Amid firestorm of criticism, FDA narrows use of $56,000 Alzheimer's drug:
Less than five weeks after granting a highly controversial approval for the Alzheimer's drug Aduhelm, the Food and Drug Administration has updated its recommendation for who should receive the drug. The update narrows the recommended patient pool from all those with Alzheimer's disease to only those with mild forms of the disease.
The FDA's initial sweeping recommendation was a highly contentious aspect of the drug's approval, because it wasn't backed by any data. Aduhelm's developer, Biogen, had only included people with mild disease in its clinical trials. The numerous critics of the approval raised immediate questions as to why the drug would be open to all.
[...] Critics quickly called the approval "disgraceful" and "dangerous." Three members of the agency's advisory panel resigned in protest. Watchdog group Public Citizen called for the ouster of three top FDA officials.
Adding fuel to the fiery criticism is Biogen's decision to price Aduhelm at $56,000 for a year's supply. One analysis estimated that if the country's 5.8 million Medicare-eligible adults with Alzheimer's began taking Aduhelm, it could cost the federal insurance program $334.5 billion a year. In 2019, Medicare spent a total $37 billion for all drugs in the same category as Aduhelm, which is a doctor-administered drug. And the eye-popping cost estimate does not include additional, pricy brain scans and safety monitoring that taking the drug would require. While Aduhelm's efficacy is uncertain, the drug's known side effects include dangerous brain swelling and bleeding.
The FDA's update to narrow the potential patient pool will bring down those cost estimates. But perhaps not by much. A recent study led by researchers at Boston University estimated that half of people living with Alzheimer's could be categorized as having mild disease. And, even if just 500,000 Medicare beneficiaries end up taking Aduhelm, it could still cost the federal government $29 billion a year.
Previously:
Three F.D.A. Advisers Resign Over Agency's Approval of Alzheimer's Drug
Member of FDA's Expert Panel Resigns Over Controversial Alzheimer's Therapy Approval
Related Stories
Member of FDA’s expert panel resigns over Alzheimer’s therapy approval:
Following the Food and Drug Administration's polarizing authorization of the Alzheimer's therapy Aduhelm on Monday, a member of an agency advisory committee that recommended against the drug's approval has resigned.
Neurologist Joel Perlmutter of Washington University in St. Louis, a member of the FDA's expert panel for nervous system therapies, told STAT in an email that he had quit the committee on Monday "due to this ruling by the FDA without further discussion with our advisory committee."
The advisory committee, which convened in November, couldn't have been more openly skeptical of the drug, also known as aducanumab. Ten of the 11 panelists found that there was not enough evidence to show it could slow cognitive decline. The 11th voted "uncertain."
(Score: 4, Insightful) by Anonymous Coward on Saturday July 10 2021, @01:30AM (28 children)
The problem isn't just that the drug is expensive, but that it doesn't work. It is based on the increasingly discredited amyloid theory of alzheimers. It's quite effective at removing amyloid plaques, and it has no impact on the actual disease.
Amyloid just isn't the problem. But hey, the drug companies spent money developing this drug, they're entitled to their hundreds of billions of dollars in taxpayer funded revenue.
(Score: 2, Touché) by Frosty Piss on Saturday July 10 2021, @02:15AM (12 children)
So says the Anonymous Coward and his/her/they extensive collection of references…
(Score: 3, Informative) by Anonymous Coward on Saturday July 10 2021, @02:29AM (2 children)
Apparently a lot of other folks agree with the GP AC, or the controversy about the approval wouldn't exist.
Your response to the GP AC was pretty low effort, and ignores the contents of the summary let alone the article that the AC was commenting on.
(Score: 3, Informative) by Anonymous Coward on Saturday July 10 2021, @06:14AM (1 child)
The controversy over approval is not because of the amyloid hypothesis. It is because drugs by the FDA are supposed to be approved for the treatment of disease. The main disease state of Alzheimer's Disease is cognitive decline. This drug didn't show consistent effect for the cognitive decline (which wouldn't be surprising even if the amyloid hypothesis was correct). Therefore, the FDA approved it based on only on the fact that it improved a biomarker of the disease. That approval based solely on the biomarker in the face of rather serious potential adverse events is what is controversial.
(Score: 2) by sjames on Saturday July 10 2021, @08:39PM
It's a little like approving concealing makeup for the treatment of skin cancer.
(Score: 2, Flamebait) by fakefuck39 on Saturday July 10 2021, @02:53AM (4 children)
This is common knowledge for anyone who would be interested in the subject, and would open up to read this article. What he says, has been on the news for the last 5 years. What's funny is that you, without a basic google, are trying to... make fun of the op?
op: 5+2=7
you: lol, according to whom, cnn?
sorry, let me correct that for your specific case:
you: lol, according to who, cnn?
(Score: -1, Flamebait) by Anonymous Coward on Saturday July 10 2021, @03:22AM (3 children)
No, they're well-suited to big pharma because they're an epigenetically morally-bankrupt people who are neurotic enough about their own bad genes to be obsessed with medicine, yet skeezy enough to use medicine to write their own meal-tickets -- because sickness will always exist and as a result will always be profitable. Well, slightly less profitable during the times when yet another society kicks them out and they have to do their skeezing elsewhere for a few more hundred years.
(Score: 0, Troll) by fakefuck39 on Saturday July 10 2021, @04:06AM
it's funny how you try to defend yourself after I called you a retard, by replying with complete gibberish. question: do you even know what year it is? do you know what city you're in right now? how much crack have you smoked, nigger?
(Score: 1) by amamandaa on Saturday July 10 2021, @08:20AM
Skeeyzy? Is that a metric skeez, or an Imperial skeez?
(Score: -1, Flamebait) by Anonymous Coward on Saturday July 10 2021, @09:12PM
Who is this "they" you're talking about in your random rant? Are you talking about gene-lottery loser fat american rednecks who can't multiply numbers with over one digit reak of BO? no nation is going to kick them out. they're going to slowly kill themselves off by making bad decisions. They're like the dodo bird - too dumb to take care of themselves, sexually attracted to fire, only able to get dumb fat women, due to being ugly and fat themselves - bring on the heart attacks and the pandemics.
(Score: 2) by coolgopher on Saturday July 10 2021, @07:02AM (1 child)
*their
(Score: 0) by Anonymous Coward on Saturday July 10 2021, @12:15PM
>> > his/her/they
>> *their
*his
(Score: 2) by Beryllium Sphere (r) on Sunday July 11 2021, @04:45AM
See the blog run by Derek Lowe, a pharmaceutical researcher. As I understand it, the drug is effective at making amyloid plaques go away, and the FDA said they could go ahead and sell it while gathering data about whether it actually made anyone better.
(Score: 2) by Beryllium Sphere (r) on Sunday July 11 2021, @08:04AM
If you would like references, there are multiple links from https://en.wikipedia.org/wiki/Aducanumab. [wikipedia.org]
More precise than "does not work" is "is not proven to work". Not all the results were negative, it turns out, although the reviewers reported "red flags" about some of the positive data.
(Score: -1, Offtopic) by Anonymous Coward on Saturday July 10 2021, @06:24AM (14 children)
If Amyloid Beta isn't the problem, then what is your explanation for genetic and early-onset Alzheimer's Disease given their strong etiological evidence of Aβ being the cause of the disease state at least indirectly?
(Score: 0) by Anonymous Coward on Saturday July 10 2021, @12:47PM (7 children)
What strong evidence?
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @04:30AM (6 children)
It is in the first few sections here. [nature.com]
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @06:21AM (5 children)
That paper says:
> Although the vast majority of patients develop clinical symptoms at age older than 65 years (late-onset AD), 2–10% of patients have an earlier onset of disease (early-onset AD)....Together, mutations in APP, PSEN1, and PSEN2 explain 5–10% of the occurrence of early-onset AD.
Even if you assume all those mutations only affect amyloid beta levels and nothing else, that would be 0.1% to 1% of cases
Does a correlation with > 1% pass as strong evidence now?
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @07:29AM (4 children)
The extremely high penetrance for certain mutations and EOAD (complete penetrance in some mutations), their mechanism of action, and the protective mutations shows that amyloid beta is, at worse, indirectly causative of AD in those cases. In addition, the other genes that affect amyloid beta in different ways also have high predictive power for AD and comprise the majority of its heritability.
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @07:47AM (3 children)
There is a very low correlation between amyloids and alzheimers, so your premise is wrong. But even if it was high that would still be wrong.
Amyloids are the lowest energy conformations peptides can form. If anything starts malfunctioning you get accumulation of amyloids, starting with whichever ones are most prone to do it in that tissue. Look up any disease + amyloid and 100% of the time you will see they either accumulate or no one checked.
It is the same as being too sick to take out the trash, accumulated trash could then also cause more problems but removing it does not address the root problem.
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @08:31AM (2 children)
They aren't correlating amyloids with Alzheimer's. Its a subtle distinction, no worries.
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @02:22PM (1 child)
Yes, they are correlating mutations that correlate with increased amyloid beta in 0.1-1% of cases.
It is a research programme that has never generated anything useful. An entire generation of researchers and funding has been wasted on misinterpreting this extremely tenuous correlation.
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @09:36PM
No, they are not. Genetic studies can be difficult when you don't understand population differentiation, conditional subpopulations from segmentation, and heterologous analysis of non-homogeneous populations. No worries.
(Score: 2) by RS3 on Saturday July 10 2021, @02:33PM (5 children)
Not expert at all here, but I often question if something is cause vs. effect. In this case, there's much thought (evidence, theory, etc.) that the amyloid beta is the byproduct of the actual disease mechanism.
(Score: 1, Informative) by Anonymous Coward on Sunday July 11 2021, @04:24AM (2 children)
I understand the perspective, but if you think that cause vs. effect is reversed, you still have to explain familial and early-onset Alzheimer's Disease even though some gene mutations that affect amyloid beta can predict early AD 100% of the time. Amyloid beta production obviously has at least an indirect causative effect in a subset of AD cases, which means any alternative must also explain that effect. That doesn't mean there aren't any, but it isn't as simple as just saying "the amyloid theory is wrong."
(Score: 2) by RS3 on Thursday July 15 2021, @12:47AM (1 child)
You've probably already seen this, which is linked in a more recent SN story about Alzheimer's: https://www.news-medical.net/news/20210628/Alzheimers-symptoms-could-be-due-to-decline-in-brain-protein-not-accumulation-of-amyloid-plaques.aspx [news-medical.net]
But you may still disagree. I'm just open-minded. Not expert, certainly not bio researcher. Just read enough to know what seems obvious: nobody knows everything about Alzheimer's, so keep an open mind. :)
(Score: 0) by Anonymous Coward on Thursday July 15 2021, @05:28AM
I did see that and actually came back in case you mentioned it. I haven't finished looking through that study and its approved comments/correspondance and crosschecking its data, but my first pass through it shows that it is the exact type of thing I was talking about. If you recall, my key points were "you still have to explain familial and early-onset Alzheimer's Disease," "some gene mutations [have complete penetrance with] AD," that any alternative has to explain how AB production has indirect causative effect for the subset of AD cases. And most importantly, "That doesn't mean there aren't any, but it isn't as simple as just saying 'the amyloid theory is wrong.'"
This explanation has the potential to do all of that. It explains the complete penetrance of some genes, the mechanisms of others, the triggering effect of other hypotheses, explains the causative effect of amyloid beta production, and even incorporates the amyloid theory itself in a minor role. This is exactly the kind of hypothesis what I was talking about that goes well beyond just saying the "amyloid theory is wrong." In fact, it says the amyloid processes and plaques are the cause some of the time indirectly, but the picture is more complicated.
(Score: 3, Insightful) by Beryllium Sphere (r) on Sunday July 11 2021, @08:11AM (1 child)
https://en.wikipedia.org/wiki/Alzheimer%27s_disease#Causes [wikipedia.org]
It's no substitute for an education or even for a deep Google dive, but it gives an idea of the uncertainty and the range of possibilities.
Boiled down to a sentence, whoever edited it last said "The cause for most Alzheimer's cases is still mostly unknown except for 1-2% of cases where deterministic genetic differences have been identified.[12]".
(Score: 0) by Anonymous Coward on Sunday July 11 2021, @08:38AM
That cited article is a pretty good overview of the various hypotheses and how they interplay.
(Score: -1, Troll) by Anonymous Coward on Saturday July 10 2021, @01:34AM (1 child)
JEWS run big pharma!
(Score: 0, Offtopic) by fakefuck39 on Saturday July 10 2021, @02:41AM
Correct. Because they have a statistically higher average IQ than your redneck ethnicity. In fact, looking at simple statistics, the difference between your ethnicity and the average jewish IQ, is the same as the difference between your iq and the statistically lower black iq. Compared to a jewish person, you are just a piss-covered crackhead nigger.
Who would have thought smarter people would be running cutting edge biotech. Well, not you. Because you're too dumb to understand that basic concept.
(Score: 0) by Anonymous Coward on Saturday July 10 2021, @02:59AM
Why, then I'd be delighted if it ended up only costing $3.345 billion! A real bargain.