Alzheimer's experts call for changes in FDA drug approval standards

Leading Alzheimer's disease researchers and a prominent patient advocate today published an analysis, "Single Endpoint for New Drug Approvals for Alzheimer's Disease," urging the Food and Drug Administration (FDA) to clarify and modernize its current approach for approving new treatments for Alzheimer's disease.

The analysis, authored by George Vradenburg, UsAgainstAlzheimer's Co-Founder and Chairman, and Drs. Howard Fillit, Dave Morgan, Marwan Sabbagh, Paul Aisen and Richard Mohs, recommends that the FDA approve new medicines that demonstrate a proven benefit on at least one therapeutic endpoint - either cognition or function. The current FDA standards require a new drug to show benefits on both proven endpoints, an unnecessarily challenging hurdle the authors say may be inhibiting investment in new Alzheimer's treatments.

The authors are members of ResearchersAgainstAlzheimer's (RA2), an UsAgainstAlzheimer's global network of more than 450 disease researchers.*

"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. "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."

The analysis points out that Alzheimer's disease biopharmaceutical research is lagging well behind that of other diseases, despite the fact that Alzheimer's diagnoses will triple in future decades and that there is no current means to prevent, treat or cure the disease. The analysis notes that in 2014-2015, there were only 135 ongoing interventional Alzheimer's disease clinical trials compared to nearly 5,000 similar trials for oncology drugs.

The authors wrote that a modernized FDA standard for Alzheimer's medications would reflect changes in the field and in treatment since the 1990s, and it would also align with draft guidance issued in 2013 for drug development for early Alzheimer's. Specifically for Alzheimer's, the authors argue, if a new drug improves memory but has less positive impact on a patient's daily functioning skills, the medication should still meet standards for FDA approval, because clinically meaningful improvements in cognition matter importantly to persons 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.

The authors emphasized that the new standard is essential at a time in which recent clinical trial failures on Alzheimer's drugs have adversely affected investment in disease research. The FDA has not approved a novel Alzheimer's treatment since 2003, and the 5.5 million Americans with Alzheimer's and their caregivers are desperate for innovation.

* While authors of the analysis are affiliated with ResearchersAgainstAlzheimer's, the viewpoints published in the analysis may not reflect or represent the opinions or positions of all RA2 members. UsAgainstAlzheimer's is an innovative, patient-centered non-profit demanding - and delivering - a solution to Alzheimer's. Driven by the suffering of millions of families, UsAgainstAlzheimer's presses for greater urgency from government, industry and the scientific community in the quest for an Alzheimer's cure - accomplishing this through its own patient-centered effective leadership, collaborative advocacy and strategic investments.

ResearchersAgainstAlzheimer's (RA2) is a global network of more than 450 Alzheimer's researchers in the United States and abroad established by UsAgainstAlzheimer's to advocate for greater research funding, policy reform and multi-sector collaboration in order to stop Alzheimer's disease. RA2 believes that an effective treatment for Alzheimer's disease is within reach if government, industry and citizens are willing to commit the resources and work together to disrupt business-as-usual to achieve the 2025 goal set by the United States and the G7.

"A cure for Alzheimer's: a fantasy, a wish, an impossible dream; the same words that were said to Galileo, Edison, Curie, Salk and whoever dreamed up the internet. Yesterday's dream is today's reality." - Trish Vradenburg, Co-Founder and Vice-Chair of UsAgainstAlzheimer's, who passed away in April 2017.

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