[HN Gopher] The Aducanumab Approval
___________________________________________________________________
The Aducanumab Approval
Author : hprotagonist
Score : 123 points
Date : 2021-06-08 12:51 UTC (10 hours ago)
(HTM) web link (blogs.sciencemag.org)
(TXT) w3m dump (blogs.sciencemag.org)
| recursivedoubts wrote:
| all in all, just another brick in the wall
|
| watching social trust be destroyed on this scale in real time is
| both amazing and deeply disorienting
| germinalphrase wrote:
| Attacking social trust is easy and profitable (across the
| political spectrum), but transitioning to a low trust society
| won't make _my_ life better.
| recursivedoubts wrote:
| in the short run
|
| in the long run, I suppose the feeling goes, we are all dead
|
| my children won't be though
| bionhoward wrote:
| Biogen's stock performance yesterday was disturbing given the
| efficacy questions... This market feels whack. Anyone interested
| in a potentially more effective Alzheimer's AND Parkinson's drug
| might be interested in Annovis Bio:
| https://www.biospace.com/article/annovis-bio-shows-alzheimer...
|
| Given better results, it's a sign the plaque theory is wrong...
| jcranmer wrote:
| Oh, Biogen's now guaranteed to make loads of money before the
| efficacy question comes back to bite them, because the FDA's
| approval greenlights them to sell it before the efficacy can be
| firmly resolved (i.e., they come out with their subsequent
| trial data which, if history is any indication, is pretty
| likely to say it doesn't work).
| sparsely wrote:
| An absolutely shocking decision, which will have immense negative
| consequences for healthcare and healthcare costs far into the
| future. A license to turn false hope into profit margins and
| commissions for snake oil salesmen.
|
| List price is $56,000 per year if you're curious.
| nceqs3 wrote:
| List price doesn't matter in US healthcare FYI.
| boreas wrote:
| The fact that pharma has managed to obfuscate true prices is
| its own indictment of US healthcare.
|
| (edit) made my comment a bit less confrontational in tone
| HPsquared wrote:
| So the drug has been shown to reduce these amyloid plaques, but
| the trials have not conclusively demonstrated any effect on
| patient outcomes. The author then claims it should be effectively
| banned for this reason. Why shouldn't someone be allowed to take
| it if they want to?
| vajrabum wrote:
| Most of the patients this could be prescribed to are on
| Medicare. At $56k per patient and 5.5 million patients that's
| $308Billion. Oh and the drug causes edema in the brain in 40%
| of patients who are APOE e4 carriers which will be
| overrepresented in the patient population. That edema lead to
| confusion and patient falls. The scientific advisory board
| voted 10:0 with one abstention against approval. I'd just as
| soon not pay for something that doesn't work and in some cases
| causes harm.
| shkkmo wrote:
| I don't understand how a drug gets approved if the science
| board votes unanimously against it... who chose to override
| that decision?
| nceqs3 wrote:
| The FDA.
| nceqs3 wrote:
| MEDICARE DOESN'T PAY LIST PRICES!
| shkkmo wrote:
| There should be a step between banning and approval.
|
| If you look at the history of hiv/aids drug approval it's
| pretty clear that there is significant harm that can be done by
| banning unapproved drugs.
|
| At the same time, removing the requirement to show efficacy to
| be approved is also quite obviously bad for the reasons
| outlined in the article.
|
| Why can't the company be allowed to provisionally sell the drug
| with the warning that the FDA has evaluated the drug as
| "unproven efficacy" until such a time as the follow up studies
| are completed?
| ceejayoz wrote:
| We have such a thing. https://www.fda.gov/news-events/public-
| health-focus/expanded...
| shkkmo wrote:
| My understanding is that while that helps in some cases,
| the restrictions on its applicability leave a lot of people
| high and dry.
| EamonnMR wrote:
| They could sell them as supplements but then they wouldn't be
| legally able to claim efficacy or be billed as medicine. They
| can't make nearly as much money without FDA approval.
| gumby wrote:
| > Why shouldn't someone be allowed to take it if they want to?
|
| That's the standard libertarian argument and we have an actual
| A/B experiment that shows what happens.
|
| People used to be able to market any old thing (such as "snake
| oil") to cure whatever they wanted to say. A regime was built
| up over time (culminating in 1963) that you had to demonstrate
| both safety and efficacy in order to sell a drug, and you could
| only describe the drug in ways you had demonstrated (which
| included manufacturing).
|
| In 1994 Orrin Hatch (he of copyright maximization and senator
| from a state with a lot of "natural" scammers") got a law
| passed to say that anything "natural" was exempt from any FDA
| rules and sellers could say whatever they want. And as a result
| unlocked a huge snake oil business that preys on people when
| they are feeling most vulnerable. Fortunately most of those
| things are harmless nonsense but sadly not all.
|
| If you decide there is no need to prove efficacy than nobody
| would spend time on it. And thus drug had its trials halted
| because nobody was being cured (not only do you not want to
| spend money on them, but it's unethical to expose people to
| side effects of a drug that doesn't help them.
|
| The FDA is saying that even though it's unethical to do in a
| trial it's OK to do in the market.
| PragmaticPulp wrote:
| > Why shouldn't someone be allowed to take it if they want to?
|
| It's not entirely an issue of freedom. It's also about whether
| or not taxpayers have to pay Medicare to provide it.
|
| By some estimates, 1 in 9 people over the age of 65 have some
| degree of Alzheimer's. The cost of the drug is estimated at
| $56K per year.
|
| If we, the taxpayers, are now on the hook to pay $56K/year for
| up to 1 in 9 elderly people in the country for a drug that _may
| not actually work_ and has some serious side effects, that 's
| not good at all. Given the data we have, there's a significant
| probability that we'd be paying $56K/year to make these
| patients worse on average given the risk of serious side
| effects of the drug with negligible appreciable upside. That's
| a major problem.
|
| It might be possible for Medicare to deny this drug due to lack
| of efficacy, but that sounds like a political nightmare.
| jcranmer wrote:
| There's a history of drugs that reduced amyloid plaques,
| perhaps with even better efficiency than aducanumab (I don't
| recall exact details) yet still failed to demonstrate any
| effect on patient outcomes.
|
| The commentary that the trials haven't "conclusively
| demonstrated any effect" is an understatement. Recall that one
| of the trials outright _failed_ the endpoint, and the attempt
| to reconcile that with the other trial smells _strongly_ of
| statistical hogwash. The advisory committee rejected it
| wholesale, with not a single vote in favor and only one vote
| not in disfavor (i.e., abstention).
| jasonlaramburu wrote:
| My understanding is that aducanumab has shown the greatest
| efficacy of any beta amyloid blocker (at reducing beta
| amyloid, with the least side effects. It definitely has side
| effects (brain swelling in 40%), but other Beta amyloid
| blockers have proven largely intolerable.
| DOTIMES wrote:
| Given the studies showing higher rates of chronic infections in
| Alzheimer's patients (like herpes viruses and periodontal
| disease), why aren't there more trials like this:
|
| https://clinicaltrials.gov/ct2/show/NCT03282916
|
| >Anti-viral therapy in Alzheimer's disease will investigate the
| efficacy of treating patients with mild Alzheimer's disease with
| the U.S.A marketed generic anti-viral drug Valtrex (valacyclovir,
| 500mg oral tablet). Valacyclovir, titrated to 4 grams per day,
| repurposed to treat Alzheimer's disease, will be compared to
| matching placebo in the treatment of 130 mild AD patients (65
| valacyclovir, 65 placebo) who test positive for herpes simplex
| virus-1 (HSV1) or herpes simplex virus-2 (HSV2). The study will
| be a randomized, double-blind, 18-month Phase II proof of concept
| trial.
|
| I understand beta-amyloid is still the most likely cause of the
| disease, but given how lackluster the results of targeting beta-
| amyloid have been, why can't alternative hypotheses be
| investigated more fully?
| raverbashing wrote:
| This, and several other discussions remind me of this post in SSC
| https://slatestarcodex.com/2014/06/15/fish-now-by-prescripti...
|
| (Though to be fair fish oil apparently has more evidence for its
| efficacy)
|
| Also the same for several purported treatments for Covid.
| Efficacy is less important than cost and potential profits.
| mchusma wrote:
| There is no reason that "banning a drug" for everyone and
| "forcing medicare to pay for things" should be 1 thing.
|
| Should this be banned? Absolutely not. Should medicare be forced
| to pay for it? Also no.
|
| COVID vaccines were the same way. Should they have been banned
| early on? Absolutely not.
| tootie wrote:
| Good chuckle, but he's missing another tack to this. Saying it's
| purely based on safety and not efficacy makes it equivalent to
| the dietary supplement market is missing a key point. And that's
| the potential upside. Taking a moonshot on curing Alzheimers is
| far more worthy than taking a moonshot on curing varicose veins.
| As the Boomers start to hit their twilight years, we're going to
| have an absolute epidemic of Alzheimers patients and exactly zero
| chance of treating any of them with anything but palliatives. If
| Aducanumab has a 3% chance of success, then by all means we have
| to try. And if Biogen gets rich off of a flop, at least it will
| encourage more pharmas to get in on the action.
| mekkkkkk wrote:
| There's a huge market already saturated with moonshots like
| this. It's called alternative medicine.
| jcranmer wrote:
| But the pharmaceutical companies _have_ tried several times to
| get an Alzheimer 's drug that work. Being the first one to get
| an approved drug is a license to print money to the tunes of
| tens or hundreds of billions of dollars--enough to cover the
| costs of development. The reason why we haven't had any yet is
| because _they don 't work_.
|
| Why should Biogen's probably-doesn't-work Alzheimer's drug get
| a pass that the dozen or more attempts that showed similarly
| bad results didn't get? Note in particular that the assessments
| of everyone who looked at Biogen's statistical claims here is
| that those claims are not substantiated by statistics.
| jolux wrote:
| They have failed to prove efficacy in a trial. Why believe it
| has a 3% chance of working? Do you think arbitrary chemicals
| have a 3% chance of curing some disease, even when it has yet
| to be shown? There's absolutely no reason to believe this.
| tootie wrote:
| It's not a random drug. It's proven to reduce plaques. The
| phase 3 trials were inconclusive in proving that it improved
| patient outcomes which is certainly a bad sign but it's
| absolutely possible that future trials would show different
| results. I'm not saying the FDA is definitely right. I'm
| admittedly out of my depth on this topic. All I'm saying is
| this blog glossed over the severity of the problem they are
| trying to solve.
|
| Dementia is absolutely terrifying. My parents have already
| told me to euthanize them if it ever happens to them. I'd
| absolutely let them try this unproven therapy before doing
| that.
| wins32767 wrote:
| Do you think you should be taxed so that everyone's parents
| can try this unproven therapy for ~56k a person a year?
| [deleted]
| jcranmer wrote:
| Derek Lowe's blog has an entire category for Alzheimer's
| that talks about the sordid history of drug candidates: htt
| ps://blogs.sciencemag.org/pipeline/archives/category/alzh..
| .
|
| Of particular note is that he, like most practitioners, is
| of the opinion that the record of unmitigated failure of
| amyloid blockers is that it means that the theory that
| reducing amyloid plaques improves outcomes should now be
| considered discredited. See, e.g., https://blogs.sciencemag
| .org/pipeline/archives/2019/01/30/an... and https://blogs.s
| ciencemag.org/pipeline/archives/2018/06/12/an...
| vkou wrote:
| The goal of medicine is not reducing plaques, the goal is
| treating Alzheimer's.
|
| If the drug reduces plaques, but doesn't cure Alzheimer's,
| it's not a 'bad sign'. It simply means the drug doesn't
| work.
|
| P-value hacking over and over again[1] until we finally get
| a study that shows us that the drug works is not the
| solution. [2]
|
| [1] https://xkcd.com/882/
|
| [2] Unless the problem we are solving is "We have too much
| money that we need to spend on snake oil."
| tootie wrote:
| I get it, I'm just saying it's not like they're selling
| essential oils or healing crystals. Reducing plaques
| could at least plausibly have some benefit. And I'm not
| arguing necessarily that the FDA are therefore justified,
| I'm just saying it's not fair to compare this to diet
| supplements. I was pointed to some other posts by this
| same guy about how bearish he is on amyloid plaque
| reduction as a plausible therapy which would have been
| useful context.
| jolux wrote:
| > I get it, I'm just saying it's not like they're selling
| essential oils or healing crystals.
|
| You might have more of an argument here if this drug was
| only going to cost as much as essential oils or healing
| crystals, ironically. Would you suggest somebody go into
| debt for treatment with this product? It's likely to be
| quite expensive.
| jolux wrote:
| This is an epistemologically leaky argument. The goal of an
| Alzheimer's drug is not to reduce amyloid plaques, it's to
| treat Alzheimer's. There is, as of now, no evidence that it
| does so. Giving people false hope of treating a horrible
| disease does actually seem more unethical than not giving
| them the treatment at all.
| tunesmith wrote:
| Sorry, just a layman's medical question:
|
| If it's been shown to reduce plaque but not been shown to improve
| outcomes, is that positive evidence that it won't improve
| outcomes? Or is it more that noticing plaque reduction is fast
| while noticing outcome improvements would take a while? In other
| words, is this a case of "it's shown not to be efficacious, so
| wtf approval?" or is this more a case of "jury's out, so release
| it in the meantime"?
| ashwal wrote:
| Important context is that all interventions that have assumed
| amyloid to be causal for Alzheimer's have failed to show
| material clinical improvement.
|
| So the bar is really much higher than "did remove plaque" and
| the prior should be this wasn't going to improve clinical
| response. The fact that it didn't _and_ was still approved is a
| complete abdication of what the FDA preaches. It 's rare to see
| a uniform response from those that report on drug dev. but it
| has been unequivocal - the FDA needs to get its shit together.
|
| I, personally, have little hope for that.
| rossdavidh wrote:
| I am no expert, but my understanding (referenced obliquely in
| the article) is that this is not the first drug to target
| reducing the amyloid beta plaque, that does not show positive
| results. This is raising the question of whether it's treating
| a side-effect of the disease, rather than actually treating the
| disease. In other words, it's not all that surprising anymore
| that a drug targeting the plaque, does not work well.
| jcranmer wrote:
| > Or is it more that noticing plaque reduction is fast while
| noticing outcome improvements would take a while?
|
| This statement is true, but the trial will have gone on long
| enough to note whether or not both plaque reduction and outcome
| improvements happen, because plaque reduction isn't considered
| sufficient evidence for outcome improvement, despite some
| attempts to force the FDA otherwise. (Although I guess the FDA
| has kind of caved in at this point, given that it's
| conditionally approving this on the basis of plaque reduction
| despite seeing no outcome improvement.)
| azinman2 wrote:
| Somewhat tangential question: there are all these new drugs with
| -mab and -ab suffixes. Why is that?
| xenophon wrote:
| The -mab suffix means it's a "monoclonal antibody," or a lab-
| made protein that specifically targets a certain antigen (for
| example, an antigen found on a cancer cells).
| folli wrote:
| See here:
| https://en.m.wikipedia.org/wiki/Nomenclature_of_monoclonal_a...
| azinman2 wrote:
| Wow that's really interesting. Good to see it's less random
| than these words seem to be! Thanks
| robbiep wrote:
| As per the other commenter, but also they have a 10 year
| development pipeline and 10 years ago some major rockstars
| started hitting the market (ipilumimab, traztuzumab ) which are
| anti cancer ones but many others also). These were essentially
| the second generation of these drugs, the first gen sort of
| came out in the late 90s/early 2000s (ie Bevacizumab,
| infliximab ) and proved so versatile that they've spawned whole
| industries.
|
| The particular advantage is that a drug company can generate an
| antibody, clonally produce it, and demonstrate efficacy but no
| one else can claim the same efficacy because any other drug
| company is going to have an antibody that binds slightly
| differently and so has a different efficacy. (Ie so a generic
| here is called a bio similar antibody).
|
| There have even been bio similar that have had the opposite
| effect to the desired one (by for example acting as a receptor
| agonist rather than an antagonist)
| Invictus0 wrote:
| Is there any way that this decision could be overturned, and is
| that at all likely?
| yborg wrote:
| Medicare could decide not to cover it, but that's unlikely. The
| story here is regulatory capture. The enormous and ever-growing
| profits of pharma give them equally enormous resources to throw
| at buying regulators and congressmen.
| Metacelsus wrote:
| And to think that the FDA still hasn't given full approval to
| COVID-19 vaccines based on "an abundance of caution". Where was
| that caution now?
| CaliforniaKarl wrote:
| I strongly recommend making Derek's "In The Pipeline" column a
| regular read, because he provides the information that answers
| your question.
|
| First, there are the number of people who would take the thing.
| Alzheimer's is terrible, but I think many more people would
| take a COVID-19 vaccine than have Alzheimer's. A drug that
| causes problems in 0.1% of people is treated differently when
| the population size is 29.8 million[0] people of advanced age
| vs. 7.8 billion people[1] of all ages.
|
| Second, the FDA has more than one advisory committee[2]. Which
| makes sense; I don't expect the world expert on CPAP and BiPAP
| machines to also be the world export on COVID-19 vaccines.
|
| [0]:
| https://en.wikipedia.org/wiki/Alzheimer's_disease#cite_note-...
|
| [1]: https://www.worldometers.info/world-population/
|
| [2]: https://www.fda.gov/advisory-committees
| adrianN wrote:
| It is unlikely that this drug will be given to a comparable
| number of people as the vaccines. That makes tail risks a lot
| less important.
| CarelessExpert wrote:
| If the FDA views its role as evaluating for harms only and not
| efficacy, then this isn't itself necessarily a contradiction.
|
| That doesn't change the fact that this approval is deeply
| disheartening and tells us the FDA is cool with any random
| product claims so long as they don't actively hurt people.
|
| But, hey, for all those folks who glory in the good ol' days
| when men were men and radium was a cure-all, the good news is
| at this rate we'll be back to snake oil and patent medicine in
| no time!
| ProjectArcturis wrote:
| The FDA just allowed Biogen to suck hundreds of billions out of
| Medicare to pay for a drug that probably doesn't work.
| BurningFrog wrote:
| The FDA only judges the medical evidence. That is complex
| enough.
|
| Adding pricing to it's concerns would make what is already a
| nightmare process even worse.
| hn_throwaway_99 wrote:
| > Adding pricing to its concerns would make what is already a
| nightmare process even worse.
|
| TBH, I believe that is a huge root cause problem with our
| health care system. We need to stop pretending that money is
| infinite, and measure the cost against the benefit (in this
| case, little or none) when deciding whether
| Medicare/insurance companies should pay for a treatment.
|
| Perhaps this shouldn't be the responsibility of the FDA, but
| SOMEWHERE there should exist a process that says, "OK, this
| drug increases lifespan on average by 1 week, but costs
| $10,000 a month in treatment, sorry we're not going to cover
| it."
| germinalphrase wrote:
| Only a few short years ago, the eminently sensible idea of
| offering end of life planning (making a will, recording end
| of life medical desires) was demonized/weaponized as "death
| panels".
|
| What you're saying seems sensible, but my cynicism...
| tptacek wrote:
| Not for nothing, but, despite all the airtime this issue
| gets, my understanding is that pharmaceuticals are a pretty
| small component of health care spending; like, if you
| zeroed it out, you'd get less than a grocery store
| discount.
| hn_throwaway_99 wrote:
| I disagree. See
| https://www.politifact.com/factchecks/2019/feb/22/amy-
| klobuc... (drug costs come between 15-17% of total
| spending). And given how you can divvy up spending
| (drugs, facilities, doctors, nurses, admin staff,
| overhead, etc.), one sixth of total spending seems like a
| very large amount.
|
| And, it's especially the case that for certain
| individuals (this page also made the top of HN today,
| https://www.goodrx.com/blog/most-expensive-drugs-period/)
| that drug costs are a gargantuan, total-lifetime-salary
| kind of expenditure.
| tomsto wrote:
| This is how it works in the U.K.
|
| The MHRA decides which drugs are safe and effective and for
| which diagnoses.
|
| Then NICE decides which of those drugs are available on the
| public health system (NHS) by evaluating PS/quality-
| adjusted life year. I don't see why Medicare should be any
| different.
|
| If it's not available on the public health system you can
| still pay for it yourself or via private insurance provided
| it's licensed by the MHRA.
| NationalPark wrote:
| Considering their own advisory committee advised against
| approving the drug because of the lack of evidence that it
| works, the FDA doesn't really judge the medical evidence very
| well!
| an_opabinia wrote:
| > I suppose only things that definitely cause harm, because
| otherwise why not just ask for the same deal that Biogen got,
| and go out and prove efficacy while you turn a profit?
|
| In a sense, this is how it's sort of always worked. The
| approval process is about protecting investors. If a drug is
| good it's usually pretty obvious. When it's not obvious,
| efficacy in a more abstract sense must account for cost, which
| is out of the FDA's jurisdiction.
|
| The real problem with its organization is that it is, as you're
| saying, Medicare's procurement agent. But it is not empowered
| to consider cost.
| PragmaticPulp wrote:
| If this was simply a matter of making the drug available for
| informed consumers willing and able to pay for it out of pocket,
| I wouldn't be as concerned.
|
| If we're going to have Medicare and private insurance companies
| pay $56K/year per patient for a drug that may not have positive
| effects that outweigh the risk of side effects, that's a major
| problem.
|
| Limiting access to experimental drugs is a difficult question. I
| know someone who has a different poorly-understood disease (not
| Alzheimer's, I'm being deliberately vague to avoid debates) who
| managed to get enrolled in a clinical trial for an experimental
| treatment. She responded fantastically well to the drug, as did a
| small number of other patients in the study. On average, however,
| the response to the drug was poor.
|
| She and several other responders have now found each other online
| and are trying multiple avenues to get back on the drug. They're
| getting desperate enough that they're pooling funds to order a
| custom synthesis from another country and have it analyzed for
| purity by a 3rd party lab. I'm terrified to think of the risks
| they're incurring, but they're so desperate to return to
| remission that they'd rather take the risks than continue to
| suffer. It must feel unthinkably unfair to be given a glimpse of
| remission, only to be forbidden to continue to buy the drug
| because it didn't work on a majority of patients.
|
| In their case, it's likely that the disease has multiple
| causative factors and the drug in question only treats one
| specific cause. Without stratifying trials by these yet to be
| determined different causes, it's difficult for trials to show
| efficacy on large populations. I wonder if Alzheimer's disease
| could be similar, in that certain subsets of patients respond to
| the drug but the average patient will not.
| adrianmonk wrote:
| > _If we 're going to have Medicare and private insurance
| companies pay $56K/year per patient for a drug that may not
| have positive effects that outweigh the risk of side effects,
| that's a major problem._
|
| Hmm, what if we added a new phase where it's approved but can
| only be sold at a low, regulated price? Then if/when it is
| proven effective, they can transition to charging more.
|
| Without getting into whether it's actually a good idea, in
| theory the high prices are meant to be a reward that makes it
| worthwhile to invest in drug development. Delaying the reward
| would still allow that incentive but keep it tied to actually
| creating drugs that work while still allowing patients earlier
| access.
| rolleiflex wrote:
| The risk with that is that it creates a 'perverse' incentive
| for the FDA to not fully approve drugs because the approved
| drugs might shoot up in price 1000x the moment they exit the
| 'purgatory approval', and granting the official approval
| might kill some people who can afford the purgatory price,
| but not the fully approved official one.
| pontifier wrote:
| So lock in the price for the ones that started taking it
| before it was fully approved.
| rolleiflex wrote:
| That then would create the 'perverse' incentive for
| poorer people who are dying of major diseases to sign up
| for any and all trials possible to lock in prices,
| regardless of relevance, and make things much, much worse
| for themselves due to misuse. Richer people would not do
| that since they can pay the full price without locking
| the old price in, so this risks converting the sick and
| poor people already battered by life into lab rats.
|
| If you instead don't enforce the requirement to actually
| take the drugs to lock in the price, then everyone signs
| up for everything and your drug companies will stop
| making any new drugs.
| mkehrt wrote:
| Mean drug effect is possibly a bad metric to measure. If the
| distribution is bimodal, or has very high variance, the FDA
| could take that into account when approving a drug.
|
| I believe SSRIs often vary in effectiveness, for example; some
| people are just very good responders. Fortunately, their mean
| effectiveness is high enough.
|
| (Not a doctor, etc)
| nceqs3 wrote:
| Medicare would pay no where close to 56k/year. There are
| mandated rebates and price controls in medicare.
| amercer777 wrote:
| Medicare would indeed pay close to that price. Medicaid would
| see a rebate but would still be on the hook for roughly 75%
| of the list price initially.
| adrianN wrote:
| Hopefully, eventually we'll be able to figure out _why_ a drug
| works for one person but not for the next so that we can get
| pinpointed approvals for just that subpopulation.
| sneak wrote:
| A simpler solution is to get rid of the approval process and
| let people operate their own bodies how they personally
| choose.
| majormajor wrote:
| So the solution to the problem of doctors and medicine
| makers not being able to figure out what's gonna be safe
| and effective for whom is to just tell the patient to try
| to figure that out on their own?
| sneak wrote:
| We know the top-down approach is ineffective. Is the
| other one worse?
| tptacek wrote:
| Yes. We don't have to wonder; we have a supplements
| industry to show us what happens.
| 35fbe7d3d5b9 wrote:
| The terms "patent medicine" and "snake oil" didn't just
| appear from the aether.
| dnautics wrote:
| Choosing != Figuring out
| jasonlaramburu wrote:
| I think the FDA was in a difficult spot. There have been no new
| Alzheimers drugs in decades and patients are desperate for
| treatments. There is reason to believe that beta amyloid
| blockers need to be given in the earliest stages of Alzheimers
| (when diagnosis is difficult) to have any impact on cognitive
| decline. The trials did not focus on early stage patients, so
| there may be positive impacts if it can be given early enough.
| hmahncke wrote:
| The trial did focus on early stage patients - mild cognitive
| impairment (MCI) and early stage AD to be precise.
| compiler-guy wrote:
| Great! So let's figure out a way to make this study happen so
| that the drug can be given effectively (if it does indeed
| work) rather than have a blanket approval of a drug that
| lacks any proof of efficacy at all.
|
| That a drug hasn't been approved for a treatment in years is
| no reason to approve one that doesn't work.
| majormajor wrote:
| > So let's figure out a way to make this study happen so
| that the drug can be given effectively (if it does indeed
| work) rather than have a blanket approval of a drug that
| lacks any proof of efficacy at all.
|
| It isn't a blanket approval, but it does sound way too
| generous in terms. From the linked article:
|
| > The agency seems to have approved it based on its
| demonstrated ability to clear beta-amyloid, and is asking
| Biogen to run a confirmatory trial to show efficacy.
|
| > They will be absolutely overjoyed to do that, of course,
| because the whole time that's going, they will be selling
| the first drug that (in theory) targets the etiology of
| Alzheimer's. The backed-up demand is going to be gigantic,
| and Biogen is going to make enormous amounts of money. They
| have nine years, as it turns out, to get this trial done,
| and I feel safe in predicting that it's going to take
| alllll niiiiine loooong sloooow years to get this done.
| jasonlaramburu wrote:
| I guess the thinking is that it could take years to know
| whether it's working or not through an RCT. I believe they
| are currently allowed 9 years by FDA to show efficacy. If
| the side effects aren't significant why not let people in
| need try it?
| robbiep wrote:
| The side effects are significant. It comes with some risk
| of brain bleeds and a high risk of cerebral oedema in
| patients who have APOe4. This is an important genetic
| profile for those who understand Alzheimer's, as
| homozygotes for APOe4 are 12x more likely to develop
| Alzheimer's.
|
| This is a cop out decision by the FDA. Efficacy can be
| determined by a RCT - instead a decision was made which
| supports biogen's share price
| bsder wrote:
| > In their case, it's likely that the disease has multiple
| causative factors and the drug in question only treats one
| specific cause.
|
| The problem is that _every_ drug that has targeted amyloid has
| failed to do jack shit in _any_ cohort--not early, not late,
| not alien--none of them.
|
| Any _rational_ science system would conclude "The amyloid
| hypothesis for Alzheimer's is _WRONG_. "
|
| But, no, the bio people just plow on further because amyloid
| gets published, and funded, and, now, approved.
|
| One day we'll look at this time as the alchemy period of neuro.
| shkkmo wrote:
| I think we can improve access to experimental and unapproved
| drugs while still protecting consumers from malicious marketing
| of these drugs or sabotaging the current drug approval process.
| epmaybe wrote:
| How much more access can you possibly want on top of the
| previous FDA expanded access guidelines as well as the newer
| Right to Try Act?????
| phkahler wrote:
| >> If we're going to have Medicare and private insurance
| companies pay $56K/year per patient for a drug that may not
| have positive effects that outweigh the risk of side effects,
| that's a major problem.
|
| The usual story for why we allow patenting of drugs and high
| prices is that development is very costly - particularly the
| cost of doing trials. If they're going to allow this on the
| market without trials that show efficacy, the least they can do
| would be to allow generics at low prices while we're doing the
| efficacy testing on the general public ;-) </sarcasm>
| sparsely wrote:
| I had a quick look at what various special interest "non profits"
| had to say about this:
|
| Alzheimer's Association -> very positive, basically an advert
| (https://www.alz.org/get-involved-now/new-day). " A new type of
| Alzheimer's treatment, aducanumab addresses the disease in a way
| that has never been done before. This therapy slows progression
| of the disease, rather than only addressing symptoms."
|
| Alzheimer's Foundation of America -> much more measured
| (https://alzfdn.org/alzheimers-foundation-of-america-statemen...)
| "We are hopeful that it will improve the quality of life for
| individuals living with Alzheimer's disease and their caregivers.
| Patient access and affordability to all of those in need is of
| significant importance."
|
| Alzheimer's Association transparency page claims that they
| received under $300k from Biogen in 2020 and that <2% of their
| revenue is from biotech - I feel like they are either selling
| themselves a bit too cheaply or being a bit dishonest. Their
| whole website feels weirdly corporate as well.
| pcrh wrote:
| The article mentions the public pressure brought by these
| organisations. They may come to regret that pressure.
| deeviant wrote:
| Coming soon: insurance companies refusing to cover this because
| efficacy has not been demonstrated and stories of impassioned
| family members fighting against evil insurance companies in order
| to cover "the medicine their love one desperately needs".
| PragmaticPulp wrote:
| This drug is primarily targeted at elderly populations, who are
| eligible for Medicare.
|
| The private insurance companies won't be paying for this one.
| Taxpayers will.
| borski wrote:
| This is one of those comments I want to bookmark and come
| back to in a decade. :)
| zamalek wrote:
| > impassioned family members
|
| Imagine how hopeful these families feel right now. I really
| hope Derek turns out wrong, but I know it's unlikely that he
| will.
| HarryHirsch wrote:
| It already happened in Britain over cancer medication:
| https://www.bbc.com/news/health-39711137
|
| You'd predict that in a few years and after a few billions
| wasted regulators will wake up to the Phase IV results and
| withdraw approval.
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