HQ Team
March 12, 2024: The FDA has issued revised draft guidance for early treatment of Alzheimer’s disease with drugmakers told to focus on medicines that are likely to predict a clinical benefit.
For patients in earlier stages, drug developers must consider the use of surrogate endpoints, according to an emailed statement from the Food and Drug Administration.
A surrogate, or substitute, endpoint can be a laboratory measurement, medical imaging, physical sign, or other assessment that is not itself an indication of clinical benefit but is reasonably likely to predict clinical benefit, it stated.
Once finalised, the guidance will provide recommendations to help drug companies develop medicines to treat patients with early stages of Alzheimer’s disease that occur before the onset of overt dementia.
Deliberations with FDA
“Drug developers considering the use of a surrogate endpoint as the primary measure of effect should discuss their plans with the FDA early in development.”
The agency strongly supported and encouraged continued research in understanding the role of surrogate endpoints and stresses their potential importance in the successful development of effective treatments for the earliest stages of Alzheimer’s disease, according to the statement.
“FDA is committed to reducing the burden of Alzheimer’s disease,” said Teresa Buracchio, MD, director of CDER’s Office of Neuroscience.
“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.”
6.5 million Americans
Alzheimer’s disease is an irreversible, progressive brain disorder affecting more than 6.5 million Americans.
While the specific causes of Alzheimer’s are not fully known, it is characterised by changes in the brain—including the formation of amyloid beta plaques and neurofibrillary, or tau, tangles—that result in loss of neurons and their connections.
Patients with early stages of Alzheimer’s disease may have minimal or no signs of cognitive and functional deficits, such as memory loss, that are typically associated with later stages of the disease.
Globally the US accounts for 42.6% of the pharmaceutical market followed by China, Japan, Germany and France.
