The U.S. Food and Drug Administration (FDA) has released an updated draft guidance for the use of biomarkers aimed at sponsors and pharmaceutical companies developing drugs intended to treat early Alzheimer's disease (AD).
The draft guidance, issued by the FDA's Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research (CDER), is an update of a previous draft guidance that the FDA released in 2018 intended to guide sponsors "in the clinical development of drugs for the treatment of the stages of sporadic Alzheimer's disease (AD) that occur before the onset of overt dementia."
The draft guidance provides a way for the FDA's current thinking on diagnostic criteria and clinical staging to be used in the enrollment of clinical trials and the selection of endpoints for the stages of disease relevant to the clinical trial.
The revisions between the versions reflect the developments occurring in AD treatment since the original draft; the agency notes that "[e]volution of the scientific understanding of AD may also influence these considerations."
The updated guidance includes a new subsection on the use of surrogate endpoints as a basis for accelerated approval, giving the example of amyloid beta burden as a surrogate endpoint that is "reasonably likely to predict clinical benefit." The agency advises that sponsors considering the use of such surrogate endpoints consult with the FDA early in development and cautions that a surrogate endpoint determined to be appropriate with one product or trial population should not be assumed to be appropriate with others.
"FDA is committed to reducing the burden of Alzheimer's disease," Dr. Teresa Buracchio, director of CDER's Office of Neuroscience, said. "This guidance document provides the agency's current thinking about diagnostic criteria and clinical staging and the selection of appropriate outcome measures, including the use of surrogate endpoints, for the early stages of Alzheimer's Disease."
