All Times EDT
Keywords: Surrogate endpoint, Hematologic product, Minimal residual disease
For regulatory purposes, a surrogate endpoint is a marker that is thought to predict clinical benefits but is not itself a measure of the clinical benefits. The US Food and Drug Administration (FDA) has a long history of reliance on surrogate endpoints to support drug approval. Surrogate endpoints can be used to support regulatory approval or accelerated approval, depending on the strength of evidence in support of the surrogate endpoint’s ability (Gormley 2017). 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 January 2020, FDA issued a guidance on the regulatory consideration for use of minimal residual disease (MRD) in development of drug and biological products for treatment. The guidance covered the use of MRD in all hematological malignancies. This presentation will overview the use of meta-analyses for validation MRD as a surrogate endpoint; in addition, statistical issues in the development of surrogate endpoints in hematologic products will be discussed. The statistical considerations and experience learned from this investigation may be applicable to surrogate endpoint research in other disease areas too.