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.
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.
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.
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.
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.
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?