“I’m completely taken by surprise that this was approved”: Aurora doctor surprised at FDA approval of the Alzheimer’s drug
WASHINGTON (AP) – Government health officials on Monday approved the first new drug for Alzheimer’s disease in nearly 20 years, ignoring warnings from independent advisors that the much-discussed treatment has not been shown to help slow the brain-damaging disease.
The Food and Drug Administration announced that it has approved the drug Biogen is developing for use in patients with Alzheimer’s disease.
It’s the only drug that U.S. regulators have said could likely treat the underlying condition rather than treat symptoms like anxiety and insomnia.
The decision, which could affect millions of elderly Americans and their families, is sure to create disagreement among doctors, medical researchers, and patient groups. It also has far-reaching implications for the standards used to evaluate experimental therapies, including those that only show incremental benefits.
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The new drug, which Biogen developed together with the Japanese Eisai Co., could not reverse the mental decline, but only slow it down in one study. The drug called Aduhelm is given as an infusion every four weeks.
The FDA’s top drug regulatory agency admitted in a statement that the drug was surrounded by “residual uncertainties,” but said Aduhelm’s ability to reduce harmful plaque clumps in the brain is “expected” to help slow dementia.
As part of the approval process, the FDA requires the drug manufacturer to conduct a follow-up study to confirm the benefit to patients. If the study doesn’t show effectiveness, the FDA could take the drug off the market, although it rarely does.
“I’m very excited about the new drug,” said Castle Rock’s Sam Van Why. “Me too!” said his wife, Donna, who was diagnosed with dementia, which is likely Alzheimer’s disease. The couple noticed that it was becoming more difficult for Donna to complete her sentences.
“She just couldn’t find the word she was looking for,” said Sam. “It was frustrating!” said Donna.
This new drug from Biogen is an infusion. It reduces plaque in the brain and aims to slow the progression of mental decline in patients with the early stages of the disease.
“I hope Donna will still qualify for it,” said Sam.
The Alzheimer’s Foundation of America hopes this drug will improve quality of life. But others offer a dose of caution.
“I am completely taken by surprise that this has been approved,” said Dr. Alfredo Rivera, the director of the Senior Unit at HealthONE Behavioral Health and Wellness Center in Aurora. The studies were divided on whether the drug was effective.
“I see Alzheimer’s every day,” he said. “I would like clinicians to have in-depth discussions with patients and their families to see the appropriateness of this intervention and lower people’s expectations.”
Biogen did not immediately disclose the price, although analysts have estimated the drug could cost between $ 30,000 and $ 50,000 for a year of treatment.
A preliminary analysis by one group found that the drug would have to cost $ 2,500 to $ 8,300 a year to have good value based on the “small overall health gains” suggested by company studies. The nonprofit Institute for Clinical and Economic Review added that “any price is too high” if the drug’s benefits are not confirmed in follow-up studies.
Almost 6 million people in the US and many more worldwide have Alzheimer’s, which gradually affects areas of the brain needed for memory, reasoning, communication, and basic daily tasks. In the terminal stages of the disease, those affected lose the ability to swallow. The global burden of the disease, the leading cause of dementia, is only expected to increase as millions more baby boomers reach their 60s and 70s.
Aducanumab (pronounced “add-yoo-CAN-yoo-mab”) helps clear an Alzheimer’s-related protein called amyloid beta from the brain. Other experimental drugs have done this before, but they do not affect patients’ ability to think, care for themselves, or live independently.
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For years, the pharmaceutical industry’s drug pipeline has been littered with failed Alzheimer’s treatments that cost billions in research. The FDA’s green light is likely to revive investments in similar therapies that have previously been on hold by drug companies.
The new drug is made from living cells that need to be infused into a doctor’s office or hospital.
Researchers don’t fully understand what causes Alzheimer’s, but there is broad consensus that the aducanumab attacked brain plaque is only one factor. Growing evidence suggests that family history, education, and chronic conditions like diabetes and heart disease may play a role.
“This is only part of the puzzle and I think all of these other options need to be explored and expanded,” said Dr. Ronald Petersen, a Mayo Clinic dementia specialist who has consulted Biogen and other drug manufacturers.
Patients taking aducanumab decreased their thinking skills by 22% more slowly than those taking sham treatment.
But that meant a difference of only 0.39 on an 18-point cognitive and functional ability score. And it’s unclear how such metrics translate into practical benefits like greater independence or the ability to remember important details.
The FDA’s review of the drug has become a focal point in longstanding debates about standards used to evaluate therapies for difficult-to-treat conditions. On the one hand, groups representing Alzheimer’s patients and their families say any new therapy – even one with little benefit – warrants approval. However, many experts warn that approving the drug could set a dangerous precedent that opens the door to treatments with questionable benefits.
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Despite a damning assessment by the FDA’s external panel of neurological experts, it was approved in November. The group voted “no” to a number of questions about whether reanalyzed data from a single study submitted by Biogen showed the drug was effective.
Cambridge, Massachusetts-Biogen halted two studies of the drug in 2019 after disappointing results suggested aducanumab would fail to meet its goal of slowing mental and functional decline in Alzheimer’s patients.
A few months later, the company reversed course, announcing that a new analysis of one of the studies showed the drug was effective at higher doses and that the FDA had recommended a review. Scientists at the company said the drug’s initial failure was due to some patients not receiving high enough doses to slow the disease.
But the changes to the dosage and the company’s retrospective analysis made the results difficult to interpret, which aroused the skepticism of many experts, including those on the FDA panel.
The FDA is under no obligation to follow the advice of its outside panelists and has previously ignored their input on similarly high-profile drug decisions.
In 2016, the agency gave the go-ahead for the first muscular dystrophy drug, despite the panel of experts agreeing that a study in 12 patients had shown no effectiveness.
In this case, an internal dispute between the agency’s scientists was finally brought about by the agency’s long-time drug director, Dr. Janet Woodcock, now serving as the deputy commissioner, decided. Their official decision concluded that the drug is “quite likely” to benefit some patients. Almost five years later, the drug’s benefits have still not been confirmed by an FDA-mandated follow-up study.
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About 600 US medical professionals participated in Biogen’s studies of the drug, and many more are expected to begin offering it. Many practical questions remain unanswered: How long do patients benefit? How do doctors determine when to stop taking the drug? Does the drug have any benefit in patients with advanced dementia?
With FDA approval, aducanumab is sure to be covered by virtually all insurers, including Medicare, the state plan for the elderly that covers more than 60 million people.
Both the FDA and Medicare are not allowed to consider costs when reviewing a new drug or treatment.
Even qualifying for the drug could be expensive. It has only been tested in people with mild dementia from Alzheimer’s or a less severe condition called mild cognitive impairment. Brain scans, which cost $ 5,000 or more, might be needed to verify a diagnosis. Insurers including Medicare do not cover the scans as their benefits are unclear, but that could change if the scans become a gateway to treatment. Additional scans are needed to monitor possible side effects, including brain swelling and bleeding.
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