“This is a remarkable and, in some ways, groundbreaking approval by the FDA. The approval is not based on aducanumab’s effect on memory or function, which is controversial. Rather, the approval is based on aducanumab’s marked effect on lowering amyloid plaques.
“FDA is taking the position that lowering plaques might likely predict future clinical benefit. They are asking Biogen to do further studies to assess actual clinical benefit. This kind of ‘accelerated approval,’ while common with drugs for cancer, has not been granted previously for Alzheimer’s disease.”
Lon Schneider is a professor of psychiatry, neurology and gerontology at the Keck School of Medicine of USC and director of the USC California Alzheimer’s Disease Center. He is one of many site investigators who helped conduct one of the aducanumab trials.
Approval “paves the way for more breakthroughs”
“There is a growing belief that a successful Alzheimer’s treatment will require a combination of treatments that can address multiple targets. To start down that path, one drug must come first. FDA approval of aducanumab sends a signal to the marketplace that there is a compelling need among patients and the market will sort it out. Any drug that modifies the trajectory of the disease will also encourage patients to seek earlier diagnosis, further expanding the market and the rewards for future innovators. If future drugs — and there are some in the pipeline — work better, they will supplant the drugs that came before.
“Today’s decision marks a point of departure from a long stretch of failure and frustration to new days of hope. With its approval of aducanumab, the FDA is fueling the chances of even more breakthroughs.”
Dana Goldman is the Interim Dean at the USC Sol Price School of Public Policy and director of the USC Schaeffer Center for Health Policy & Economics. These remarks were excerpted from a piece in STAT co-written by Goldman and Darius Lakdawalla, Director of Research at the USC Schaeffer Center and the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the USC School of Pharmacy.
New approved therapy targets beta-amyloid
“This is the first FDA-approved therapy that actually targets what many scientists consider to be a primary causal factor in Alzheimer’s disease pathogenesis: abnormal accumulation of the protein beta-amyloid. If beta-amyloid levels can be reduced early in the disease course, it is believed that downstream events including cognitive decline can be mitigated.
“Although aducanumab and related interventions have been shown to reduce beta-amyloid, robust cognitive benefits are less certain, which has resulted in some controversy regarding the true efficacy of the drug. The hope is that initiation of aducanumab treatment very early in the disease course will prove beneficial.”
Christian Pike is a professor at the USC Leonard Davis School of Gerontology who studies Alzheimer’s disease with the goal of pursuing translational strategies for the prevention and treatment of the disease.
Alzheimer’s disease-modifying drugs could save Medicaid programs billions of dollars
“With the introduction of an Alzheimer’s disease-modifying drug, Medicaid programs would be in a position to reap significant savings from avoided or delayed nursing home admissions because of reduced dementia progression and dependency on care.