FDA grants historic approval for first and only drug to slow progression of Alzheimer’s disease despite independent experts said unproven benefits

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https://www.statnews.com/2021/06/07...mers-drug-designed-to-slow-cognitive-decline/​

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FDA grants historic approval to Alzheimer’s drug designed to slow cognitive decline​

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By Adam Feuerstein and Damian Garde June 7, 2021

 

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The Food and Drug Administration on Monday approved the first new treatment for Alzheimer’s disease in nearly two decades, a landmark decision that has been eagerly awaited by millions of Americans diagnosed with the condition but that will be hotly contested by some in the scientific community who doubt the drug’s effectiveness.

While other drugs treat symptoms of Alzheimer’s, the new medicine, called Aduhelm, is the first to attack what some believe is an underlying cause of the disease and slow cognitive decline, albeit marginally. It does so by eliminating clumps of a toxic protein believed to destroy neurons and cause dementia. Aduhelm is not a cure for Alzheimer’s, and it doesn’t reverse the disease’s progression.

The drug — which just two years ago was declared a stunning failure — is now expected to generate billions of dollars in revenue for its maker, Biogen.

 

[ginfreely]

“This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer’s disease,” Biogen CEO Michel Vounatsos said in a statement. “We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come.”

But the approval of Aduhelm, also known by its scientific name aducanumab, is all but sure to become one of the most controversial and disputed decisions on a drug application in recent years. The FDA granted marketing clearance to the drug over the strong objections of a panel of independent experts it convened in November. Those advisers reviewed Biogen’s clinical data and concluded overwhelmingly that there was insufficient evidence that the treatment had significant benefits for patients. They argued the drug should not be approved, as have many outside experts. Others saw enough evidence of efficacy — and a desperate need for new treatments.

 

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Instead of judging Biogen’s treatment solely on its effects on cognition, the FDA granted a conditional approval based on Aduhelm’s ability to clear the toxic proteins, called beta-amyloid. In order to continue marketing the drug, Biogen will need to complete a large clinical trial to confirm that removing the plaque has cognitive benefits, the FDA said. If that study fails, the FDA has the authority to rescind its approval.

In approving the drug on a conditional basis, the agency departed from decades of regulatory precedent, setting a new bar for treatments with considerable potential but unproven benefits — a standard that could also be applied to other devastating diseases.

“I’m quite surprised. The most compelling argument for approval was the unmet need but that cannot, or should not, trump regulatory standards,” said Caleb Alexander, a Johns Hopkins epidemiologist who served on the FDA’s advisory panel and voted against the approval of Aduhelm. “It’s hard to find any scientist who thinks the data are persuasive. Unmet need is an important contextual factor but it’s not an evidentiary threshold.”

 

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Aduhelm’s development has sharply divided Alzheimer’s experts. Now that Aduhelm is approved, they face a new challenge: prescribing the drug without overselling its effects.

“For patients who qualify, I’m hopeful for them,” said Ronald Petersen, a neurologist who leads the Mayo Clinic Alzheimer’s Disease Research Center. “Now we have a potential treatment for them that may modify the underlying disease course. But the largest responsibility for us is to educate patients and physicians as to what this means. This is not going to be penicillin for Alzheimer’s disease.”

 

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To many patients and their caregivers, Aduhelm’s approval provides hope for a better life, despite the drug’s limitations. Alzheimer’s affects nearly 6 million people in the U.S. Current medicines for the disease have only short-term effects on symptoms and bring side effects that make them unusable for some patients.

Aduhelm offers the promise of treating the root cause of the disease.

“I know this medication isn’t going to save my life. I know it’s not a cure,” said Jeff Borghoff, who, at 57, has been living with the symptoms of Alzheimer’s for six years. Borghoff has been receiving Biogen’s drug on and off since 2018, first in a truncated Phase 3 trial and then in an extension study. He has seen improvements in his cognition and ability to focus, he said, which has been massively positive for his family.

“I just saw my middle daughter get married,” Borghoff said. “She’s talking about having kids now. I have two other kids talking about marriage. That’s what I want. I want more time to spend with my family so that I can create memories for myself and for them. Ultimately, I’ll lose those memories with this disease, but they won’t.”

 

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Biogen said the yearly cost for a maintenance dose of Aduhelm, based on an average patient’s weight, would be $56,000. That’s a list price, not the net price or the price paid by patients with insurance. The out-of-pocket cost for patients with insurance will vary depending on their coverage. Analysts had expect it to cost between $10,000 and $25,000 per year, which would have already placed it among the most expensive medicines marketed to primary care physicians. The company expects to start shipping the drug in about two weeks.

The treatment, administered intravenously once a month, is approved for all patients with Alzheimer’s disease. Clinical trials tested it only in patients with early-stage Alzheimer’s who have had a PET scan confirming the presence of amyloid in their brains.

 

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For decades, scientists who study Alzheimer’s have focused much of their research on amyloid. A succession of companies including Eli Lilly, Pfizer, Roche, and Merck have attempted to treat Alzheimer’s with various types of antibody drugs that work by targeting and eliminating beta-amyloid — all without success.

Biogen believed it could succeed where the others failed; it designed Aduhelm to be more potent and therefore better able to eliminate amyloid plaques than earlier drugs. The company also focused on administering Aduhelm to people with mild cognitive impairment or the earliest signs of Alzheimer’s, before brain cells were too damaged to stem the tide of the disease.

 

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FDA grants historic approval for first and only drug to slow progression of Alzheimer’s disease despite independent experts said unproven benefits AFTER BIG PHARMAS FAILED FOR DECADES TO TREAT ALZHEIMER’S​

 

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FDA grants historic approval for first and only drug to slow progression of Alzheimer’s disease despite independent experts said unproven benefits AFTER BIG PHARMAS FAILED FOR DECADES TO TREAT ALZHEIMER’S​

Everyday so many good news from Angmoh the best indeed.

 

[maxsanic]

The problem with such an approval given is that it will further accelerate the already unsustainable medical inflation in the US and add more financial devastation for normal middle class American citizens.

Currently, the US medical system is run almost exclusively by private sector hospitals whose revenue are heavily reliant on reimbursements by medical insurances.

It’s OK if this was some alternative therapy that is open solely for the rich who can afford to pay US$56,000 per year to give it a shot, but when it becomes a formally approved drug claimable by insurance, medical claims would escalate significantly impacting loss ratios and ultimately everyone ends up paying even higher medical premiums for something that has no evidence of working.

This whole unbridled “free market” medical system plus insurance is how the US healthcare came to the decrepit state it is today. While the very rich who can afford do get the best treatments the world has to offer, the vast majority of the middle and lower class are either paying through their noses for extremely exorbitant medical insurances and deductibles or worse still drop out of the system and have to give up treatment options.

Just as a rough comparison, in Singapore let’s say if you are a relatively healthy 40 year old adult without congenital conditions, you only pay around S$1.2-1.5k (Medisave + Ridershield) a year for a top end integrated as charged plan for private hospitals. The same plan in the US would cost approximately US$400 per month which works out to about S$6.5k a year. Most middle class PME Americans cannot afford that and usually go for lower end plans with lower limits, more restrictions and high deductibles. Even then they aren’t cheap ~US$200 – US$250 per month.

 

[syed putra]

Right wing capitalist will vouch the effectiveness of US medical industry and how well those who can afford are treated in the hospitals with the latest innovation and techniques. They lead the way to new ways of treating all kinds of diseases.

 

[Loofydralb]

As usual with big pharma. They get a drug that is mildly effective but with some side effects, they have many ways and methods to works around the safety protocols (mind you they have decades of experience), bribe FDA officials, ignore, silence or threaten critics and then roll out their money making product.
Ditto Covid vaccine.

 

[maxsanic]

Right wing capitalist will vouch the effectiveness of US medical industry and how well those who can afford are treated in the hospitals with the latest innovation and techniques. They lead the way to new ways of treating all kinds of diseases.

The capitalists are not wrong. If you are very rich, indeed it is likely you can get the best treatment in the world. The original idea amongst Conservatives/Libertarians/Right Wing is that once the rich pay enough to the medical companies, these medical companies will be able to reinvest their profits to pioneer novel treatments and R&D even more newer medicines thus brining down prices of existing medicines and healthcare.

The problem is greed and avarice afflicts everyone and Americans are no different. Very soon, pharmaceutical companies, hospitals, law firms and insurers in the game realize it is far more lucrative to make big margins off a minority of affluent people than waste time and effort in eking out minor profit margins from a bigger middle and lower income class group. In order to preserve the gravy train, these people spend truckloads funding lobby groups, politicians and think tanks to perpetuate the system.

That's why average Americans have been complaining about their broken healthcare system for decades, every politician has promised to change the system during election campaigns but nothing ever changes. I hope Singapore never comes to a stage where a household of 4 needs to spend $15-$20k a year to buy a private hospital IP.

 

[ginfreely]

The problem with such an approval given is that it will further accelerate the already unsustainable medical inflation in the US and add more financial devastation for normal middle class American citizens.

Currently, the US medical system is run almost exclusively by private sector hospitals whose revenue are heavily reliant on reimbursements by medical insurances.

It’s OK if this was some alternative therapy that is open solely for the rich who can afford to pay US$56,000 per year to give it a shot, but when it becomes a formally approved drug claimable by insurance, medical claims would escalate significantly impacting loss ratios and ultimately everyone ends up paying even higher medical premiums for something that has no evidence of working.

This whole unbridled “free market” medical system plus insurance is how the US healthcare came to the decrepit state it is today. While the very rich who can afford do get the best treatments the world has to offer, the vast majority of the middle and lower class are either paying through their noses for extremely exorbitant medical insurances and deductibles or worse still drop out of the system and have to give up treatment options.

Just as a rough comparison, in Singapore let’s say if you are a relatively healthy 40 year old adult without congenital conditions, you only pay around S$1.2-1.5k (Medisave + Ridershield) a year for a top end integrated as charged plan for private hospitals. The same plan in the US would cost approximately US$400 per month which works out to about S$6.5k a year. Most middle class PME Americans cannot afford that and usually go for lower end plans with lower limits, more restrictions and high deductibles. Even then they aren’t cheap ~US$200 – US$250 per month.

I bet Spore private shield plan will never allow such expensive treatment to be claimed yet my premiums have been increasing at ridiculous 20 or 30% these years and now even specialists cannot choose and insurers can unilaterally remove rider benefit to enforce co payment. WTF

 

[ginfreely]

As usual with big pharma. They get a drug that is mildly effective but with some side effects, they have many ways and methods to works around the safety protocols (mind you they have decades of experience), bribe FDA officials, ignore, silence or threaten critics and then roll out their money making product.
Ditto Covid vaccine.

According to below link, 35% of the Alzheimer’s drug trial patients got side effect brain swelling. Ok lah not like those cancer treatments 90 or 100% got side effect.

https://www.biopharmadive.com/news/aducanumab-biogen-alzheimers-drug-ctad-presentation/568583/

In both ENGAGE and EMERGE, around 35% of patients on high-dose aducanumab experienced a side effect known as ARIA-E, a type of brain swelling that can cause headache and nausea. Usually these symptoms are mild and the swelling goes down on its own.

Even so, brain swelling is a side effect likely to unnerve FDA officials, particularly for a drug that could be prescribed to millions of U.S. patients. Another type of ARIA was fairly common and consistent across the trials, with around 18% of high-dose patients experiencing a micro-hemmorhage because of it.