FDA Approves Alzheimer's Drug Despite Fierce Debate Over Whether It Works (nytimes.com) 90
The Food and Drug Administration on Monday approved the first new medication for Alzheimer's disease in nearly two decades, a contentious decision, made despite opposition from the agency's independent advisory committee and some Alzheimer's experts who said there was not enough evidence that the drug can help patients. From a report: The drug, aducanumab, which will go by the brand name Aduhelm, is a monthly intravenous infusion intended to slow cognitive decline in people with mild memory and thinking problems. It is the first approved treatment to attack the disease process of Alzheimer's instead of just addressing dementia symptoms. Recognizing that clinical trials of the drug had provided incomplete evidence to demonstrate effectiveness, the F.D.A. granted approval on the condition that the manufacturer, Biogen, conduct a new clinical trial. During the several years it could take for that trial to be concluded, the drug will be available to patients, the agency said. If the post-market study, called a Phase 4 trial, fails to show the drug is effective, the F.D.A. can -- but is not required to -- rescind its approval.
I'd take it, except... (Score:3, Funny)
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They shouldn't have stopped the test.
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Looks like the FDA screwed up. They stopped the test because it did not show effectiveness, but a analysis of the data after the test was stopped did claim effectiveness.
They shouldn't have stopped the test.
Would it have mattered? We can't let 'science' get in the way of Pharma companies making a tidy profit.
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lab rat.
i know of a very large one that wonders around maralago.
i think mister science would agree
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A new study should be designed and started anew, which appears to be what is happening here.
Right. But FDA is allowing them to start the new study to prove it's effective after the FDA approved the drug.
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Re:I'd take it, except... (Score:4, Insightful)
The real fight will be over whether insurance companies (especially Medicare) will pay for it. If they won't pay for it, then no one will take it (except for a few wealthy people).
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Re: I'd take it, except... (Score:2)
Vanishingly small numbers of Americans pay for their own prescriptions, particularly those who are experiencing symptoms of this disease.
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Please, explain to me how such complications regarding future drugs are at all relevant for a human to arbitrarily manipulate his/her own body: is that not a basic right? Literally the "optics" of a failing medicine now become the responsibility of someone fighting for their mind/life/health? Why is that their problem? The fact that our behavior affects others should have no bearing on whether it is legally permissible, rather whether those specific actions violated another's rights (e.g., direct physica
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Yes.
You can *always* find a subpopulation that "responds" to the drug.
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There is no ethical question if you believe in human agency: let those who wish the drug to take the drug, if they accept all the consequences. If people have a basic right to drink, smoke, and eat themselves to death, if people can voluntarily engage in arbitrary sex acts (some which may involve "harm"), if people can defend their very life from an attacker (especially when no other help is available), then who are we to stop them from taking a drug that they believe may help themselves. There is no inte
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Yes, they should have (and it was biogen that stopped the trials). They fudged up, then tried to change their protocol in the middle to fix it. The right thing to do was to stop, plan, and start another, properly designed trial.
Naturally, since you're a business, you throw some bullshit at the FDA to see if they'll bite anyway. I expect Biogen is pretty shocked it worked.
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>I can't remember the name
the price sounds familiar, though.
Maybe I should plug in my modem and see if altavista can tell me why that price of "56k" sounds so familiar . . .
For shame (Score:5, Insightful)
Demonstrating safety shouldn't be enough. We should be able to have confidence that FDA-approved treatments are more than expense placebos.
Then don't take it (Score:3)
If you would rather continue to experience metal decline while waiting for additional studies, then do so. I'd rather have the choice available. It's not like your insurance companies is lining up to write checks for a treatment that doesn't work or doctors are waiting around for some snake oil to fool their patients. If they are, then we have bigger problems than the FDA giving conditional approval of a product pending an additional study.
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If you would rather continue to experience metal fatigue while waiting for additional studies, then do so.
FTFY
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If you would rather continue to experience metal fatigue while waiting for additional studies ...
Yep, if I have to listen to one more Judas Priest album while the FDA fiddles its thumbs I'll go nuts.
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This is a great reason why the FDA shouldn't be in bed with insurance or medical drug funding; just science.
Re:For shame (Score:4, Interesting)
Demonstrating safety shouldn't be enough. We should be able to have confidence that FDA-approved treatments are more than expense placebos.
The BBC ran a story about this and they profiled a British surgeon in his late 60s who has been taking it for a while. He says it definitely helps him. Now if he forgets where he placed something he has a chance to actually remember it. Without it, he won't even remember that he lost the thing he misplaced. Sounds like a more real response than to a sugar pill to me. One of the things we are finding out with medicines is that some people respond better than others to certain drugs because of their DNA so I can certainly conceive of it being possible that person A will respond in a more positive way to the medicine than person B just because of genetic differences. There are cancer treatments that we know work well on people with certain genotypes and we're not refusing to use them on everybody just because they only help some people.
Re:For shame (Score:5, Interesting)
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The BBC ran a story about this and they profiled a British surgeon in his late 60s who has been taking it for a while. He says it definitely helps him. Now if he forgets where he placed something he has a chance to actually remember it. Without it, he won't even remember that he lost the thing he misplaced. Sounds like a more real response than to a sugar pill to me.
And reactions like yours to anecdotal evidence are exactly why the FDA needs to stand strong and keep to the science even when there is immense pressure for them to approve a drug. If they don't, it just becomes a stream of charlatans hoping to manipulate their data enough to get conditional approvals and the chance to sell their still unproven drug for a few years.
Yes. Also, it probably works and there are no opti (Score:2)
I agree with you, for the general principle, and that's how the FDA works.
In this case, there is a treatment that *probably* helps and there are not other options available. (Buy maybe it doesn't help).
Ideally, we'd want to put everything on pause and do more testing. We'd like to tell Alzheimer's "hold off a couple years on destroying these people's brains while we do do a different trial". But Alzheimer's won't cooperate - it's going to continue destroying brains for the next couple years.
So it's tricky -
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Maybe the rule in cases like that should be, "You can give it to patients for free until the science is done."
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Something like that might be reasonable.
We do want people getting important treatments; we don't want people getting ripped off.
I'm sure there is a lot more to know about it than you and I know, but the basic idea seems sound-ish.
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Science is never done. There a popular delusion that hitting a particular p-value or other statistical threshold is somehow magical, but it just quantifies a judgement call. Of course many are now aware of the non-repeatability scandal in science. We should stop centralizing value judgements in authorities like FDA, who have no real skin in the game, since their life isn't on the line, just their paychecks.
How about: don't buy the drug until the evidence is sufficient? You pay the price, you take the r
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TYPO: Just because some sleazy drug company offers a useless product doesn't mean they should *NOT* have a right to peddle it.
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Not allowing people to have a medicine that could help save their brain would be bad. Having them waste money on it would be bad if it turns out that it doesn't help significantly. I'm glad I'm not the one who has to make this decision.
The key is that this is a trade-off involving a value decision. Different people will make different calls on this trade-off, and we ought to recognize their right to do so by limiting the power of the FDA to mandate studies about effectiveness. The correct choice isn't a single uniform rule enforced by a centralized authority like FDA, but more of a probability distribution, so decentralization is more appropriate.
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> limiting the power of the FDA to mandate studies about effectiveness. The correct choice isn't a single uniform rule enforced by a centralized authority like FDA, but more of a probability distribution,
So you want to be injected with something that has *probably* been tested?
When you have deadly illness, you don't want the medicine that will save your life. You want to roll the dice and maybe you get the medicine that works, maybe you get motor oil (who knows, it might work, we haven't tested it), may
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The FDA has a very misunderstood purpose. They do not regulate the function of a drug. Rather, they regulate the marketing of a drug. Yes, they also look at safety, but understand that every time you see a drug commercial it's often 30 seconds of benefits and 1 minute+ of disclaimers, because in their marketing the drug company explains the side effects.
Bottom line here is you can't sell anyone anything if they don't know it exists, so the FDA has the authority
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Yes, the FDA does regulate the function of drugs. Before a drug can be sold at all, it has to go through clinical trials to prove it is safe and effective for the purpose the manufacturer claims it is suitable for. The FDA absolutely has the power to prevent it from being sold at all if it fails in clinical trials. They can and do monitor drugs after their initial approval and can rescind the approval if the drug has safety problems that are discovered after
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What? This is all bullshit.
The FDA has licensing authority for drugs. If you want to be able to sell it (NOT just market it), you need FDA approval. And the FDA absolutely has a mandate to, and does, base licensing decisions on efficacy.
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Why are we making the bar so here for legit pharam companies when herbal supplement vendors can make all kinds of claims about their plant products and have zero consequences for it?
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The greater freedom that herbal product companies have is partly historical; plants are assumed to be safe because they're been in use "forever", and often they are actually safe. The term is "GRAS", Generally Recognized As Safe". Sometimes a herb is found to be potentially toxic and the FDA will discourage its distribution, that's why it's more difficult to get comfrey than it used to be. Sometimes a herb is known toxic if overused, yet it stays on the market: black cohosh can make you feel quite bad at on
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No, we shouldn't. The FDA should not have the power of god over a person's attempts to help themselves. They should go back to a pure safety mandate, and perhaps ensure that all knowledge about particular drugs is readily available, so that people can make decisions appropriate to their circumstances, risk tolerance, and evaluations of uncertainty. Frankly, even that more limited mandate isn't strictly necessary, but even with the safety mandate it would be a massive step in the right direction... where
Thin line. (Score:3)
Does anything else work? That would be one of the deciding factor*, between something works, and something MIGHT work.
*Ethics would be the other.
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Does anything else work? That would be one of the deciding factor*, between something works, and something MIGHT work.
It costs from $30,000 to $50,000 per year - for something that might do something. That's $4000 per dose. People make irrational decisions when a loved one falls ill. You can't explain to someone who's mother is dying that it's not worth blowing all the family assets on something with no proof of effectiveness.
If it were up to me, I'd tell the maker that the price is capped at $100/month until they can definitively prove that it works.
Oh, here's one of dozens of non-paywalled articles [cbsnews.com] that came up when I
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I guarantee that drug would cost $40 if you lived in the EU. As it stands now it’s cheaper to fly round trip to Germany to buy insulin than pay out of pocket in the USA. Are you going to tell me diabetes is different in Germany?
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The drug would cost the same in the EU, just paid for differently. If I can fly to Germany, get free care, and then fly home, that's totally unfair to the German tax payer who is footing the bill. Very nice of them, of course. Thanks!
The typical Alzheimer's patient is pretty old. So in the U.S. that person would be on socialized medicine and wouldn't have to pay for this treatment. Instead the taxpayer would. Call me cold and heartless, but I don't want to have to pay for a $50,000 treatment that does
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The drug would cost the same in the EU, just paid for differently.
This is not true at all [commonwealthfund.org]. The EU countries with single-payer health care systems buy medications in bulk at negotiated rates. If the EU pharma buyers believe they need the drug at that price they'd pay it. They generally set a price they want a drug at and it's up to the manufacturer if they want to deal or not. In the US the manufacturers deal to various entities (hospitals, etc) independently and are able to keep much higher prices that way.
This also has the side-effect that some drugs are unavailable in
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The drug would cost the same in the EU, just paid for differently.
I can buy Eli Lilly Humalog for $30 from a UK online pharmacy. The same insulin that costs $300 a vial in the US.
https://pharmacyoutlet.co.uk/p... [pharmacyoutlet.co.uk]
https://www.bbc.co.uk/news/wor... [bbc.co.uk]
It isn't being subsidised by anybody. Lilly are still making a nice profit at $30 a sale. US drug companies charge what the market will bear.
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Have you seen all those ads for Canadian pharmacies? US pharma tourism is a thing, no need to fly to Germany. And no, if you're an American and you show up to a Canadian pharmacy the Canadian taxpayer isn't footing the bill. You're benefitting from the negotiating power of a nation, and the pharmacy is absolutely making money off you.
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Actually, you're benefiting from the marketing power of a Province, not a nation, though there is a movement to change that. It is the reason drugs are so expensive in Canada, 3rd highest in the world.
It does say something about the prices in America that you consider the 3rd most expensive place to be really cheap.
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You're probably not going to want to visit Quebec anytime soon....
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It costs from $30,000 to $50,000 per year - for something that might do something. That's $4000 per dose.
From Biogen CEO says $56,000 annually for Alzheimer’s drug is 'fair,' promises not to hike price for at least 4 years [cnbc.com]
Biogen CEO Michel Vounatsos told CNBC on Monday that the list price of $56,000 per year for the company's FDA-approved Alzheimer's disease drug aducanumab is "fair."
The price of the drug is a reflection of "two decades of no innovation" and will also allow Biogen to further invest in its pipeline of medicines for other diseases ...
From articles I've read, drug "development costs" tend to be based more on what the company could have earned by investing in the market, than the actual development costs and the resulting drug costs are based on that and monopoly pricing. Investors and shareholders want their ROI.
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The FDA is already supposed to include the availability of alternative therapies in their decisions about effectiveness. The problem in this case is that they're not actually looking at the desired outcome, reduction in symptoms of dementia. Instead, they're looking at a surrogate measure, reduction in amyloid plaques. That's already questionable, but it's especially bad because there's a lot of reason to think the plaques are a bad surrogate.
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Herbal supplements (Score:3, Funny)
No no, take a random herbal supplement instead, who knows if the plant it comes from has poisons. Plants do not have molecules or chemicals in them. All plants are natural as hemlock and poison ivy, so how can it be bad for you? Jesus will not make a plant that can hurt you. Plants want to be eaten and smoked. They are your friend, because you are important to the plant it is happy to be eaten by you.
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context (Score:4, Interesting)
The context is that the Alzheimer's research community is heavily invested in the theory that because the presence of beta amyloid brain plaques correlates with Alzheimer's disease that those cause Alzheimer's disease; To prevent their formation or remove those plaques is to cure Alzheimer's. So much invested that faith in that theory unoficially became a standard of research validity and anyone testing an alternative hypothesis pretty much could not get funding.
After decades of research and a gazillion dollars of funding that theory is looking completely wrong. Actually, it seems that brain cell death is caused by chronic inflammation and the formation of amyloid plaques is a repair mechanism that does not impair cognitive function.
So "incomplete evidence" is probably that aducanumab reduces beta amyloid plaques but does not improve cognition.
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This has nothing to do with that.
As has been pointed out already a couple of times in this thread, the FDA's role is not to certify a drug's efficacy, it's to certify a drug's safety.
Americans should be happy about that, should they not ? Aren't they always complaining about governement overreach, nanny state, the market will sort it out, etc ?
Well, there you have it: A governement body that does the bare minimum that should be expected of a civilized society: Make sure that their citzens aren't sold produc
Re:context (Score:5, Informative)
Re:context (Score:5, Informative)
And you're wrong about what the FDA's role is. It's to certify what drug companies say about the drug; they regulate the marketing of a drug. Even with safety, what they're regulating is the ton of disclaimers at the end of every single drug commercial; they require the drug companies to explain the side effects of a drug so users can make an informed decision.
The problem here is exactly the beta-amyloid theory. This is a beta-blocker; it stops the formation of the plaques. Biogen can now say in their marketing of a drug that it will stop the formulating of the plaques associated with Alzheimer's, and side effects of the drug are potential brain swelling in high doses for those genetically pre-dispositioned to Alzheimer's (this is the actual results of the trial).
The problem with this is the controversy over the beta-amyloid theory is that some people believe the beta-amyloid is a repair mechanism attempting to stop Alzheimer's degradation, and that brain swelling due to inflammation (which this drug has a side effect of) is actually a core root-cause of Alzheimer's. However the FDA approval allows Biogen to market the drug as reducing beta-amyloid associated with Alzheimer's, when others believe it's actual function may may make it worse, and at least may do nothing yet charge people $56,000/year for a real inconvenience (it's intravenous every month, not a pill) to give them a false hope. But, the drug does what Biogen will say it does, so they get to sell it and make money (their stock is up 60% already today).
To the FDA's credit, they are requiring Biogen to run a much more extensive clinical trial to determine if it actually reduces Alzheimer's symptoms, which are entirely subjective and difficult to do, but still it feels a little like human experimentation releasing this as it's difficult to market this drug at all when the science surrounding Alzheimer's is in the middle of a very complex technical battle.
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ZombieCatInABox:
... the FDA's role is not to certify a drug's efficacy, it's to certify a drug's safety
The FDA [fda.gov]:
The Center for Drug Evaluation and Research (CDER) performs an essential public health task by making sure that safe and effective drugs are available to improve the health of people in the United States.. as part of the U.S. Food and Drug Admininstration, CDER...
also ZombieCatInABox:
As has been pointed out already a couple of times in this thread...
Not an authoritative citation.
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You may have pointed it out before, but you're still bullshitting.
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The scientific and therapeutic landscape of Alzheimer's is littered with the data from countless failed trials and compounds, and a big part of the reason why has to do with a lack of understanding of AD pathophysiology. But it is not the only reason. In fact, there are other considerations in the context of Alzheimer's disease that are in some sense precursors to why we have not affirmatively established the disease mechanism.
Foremost is the fact that AD is not the only type of dementia, and that until t
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The FDA has shot down lots of unsuccessful amyloid treatments. So many that when something like this comes along that maybe, sorta, shows actual functional efficacy they're tempted to approve it.
"Incomplete evidence" is that the drug improves cognition (relative to placebo), but that evidence was only discovered on a post-analysis of one of the two trials after both trials were stopped early.
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I didn't assume anything of the kind. It's true though, most of the people on approval committees are either leading people in the research area, or non-researchers who still specialize in the field. They are intimately familiar both with past attempts and the present state of the art.
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I saw another documentary a while ago where I think they found the same plaques in the appendix; they theorized that it was the body's way of trying to keep parasites out of the organ.
I've also seen they're starting to use the term "type 3 diabetes" for Alzheimer's; as mentioned by another poster, the suspect is s
Genesis (Score:2)
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Thank SCIENCE (Score:4, Insightful)
Alzheimer's is hell. Worst possible disease. You slowly lose everything that makes you you. It usually takes a DECADE for you to die. All the while your family has to spend their time caring for you. While you personally end up making their lives a tiny hell.
Your body is unaffected, so you get into trouble. You wonder around, perhaps getting assaulted by an idiot Policeman.
Any treatment should be tried. Before it is proven. If it doesn't help, maybe it will at least kill you earlier, putting you and your family out of hell.
It's enough to make you an atheist.
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Pretty much all the neurodegenerative diseases including Parkinson's get virtually no research funding on the fundamental science level. Billions are being spent on stuff like "treatment management" and lifestyle-changes research. As a consequence of that, we've gotten good at prolonging the suffering.
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Pretty much all the neurodegenerative diseases including Parkinson's get virtually no research funding on the fundamental science level.
Actually, over 3.5B per year from the NIH alone. And Pharmas (Especially Eli Lilly) has been throwing billion after billion into the black hole that is Alzheimer's drug development.
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That $3.5 billion is being spent on useless research like adjusting dosage of existing ineffective drugs and seeing the effect. Or seeing whether lifestyle changes can have an effect (it really can't, it's stupid.) We should be spending it on understanding the molecular biology of alzheimer's. What's being spent on that? A fraction of that $3.5 billion.
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Ah! I have a miracle treatment for Alzheimer's! It only costs $50,000 a year. When can I expect your cheque?
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Don't listen to ceoyoyo, he's a charlatan! I have the only cure for Alzheimer's! And mine is clearly better, since it costs $75,000 a year! Plus it's all natural and homeopathetic!
This is why the FDA should absolutely NOT let unproven medications be sold. Charlatans will take advantage of people who are desperate for relief. Yes, Alzheimer's is horrible, for the person who has it and even more for the people who care about them. My grandmother and my mother-in-law both suffered from it, and neither
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Dude, mine taste like grape. GRAPE! You know it's good because it tastes like grape!
Fake medicine doesn't just steal money from vulnerable people, it often keeps them from getting effective treatment. Biogen's marketing department is going into high gear now. They've got nine years to sell all the snake oil they can until the FDA wants to see some evidence it works. They are absolutely going to push that stuff as hard as they can. And if an effective therapy shows up in the next decade? It will have to comp
Can't be too soon (Score:2)
This is great, particularly since Biden warned us in his speech on May 27th "You know, if we don’t do something about Alzheimer’s in America, every single, solitary hospital bed that exists in America—as the nurses can tell you—every single one will be occupied in the next 15 years with an Alzheimer’s patient—every one"
And so (Score:2)
aducanumab
Cute. That'a "bamunacuda" backwards.
Only in The United States of Avarice (Score:2)
Best use of $$$? (Score:2)
Very expensive drug of dubious use gets approved so we can all contribute to a $56,000 a year maintenance plan for some people. That price tag is many times higher than the poverty level. Don't mean to be heartless, but if you compare quality of life gains between providing one person with this drug versus lifting several people out of poverty, it seems a no brainer.