An ALS drug fails, again
science.org
science.org
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.https://www.congress.gov/bill/118th-congress/senate-bill/190...
Give patients access and do the right science. Make sure it is safe and allow people the right to try.
" 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 "
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.
anybody can sell sugar pills or any 'generally approved as safe' consumptive good, repackaged as something big pharma is hiding from you
I recommend you read it yourself and understand what it is saying. Not to make broad assumptions that “snake oil” will be allowed.
Here is a summary I wrote about this bill based on a condition I have that can benefit from it:
Noting that ketogenic diets are already proven to improve outcomes in other neurodegenerative diseases.
Phase II trials are more about safety than efficacy, so I'd say the system worked as designed.
It's not true that safety is fast and easy to check.
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.
[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.
https://secure2.convio.net/alsa/site/SPageNavigator/blog_090...
It's sad that the drug didn't work (and I had a cousin recently die of ALS), but it's important to let the FDA do their work.
If anything we live in a world where we need better provisions for these illnesses similar to https://www.congress.gov/bill/118th-congress/senate-bill/190...
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.
https://www.regulations.gov/docket/FDA-2023-E-2605
Interesting!
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
That said, I feel like there should absolutely be an almost automatic approval for stuff sold in multiple other countries for decades.
This is the flip side of that.
This was patients lobbying Congress to skip safeguards. The FDA did not want to do this. https://apnews.com/article/science-business-health-lobbying-...
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.
The FDA literally has no authority to set the price of anything. This comes up so often it's actually an FAQ on the FDA website. https://www.fda.gov/about-fda/center-drug-evaluation-and-res....
> 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 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.
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.
Still need some kind of process to weed out the grifts.
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.
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 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.
Quacks won’t be allowed in such regulated environments.
https://www.congress.gov/bill/118th-congress/senate-bill/190...
Presumably, however, pharmaceutical companies striving to maximize shareholder value at all costs are allowed which is probably much worse in the end.
This might be helpful to understand context too: https://youtu.be/ma0xH0EI7Us?si=Qhg5pz6-FgYC0j_o
You clearly didn’t explain what the bill is saying.
Here is a summary https://youtu.be/HgcRCKdf9qg?si=7F0saD9UDEMkgFn9
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.
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.
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.
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.
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.
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.
Or do manufacturers simply not think it is worth their while / liability if Medicare and other governments will not pay for it?
If a billionaire went to the employees of Amylyx Pharma, and asked them to make this medicine for them, would they refuse?
Neither of those entities have the power to approve a drug.
Keep in mind 90% of drugs fail clinical trials. The situation cannot be THAT bad.
I don’t see the purpose of complaining about it in a situation where the institutions are working as intended (and desired).
So: there's a purpose in complaining about it and hoping that regulator and regulation can be improved.
This kind of failure seems relatively benign.
You are overselling the effects of this drug harder than the manufacturer did.
According to the article, the evidence for this drug was never substantial. We know it doesn't work now, onto the next.
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?
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.
You saw the price? $158k per year list price, though it is implied people probably paid less.
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.
The manufacturer had agreed to conduct a phase III and remove it from the marketplace if it failed the next trial.
It has now failed that trial.
[0]: https://finance.yahoo.com/m/b15fb91a-1e90-3259-844e-cfdb2563...
What you don’t do is make lofty promises to vulnerable patients.