When the Food and Drug Administration approved a controversial new Alzheimer’s treatment on June 7, it was over widespread expert dissent—including from the FDA’s own advisory panel. The medication, Aduhelm, was criticized for being fast-tracked despite a lack of clinical evidence that it’s effective. Its entry into the market could have catastrophic financial consequences for millions of people, and even Medicare itself.
“It raises false hopes for millions of patients with Alzheimer’s and their families,” Dr. Michael A. Carome, director of the Health Research Group at Public Citizen, told Filter. “My mom had Alzheimer’s disease; she died from it. I understand how there’s a pressing need for treatments … but what [people] need is drugs that can work and are safe. And we don’t have that here.”
Carome said that Aduhelm has not been shown to provide clinically meaningful improvements to Alzheimer’s symptoms. Clinical trials testing the drug’s ability to slow decline in cognitive function—like memory and timing—had mixed results. The FDA and Biogen, the drug’s manufacturer, presented findings to the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee in November 2020. With the inconsistent data, the experts had overwhelmingly recommended not to approve the drug.
Three members of the advisory committee have resigned in protest. One of them, Dr. Aaron S. Kesselheim, wrote that the decision “was probably the worst drug approval decision in recent US history.”
The FDA is meant to approve drugs only after they’ve been thoroughly proven safe for the patient, as well as demonstrated some sort of efficacy.
“In this instance, it seems like some of that decision has been pushed to the physician and patient,” David Whitrap, vice president of communications and outreach at the Institute for Clinical and Economic Review (ICER), told Filter. “Alzheimer’s is an awful disease that is destructive for families. As families go through this tough process, to ask them to weigh clinical trial data to determine if it’s worth it for their loved one is a tough position to put them in.”
Some doctors have said they will not prescribe Aduhelm. The FDA approval required Biogen to conduct a further trial to prove it is effective—but only sometime within the next 10 years.
Experts worry that over the next decade, countless patients could be prescribed a drug of dubious efficacy and safety. Aduhelm’s presence on the market could also disincentivize clinical trials of other, potentially more effective Alzheimer’s treatments, as patients may prefer what they perceive to be an already-proven drug as opposed to participating in clinical trial for unproven ones.
“It’s damaged the credibility of the FDA as a science-based gatekeeper.”
Aduhelm is also extremely expensive. ICER conducted a pricing analysis and found that if the drug is somewhat effective, it should be priced between $2,500 and $8,300 for one year of treatment. If highly effective, between $11,000 and $23,000. Biogen has priced the drug at $54,000.
For marginalized and economically disadvantaged patients, that cost could be ruinous. An estimated 80 percent US Alzheimer’s patients are likely to be beneficiaries of Medicare. With a patient population of 6 million, the impacts on the national budget could be staggering—potentially billions of dollars per year to Medicare. For patients and families, treatment with Aduhelm requires costly ongoing tests like PET scans and MRIs, some of which aren’t covered by Medicare Part B.
The FDA has defended the drug’s fast-track approval by claiming it is a unique form of treatment.
“Currently available therapies only treat symptoms of the disease,”Patrizia Cavazzoni, MD, director of the FDA’s Center for Drug Evaluation and Research, said in a statement. “This treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s.”
However, other research already suggests that reduction in the underlying disease process—specifically, the presence of amyloid beta plaque in the brain—is not predictive of improvements in cognitive function.
Many experts and advocates, including Public Citizen, are questioning the relationship between the FDA and Biogen. Aduhelm’s FDA approval spiked Biogen shares by more than 40 percent. The FDA itself however, might suffer.
“This sets a precedent for lowering the standard for approving drugs for anything,” Carome said. “It’s a lasting impact for public health. It’s damaged the credibility of the FDA as a science-based gatekeeper.”
Advocates stress that for Alzheimer’s patients and their loved ones, the potential outcomes of moving so quickly on inadequate data can’t be overstated. “Think about what else that money could be going to,” Whitrap said. “If the family of someone with Alzheimer’s were given $56,000, would they put it toward this treatment, or toward a well-paid caregiver?”
Photograph by via Food and Drug Administration