Alzheimer's researchers seek changes to FDA drug approval standards

Advocates and lead researchers of Alzheimer’s disease, published “Single Endpoint for New Drug Approvals for Alzheimer’s Disease,” urging the Food and Drug Administration (FDA) to clarify and modernize its approach for approving new treatments.

Currently, the FDA standards require a new drug to show benefits on both cognition and function endpoints. In the analysis, the authors who are members of ResearchersAgainstAlzheimer’s (RA2), recommend the FDA approve new medicines that demonstrate a proven benefit on at least one therapeutic endpoint.

"If the FDA were to state that meaningful efficacy on a single endpoint is sufficient for approval, we believe that it would impact prospective investments in this therapeutic area as well as clinical-trial design," wrote the authors in a statement. "We believe a clarified and modernized FDA approval standard for Alzheimer's disease would catalyze renewed investment in the discovery and development of new medical advances for Alzheimer's disease, particularly in early-stage companies and for venture investment."

In the analysis, the authors outline that Alzheimer’s disease biopharmaceutical research is behind that of other diseases even though diagnoses will have a sharp increase in future decades. The authors also note that in 2014 and 2015, there were only 135 ongoing interventional Alzheimer’s disease clinical trials, as opposed to nearly 5,000 similar trials for oncology drugs.

The authors argue that if a new drug has the ability to improve memory but has less positive impact on a patient’s daily functioning skills, the drug should still meet FDA standards because clinically meaningful improvements in cognition matter most to patients with Alzheimer’s independent of functional improvement and vice-versa.

"Irrespective of the degree of impact on secondary measures, the notion that the FDA would deny approval for a safe and well-tolerated drug candidate that achieves its primary endpoint of improving cognition in patients with Alzheimer's disease is almost unthinkable," the authors wrote.