Office Director US FDA Silver Spring, Maryland, United States
Development of new therapies to treat neurodegenerative diseases is an important unmet medical need. Biomarkers can aid in assessing new therapies at various stages of drug development, both in the preclinical setting and in clinical trials. In clinical trials biomarkers can serve as safety biomarkers, prognostic biomarkers, pharmacodynamic biomarkers and in assessment of efficacy as surrogate endpoints for traditional approval or reasonably likely surrogate endpoint biomarkers for accelerated approval. Dr Siegel will discuss the regulatory framework at FDA for incorporation of biomarkers in drug development programs, including how the context of use for the biomarker determines the requirements for analytic and clinical validation. Examples relating to biomarkers of neurotoxicity will be discussed.
