This drug was approved after Phase II trials largely failed and a second review panel was convened to override the first. Now it has failed again in Phase III after being given to many hopeful patients at great cost.
The last lines rang true:
You need what you've always needed: efficacy and safety. Boring, tedious,
expensive efficacy and safety. Desire and despair don't figure in much - in a
much different universe, one whose physical laws respected and validated human
emotions and human suffering, they would. But we don't live in that world.
On an individual level that's rational, but on an institutional level the safeguards and caution are also rational. If you are able to make the drugs/experiments yourself, knock yourself out. But don't expect huge corporations/governments to do it for you
The safeguards and caution are even more immoral from a rationalist point of view.
By delaying potential medical breakthroughs you cause many future humans some amount of additional suffering because medical breakthroughs build on one another.
If you delay an ALS cure by one year, you are not only harming all those who have ALS for that year but all those present and future humans that have other diseases that could benefit from the future research tree branching out from that ALS cure.
Suppose for example that our ancestors were more risk-averse, more focused on the moral "do no harm" and thereby agreed upon 20 year trials for all new surgical procedures. What would 'modern' surgery look like?
We should be celebrating the risk-takers as willingness to take an experimental drug or an experimental treatment as it is often a greater gift to humanity than an organ donation.
Sometimes when you get a random stupid downvote, someone else will come along and fix it. I've been on both sides of that. But when you complain about it, that's pretty near a guarantee that I won't touch it. Because the point of downvotes is to suppress useless content, and complaining about how you're being "silenced" because someone downvoted you on a web forum definitely qualifies.
You're not entitled to be heard. A forum is a shared space, so it doesn't work unless we all have a vote on what happens here. If you somehow create the universe where people have "complete control", and you say annoying or even boring stuff, you won't get downvoted, you'll just get blackholed. Do you think that'll feel better?
Anyway, don't take downvotes so seriously. Some days the hive is just cranky.
More to the point, I was going to vouch the comment (which removes the flagged status) till I got to the edits. It’s not possible to vouch it in its current state, because at best I’d be making a mistake, and at worst I’d lose my vouch privileges. So you’re literally making it impossible to salvage your comment when you go off on rants about censorship.
There are thoughtful ways to comment about the moderation. If you take the time to phrase things in a substantive non-emotional way, and ask yourself "what am I trying to communicate?" you often end up with something that won’t be flagged. But the combination of user flags and manual mod review with the vouch system adding even more variability means that it’s a tossup whether any given decision will be reversed (or even can be reversed, since e.g. this one can’t be). It’s best to think of it like a casino and not worry on the days the cards don’t favor you.
Amusingly, the change to rank flagged comments above downvoted comments in an effort to push them to places less-seen has catapulted this subthread to the second top comment of the whole submission, which I haven’t seen before. HN never fails to surprise.
Your [2] is interesting in this regard. Mastodon is pretty much the modern tech ecosystem's answer to this whole scenario, and you already don't find that satisfactory. I don't disagree with your reasons, but: your objection is not really technical, but about what people do with delegated moderation authority.
You counter by saying each individual should completely control what they see, but here's the dirty truth: almost no one actually wants to do that. Even if they say they do, that's a lot of effort. Even you propose something similar with "Different teams can expose their own sets of labels". Most people are just going to block a bunch of labels with no further analysis, which puts you pretty close to where we are now. In practice, delegated moderation will be rampant, and it will definitely make mistakes.
So this is the detailed version of what I said in my first comment: if you get the world of protocols you say you want, and then make comments people don't like, then you won't get visible downvotes, you'll just be blocked. That really doesn't sound like it solves the problem you're raising. For my part I really don't think it's solvable at all, moderation is just going to be very difficult forever.
Interesting. It wasn't flagged when I commented. So it got flagged, vouched, and flagged again?
I hadn't heard of/seen that ordering change. Also interesting. Though I can't agree with your last line, HN is very predictable sometimes. For instance, any thread about dark matter is reliably a mess. :D
Bingo. It was necessary for the mods or the users to re-flag it, because it’s unsalvageable in its current form.
You likely won’t hear of such ordering changes. I spend a long time watching carefully to notice them. Sometimes it feels like I’m the only one who finds it fascinating.
HN is so predictable as to be deterministic in certain ways, but then the complexity of the social software ends up being delightfully surprising in many more.
Well, yes. How else would you deal with people who abuse the vouch system? And there’s no way to distinguish between someone egregiously abusing the vouch system vs someone whose idea of what should be vouched differs enormously from what the mods want to see.
Here’s how I’ve come to feel about it: the mods run the site. The site has to be run. It can’t be left to the community, because the community makes bad decisions, where "bad" means "drives intellectually curious people away from the site". Therefore, the way to get a lot of HN privileges is to create a lot of content that the mods want to see. The converse is also true; do things they don’t like, and you’ll swiftly find your privileges revoked.
In many ways, it’s a proof by contradiction: it proves that centralized moderation is a strong asset, which contradicts Reddit’s philosophy of "if you don’t like it, you can go elsewhere." There’s nowhere else to go. And — fortunately or unfortunately — that’s a feature which keeps this community unified, for almost two decades.
It's a good point; I have to say that, whatever other gripes I may sometimes have about it, HN is probably the most well moderated community I'm a part of.
> How else would you deal with people who abuse the vouch system?
So I think what I'm curious about is what constitutes "abuse" and how it's enforced. Like, are we saying that dang and friends have the ability to manually revoke someone's ability to vouch if they vouch for unhelpful comments on a frequent basis or if it's determined they're a sockpuppet account or something, or is there some more automated system that revokes a person's ability to vouch after some number of comments they've vouched are re-flagged?
I suppose I'd always imagined that the (fairly high, in the case of downvoting) karma threshold you have to reach to obtain privileges like vouching and downvoting would do a reasonably good job at filtering out those who might abuse such privileges, and that other remedies would be used rarely - and certainly not often enough to merit your earlier comment:
> It’s not possible to vouch it in its current state, because [...] at worst I’d lose my vouch privileges.
> are we saying that dang and friends have the ability to manually revoke someone's ability to vouch if they vouch for unhelpful comments on a frequent basis
Yes. This is an assumption on my part, but it’s a safe one, based on all the other things they can do. Having all your comments dragged to the bottom regardless of upvotes, for example, or preventing you from posting more than five comments every three hours.
The vouch system is a proxy for moderator decisions. If you’re not a good proxy, then one way or another, you won’t get to make the decisions. And I like being able to vouch comments, which is why I’m careful.
> and the sentiment of fear I read into it.
Oh yes. You don’t want to be their enemy. My full year ban was not a happy one.
I don’t think it was warranted. They did. Guess who had the final say?
You serve at their pleasure. We all do. And the best way forward is to embrace this fact and turn it to your advantage. They won’t ban you if you’re doing what the community wants. And what this community wants is to be entertained. It’s as simple as that.
You can pretty much figure out with >90% certainty what will or won’t attract moderator attention using this heuristic. If it’s entertaining to most thoughtful people, it’ll usually get a pass. Most thoughtful people don’t care to hear a rant about censorship on their favorite entertainment site, which is why this comment didn’t pass.
If any of this seems unlikely, feel free to email them and ask. Dan is nothing if not responsive. He’ll likely say that so-and-so portion of my comment is slightly wrong but that overall it’s correct in spirit, and he’ll explain the reasoning better than I have here. Or, just watch carefully which comments are flagged; in some ways it’s better to watch their actions than to read too far into what they say.
Moderation at this scale is uncharted territory. Dan’s team is figuring it out as they go, and it’s evolved a lot over the last decade. The vouch system is a minor part of the overall picture, but it’s one I love.
There are other countries, ones where it's legal to do medical experimentation upon any consenting party. Unsurprisingly, they don't create significant amounts of novel drugs. Why? Because the US system is more profitable, and, similar to how writing proprietary software is the most common way to earn a living as a software engineer, people like money.
Similar to platforms that claim to "democratize" a given hobby while at the end of the day being proprietary SaaS, people can claim as good of motives as they want, but at the end of the day, they want to make enough money to sit on their heels. The government strongly incentivizes drugs that are economically worthless without it.
The US system ensures that everyone gets paid. Laissez-faire drug development wouldn't. As better in theory as it may be, just like free software, in practice, you'd probably get fewer returns than the controlled system, just because people aren't altruistic, and nobody cares about niche diseases that doesn't have direct motive.
Not once did I propose changing the economic model. Only that we should give consenting, perhaps eager, terminal patients more options in participating in research and access to promising new drugs. It's unfair that there's a decade of waiting when lives are so quickly and pointlessly lost.
You're alive as a conscious participant in the universe just once, and then there's an infinity of nothing. You should have the option to try to keep that spark alive. If a chance, even fleeting, lies behind a glass door, that's cruel.
If you did that, there would be a rush of (not necessarily) terminal patients signing up and mostly dieing anyways (hopefully of their terminal illnesses)
There would be, as expected, a large contingent of patients who were poor, alone and maybe not very terminal. The very same reason which lead to the creation of ethics committes
Can you expand on this? This bill seems to enable exactly what the author is attacking: exploiting the desperate hopes of terminally ill patients to sell ineffective drugs.
I agree with removing red tape and allowing accelerated trials when the drugs are for these purposes, but it seems like today, drug companies can make a profit off of selling snake oil. They already stand to benefit from the reduced regulations, so why allow them to sell the drug at all under these circumstances? If they're so confident in the efficacy, they should be willing to give the drug away for free to benefit from an accelerated path to approval.
This bill seems to allow drug companies to continue to sell under these regulations, and in fact goes even further by removing most liability from them.
You could compromise by allowing a doctor to sign off if they feel the expected benefits outway the costs. That would put up a barrier for useless treatments but leave the door open for people to try stuff without 10 years of beaucracy.
In the case of the parent article drug, it claims it was the pressure of patient advocacy groups that got the drug approved. Giving people the right to try unfortunately at present means everyone else paying for it (or paying for the harm it causes), unless unapproved drugs were required to be covered by the individual on their own.
" But ALS patients (or their insurance companies) had the chance to pay for a drug ($158,000/yr list, who knows what the real price was somewhere below that) for what is in the end a combination of two generic medicines. And they might as well have been given peppermint candy for all the good it did.
...
patient advocacy groups took victory laps after Relyvrio was approved, and they were a big part of the pressure that made the FDA reverse its initial correct decision "
My Dad has ALS and has participated in a couple trials for different treatments. It's hard to say if any of them made a difference at all, and the justification behind some of these drugs seems extremely flimsy (one he was doing was for a drug that had some weak correlation during covid for slightly slower progression in one symptom), and the quality life impact of participating in that trial (having to fly to Boston, drug side effects, multiple lumbar punctures) leaves me frustrated that doctors recommend these at all. I feel like they're trading favors to get patients into studies with doctors they know, meanwhile, there are more promising ones that are being tested that he should be a candidate for, but the doctor won't even have a conversation about trying to get him in those trials. It leaves a bitter taste in my mouth. I hope something comes from all this, but right now I don't have a high view of the drug research and treatment process for this condition.
> The Phase III trial has just read out, and Relyvrio shows no hint of actually working.... It did have a very good safety profile, fortunately, so it seems unlikely that anyone was physically harmed.
Phase II trials are more about safety than efficacy, so I'd say the system worked as designed.
The problem is not the trial. The problem was an out of order early approval after only a phase two trial with very weak evidence. The phase three trial of course would go on even without such an out-of-order approval. The false hope such an approval creates doesn’t just affect patients directly. It may lead other companies to abandon or slow down therapies that might have competed in this indication. Approval of a drug is a huge deal. Perhaps eventually our drug approval system will change, but right now approval gives a company the power to market a drug to doctors, and doctors can prescribe it to patients, patients can ask for it, insurers cover it, and so on… This one was a conditional approval and the condition failed, which means the system still works, however it now has a possible loophole for how to bootstrap a pharma company. It is not clear yet if that loophole may increase human suffering on average, or not, and it likely depends on how the companies that take advantage of such a loophole grow. Drug discovery, design, and development are capital intensive operations and suffer from lack of innovation, but they also attract greedy people at the executive level who don’t bring therapeutic innovations to the table but rather find out how to draw investor money.
[T]he patient advocacy groups took victory laps after Relyvrio was approved, and they were a big part of the pressure that made the FDA reverse its initial correct decision.
Motivated reasoning, whether by commercial interests, patient advocates, or any other group, is inimical with truth-finding.
Though on the grounds of potential benefit vs. known harm (which is assessed in Phase I/II trials) and partial knowledge, a provisional approval in the case of a treatment for a chronic, progressive, and fatal condition is reasonably defensible. The FDA's conditional approval was based on Phase III trials showing efficacy. The dice were rolled, though the gamble in this case proved fruitless.
In this case, the FDA had approved the drug, though conditionally.
Second 'graph of TFA:
A second advisory committee meeting was convened, and if that phrase sounds odd to you, it should. That's a very unusual thing to do. This one voted for approval, and the FDA did approve the drug in September of 2022. There was a condition, though: Amylyx was already working on a Phase III trial, and they committed to withdrawing the drug if this trial showed no efficacy.
Why would you test something for efficacy before you're sure it's safe? You'll end up testing a bunch of harmful substances that don't actually do anything.
Maybe because the real standard is "the benefits out weigh the risks" you might want a drug that has a 10% chance of harm if it has a 90% chance of benefit, for a condition that is terminal if untreated.
Waiting for phased trials when you understand the research and hypothetical risks is torture. Especially when many drugs have proven safety profiles where the only harm is financial.
This particular drug was just a combination of two already-approved generic drugs. Anyone could have tried them with a doctor's Rx without a change in the law.
The FDA has been 'working' on authorizing sunscreen widely used in the rest of the developed world for the last 20 years, or since the original Dubya administration. This is not a typo. They are glacially, bureaucratically slow & cautious
Now it's just getting worse and worse. ALS patient communities used the questionable approval of Aduhelm by desperate Alzheimer’s patients to strongarm the FDA into approving this. Every junk pharma product is now going to come with an advocacy group that says "let's just try it and see".
Grifter pharma corporations will of course take advantage of this. They'll mobilize patient communities to keep skipping the science. And the result will be more wasted lives and more misallocated resources. Instead of working toward drugs that help people, we'll be working toward drugs that sound good to patient communities and Congress. This is a disaster.
The FDA might be slow. But it's slow and deliberate for a reason.
Imagining the perspective of someone with ALS I can see why they rightly would be advocating for a quicker process even with the risk. Most people diagnosed with ALS die within 1-5 years depending on how quickly it progresses. They don't have the benefit of waiting on the normal timeline for FDA approvals. The downsides of taking an experimental drug that harms them or just doesn't work is likely worth it for someone in that situation when they look at the potential upsides if it does work.
For quickly degenerative diseases maybe we need a different process that takes into account the context of what the drug is used for and what the acceptable risk is by the regulatory bodies. Making the standards and process clear will help provide the framework to reject those with unreasonable demands.
The other approach is simply accepting one's fate. No, there isn't some cure just around the corner with the only roadblock being FDA approval. There just isn't. There are cancer treatments with terrible side effects but they are approved because they have measurable efficacy. There are no ALS drugs because none of them work. Wishing hard doesn't change that.
The FDA is not that slow though. People misinterpret that it is actually the trials that are slow. Many things get approved quickly for as large of an enterprise as the FDA.
My hot take is that if you have a disease like ALS that's pretty much always fatal, you should be able to skip whatever safeguards the FDA has setup as long as you're informed that this medicine could hurt you or even kill you before the disease does.
Seconded, I think that's the most rational approach. I also think the patients should be thoroughly coached by medical professionals or maybe even be required to take a required orientation session on potential down-sides so that the risk is truly emphasized. We consume some pretty harmful things on a daily basis - alcohol and tobacco at the top of the list - and those are perfectly legal for people to decide whether or not they want to put into their bodies. It's not a huge leap to give people that same personal choice when it comes to experimental drugs that might give them even a small chance of surviving a terminal illness.
Its a nice sentiment, but it ends with desperate families spending all their savings on an ayahuasca-carrot juice cleanse to try and save their loved one.
Still need some kind of process to weed out the grifts.
Is it wrong for desperate patients to spend all their savings on ayahuasca-carrot juice? I don't think it's very ethical of the juice seller to exploit patients by selling them hope, but it doesn't feel like it should be illegal, since the patient does get some value out of the purchase, even if it's just (false) hope
Yes, it is unequivocally wrong for us to allow desperate people to get scammed. I almost can't believe I have to say that, but I've been on the internet long enough to know better.
What if for example they get conned into spending their life savings on fake treatments that exist today and then have nothing left when a promising legitimate treatment is developed six months from now?
And what about their heirs? What if they get conned into wasting away wealth that they could have passed down based on a false idea that it could help them be cured?
Not to mention that it hurts society as a whole to allow money to flow to grifters whether or not the subjects of the con successfully get false hope out of it.
It’s extremely hard for people to look past the current environment of, in general, knowing that whatever drug they can get their hands on (commercially) is going to at least be safe and probably quite effective.
But what's the harm here? The drug is safe. The only burden patients had was financial. Now what if the drug was effective? The harm it would incur to patients by delaying it would be much more extreme.
Even if it's safe, the harm is in rewarding drug companies for producing very expensive products that don't work, and selling them to desperate people and their families who will sell off everything they own and go as far into debt as they can to pay for them. This creates incentives that we don't or should not want.
Extending this further, the belief that a competitor has an effective drug gaining traction in the market while you are still at an unpromising point in research could reasonably lead companies to focus their research elsewhere. After all, your costs don’t go down for research if a competitor is already delivering, but your likely returns do drop.
The FDA could impose a ceiling over the price the company is allowed to charge if they are in a situation where there is reasonable expectation of efficacy but the trials haven't been consistent. Would that appease your concerns?
> We understand that high drug prices have a direct impact on patients—too many American patients are priced out of the medicines they need. However, the FDA has no legal authority to investigate or control the prices set by manufacturers, distributors and retailers.
The harm here is the medical industry sticking with the amyloid hypothesis in the face of repeated and expensive failures.
The funding needs to move on to something that might actually work. Propping up these failed drugs with "what does it hurt" is in the way of getting an Alzheimer's drug that might actually benefit people.
the harm is not all drugs are safe - even if this one was (and we aren't 100% sure of that either), and pharma companies using social media and/or sympathy stories to pressure regulators into approving a drug that would not otherwise get approved is gross.
It's a ton of wasted money. We should spend limited resources on treatments that actually work.
I feel tremendously for patients and their loved ones frustrated with the FDA's sluggishness, but results like these illustrate why it's needed. Especially since many of the drugs have much worse side effects than this one.
Said no patient ever. Everyone wants right to try, understandable that insurance would only cover sufficiently proven treatments. But if I can raise money, I want to at least take the most promising currently and if it doesn't work, oh well.
I think the important detail here is that, to anyone paying attention to the results of the clinical trials, this drug never showed promise in the first place.
The system we have is slow because it's thorough. Sometimes we have to move faster, and that's what EUAs are for. The worst versions of "right to try" policy would open the floodgates to all kinds of quack drugs with astronomical price tags. The resulting system would extract money from the desperate, and do very little to cure the ill.
Links are not comments. If you're unable to even try to summarize the content in a comment then you shouldn't expect other people to parse through it for a random online discussion. Either you care enough to actually participate in this discussion including making substantive comments or you don't. If you don't then don't get butt hurt at others because they call out your own laziness and refuse to make up for it.
Either you care enough to read my comment and link to comment back or you don’t. That’s called decency. Not my laziness to be quite honest with you. There’s now two comments and yet you still haven’t cared to respond about the original comment. Anyway here’s the last comment I’ll make.
This bill was written for ALS folks. It allows them to get access to safe drugs while clinical trials continue on efficacy. These provisioned drugs/illnesses like ALS, Cancer, etc provide dying people a chance to try things they would not be able to get, even through expanded access programs.
This has little to do with quacks entering the market or cynical capitalism. The drugs must demonstrate safety already and have some scientific evidence that they are on a “promising path” to efficacy while going through later phased trials.
We live in a day and age where much of our medicine is quite safe and goes through rigorous testing. But clinicians cannot get access to these drugs until they have been EUA or approved. This process takes many years to do and people suffering from these diseases have a right to try “unproven”, promising things.
ALS is a cruel disease. My mum is 83 and had great difficulty speaking and swallowing much of anything. Food was getting trapped in her throat and blocking her air way was happening more often. she battled for each breath. The riluzole did very little to help her. The medical team did even less. Her decline was rapid and devastating. The psychological support from the medical centre was non-existent and if it were not for the sensitive care and attention of our primary physician, there she would have died. There has been little if any progress in finding a cure or reliable treatment. Acupuncture eased her anxiety a bit. this year our primary physician started her on Natural Herbs Centre ALS/MND Ayurvedic treatment, 6 months into treatment she improved dramatically. It has been a complete turnaround with her speech, she no longer needs the feeding tube to feed, the treatment is a miracle. She recovered significantly! Visit Naturalherbscentre. com
I’m a little bit hesitant posting about my moms progress using Ayurvedic treatments but I’ll post in hope that someone finds this helpful despite the many herbal snake oil I see been advertise on this group. My mother who had been diagnosed with Lou Gehrig’s disease for 3 years at the age of 82 had all her symptoms reversed with Ayurveda medicine from natural herbs centre after undergoing their ALS/MND Ayurvedic protocol, she’s now able to comprehend what is seen and she no longer needs the feeding tube to feed,. God Bless all ALS disease Caregivers. Stay Strong, take small moments throughout the day to thank yourself, to love your self, and pray to whatever faith, star, spiritual force you believe in and ask for strength, their website is naturalherbscentre. com She’s getting active again since starting this treatment program...
> An uncharitable view would be that Amylyx ended up presenting the clinical package from the only angle that made it even possible to believe that the drug worked, and the FDA advisory committee ended up voting against approval... I hate to be this way, but I continue to believe that unmet medical need is not by itself enough reason to approve a drug. You need what you've always needed: efficacy and safety. Boring, tedious, expensive efficacy and safety.
I can either take from this that he believes the FDA and Amylyx were engaged in irresponsible behavior letting it get this far, or that (thank God!) an ineffective drug will not be approved, and we should all be glad there is a rigorous testing process that worked again. I get the sense that he believes the former, but can't ethically say so.
There are bigger things than a failed trial. There was a lot of money invested in this and when it was found to not be harmful there was a large push to show success. A lot of careers will be cut short because of this. There’s a lot of motivation to show something works. I was told to avoid research because of this kind of thing. There were a lot of highly paid people in a room that were not scientists but tasked with showing how much money could be made. That sounds evil but their intentions are good, that doesn’t help ALS people but there’s enough money put into this I wouldn’t be surprised if some progress is shown somewhere.
ALS or as Americans call it Lou Gehrig disease is a high-profile disease because of the ice bucket challenge despite the low incidence (6 out of 100,000) compared to other diseases like coronary disease or some cancers.
The disease is cruel, and it hits home for many of us as there are no mental deterioration like with dementia, Alzheimer's or FPD but you lose the ability to use your body and survival less than 3 years.
I can see why the FDA and congress are desperate to be seen to do something.
It's crazy how this drug got approved despite the evidence. A mixture of greed from the drug manufacturer and wishful thinking from the afflicted. Ugh. I've lost my trust in the system.
Agree, the Alzheimer's and ALS treatments that were approved recently were both cases where the patient advocacy groups stopped listening to experts and ironically harmed the people they are trying to help. It shows how important it is that the FDA keeps some emotional distance from the people they are trying to protect.
That's an interesting narrative, but are you sure that's actually what happened? It seems to elide the role of the drug manufacturer who pays for all these trials in the first place. Why would they pay for such doomed trials, just for the emotional hopes of patients? Are they that irresponsible with their stockholder's money? If so, they should probably be ousted and replaced with people who have more business savvy.
Or alternatively, this really didn't have anything to do with hopeful patients or their advocates, but rather the greed of the pharma companies, hoping to push through unproven drugs for the sake of profit.
I don't have any special insight into which narrative is true, but we've seen a lot of the second narrative historically.
They can't profit if it doesn't work. There is that bizarre case with the Alzheimer's drug, but that seems rare and not something they should pin their hopes on.
It sounds to me as if the drugmakers and the patient advocates are coming from the same place. They are desperate for it to work, and will convince themselves of it on any thin evidence. Probably something like "it did X in a petri dish so it must work eventually."
There are surely executives making a calculated risk, but I think they couldn't do it if there weren't also scientists who feed them over-optimistic guesses because they want it to work for the patients too.
The FDA won't usually approve a drug that doesn't work. Yeah, regulatory capture occasionally happens, and the FDA's definition of "work" is looser than you might hope. But it's just not a profitable strategy to keep throwing good money after bad on a drug that simply doesn't do anything.
And, curiously, the Alzheimer drug do work, just like hypothesized by the "dumb advocacy groups". You just have to take them earlier in the profession of the disease.
This would be true if research funding weren't a zero-sum game. Every dollar spent developing and marketing a drug that doesn't help is a dollar another research team needs badly but doesn't get.
I guess there’s the fact it made it to Phase III before they canned it meaning more people were hopeful than if action were taken to stop progress sooner.
But, how can progress be made if failure is disallowed because of unknowingly giving people false hope?
It made it to phase III: fine, there was enough evidence in the dubious phase II to perhaps justify a further trial.
But selling it to patients as an ALS therapy in the meantime outside of that trial, when it looked very dubious that it did anything-- that was a little questionable. The FDA overruling their own advisory committee to make this possible looks like a regulatory failure.
People take all sorts of stuff they hope will help but probably doesn't. Vitamins, crystals, cups of tea etc. I can't see how they make things much worse.
I don't think there were many better hopes out there for ALS. That's why they approved the drug despite weak data supporting it. This outcome is devastating for people with ALS and their families.
As someone with ALS, This drug was prescribed for me by my doctor as equivalent to other ALS medications, namely that they barely slow the progression of the disease. We are talking three to six months of life extension.
You are overselling the effects of this drug harder than the manufacturer did.
Drugs shouldn't be approved on hope. They should be approved on evidence. And there was never any evidence for this one. Just letting it inspire hope was hurting its victims.
You don't have to worry about that, because companies are the ones financing it and if they don't believe the drug have a good chance of working they won't fund it.
That's why quacks are so infuriating other than money they take (even in social medical systems) time is lost which means disease progression. For many diseases there is now cure but there may drugs to slow it down. The drugs may take a long time to work so years spent on fake crap may mean it's too late for a person to go on a drug.
While the current participants in the system might not be perfect, it should be appreciated that humanity has seldom had it better in terms of being able to trust civic institutions.
I don’t see the purpose of complaining about it in a situation where the institutions are working as intended (and desired).
Here, the institution has approved a couple Alzheimers drugs and an ALS drug that do nothing-- overcome by both things mentioned above that should not have the power to approve a drug -- "greed from the drug manufacturer and wishful thinking from the afflicted" -- indeed, the exact purpose of drug regulators is to safeguard is from those things.
So: there's a purpose in complaining about it and hoping that regulator and regulation can be improved.
What percent of FDA scientists and committee members who are in charge of approving drugs have been part of the “revolving door?” And how do we know this is inherently bad? It may be, but I can also see how niche expertise would cause you to float between entities as appropriate.
Keep in mind 90% of drugs fail clinical trials. The situation cannot be THAT bad.
Nonsense. I'ld rather have a safe drug with >50% of chance of working approved than waiting for 3 more years and find out when I am already debilitated. That some drugs turn out to not work is not a reason to "lose trust in the system", unless you are an immature child.
What would you rather have in this scenario? Earlier phases almost certainly started on animal models. How do you know it will or won’t work in humans if you don’t do clinical trials? Keep in mind 90% of drugs fail clinical trials, and that’s almost certainly after showing success in animal models.
The last lines rang true: