Drug regulators want to let drugmakers test Alzheimer’s disease treatments on patients years before the disease shows outward signs, and could approve the therapies based on subtle biological signals rather than proof they alleviate symptoms.
The Food and Drug Administration proposal will open new paths for drugmakers after repeated failures from companies that include Pfizer, Eli Lilly and Merck.
It also poses a scientific challenge. Researchers don’t fully understand the biological progression of Alzheimer’s disease, leaving the industry without a clear finish line or target.
While the FDA acknowledges the lack of an agreed-upon target, it loosened the standard for drugmakers to move ahead. The proposal cuts a line from a 2013 policy calling for “widespread evidence-based agreement in the research community” about the right biological signal.
The new guidance “is a big deal to companies,” said Maria Carrillo, chief science officer for the Alzheimer’s Association. “It is a clear statement that the FDA understands that the science of Alzheimer’s has evolved.”
In the proposal, the FDA said it quickly could approve a drug for people who haven’t shown outward signs of Alzheimer’s, if the therapy affects a biological marker of the disease — similar to how lowering blood pressure reduces the risk of a heart attack.
Nothing like that currently exists for the disorder, which is the sixth-leading cause of death in the United States.