. A clinical trial can use either the real, objective endpoint or an approved surrogate endpoint in the study. In oncology trials, for example, tumor shrinkage can be shown to predict longer survival in clinical trials for drugs intended to treat some cancers. When the biomarker is a novel candidate for a clinical endpoint, a type C meeting is recommended between the sponsor and the Food and Drug Administration (FDA) in the preliminary stages of clinical development for appropriate strategy. The area of validating biomarkers as surrogate endpoints is an active area of research in the FDA.
