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.
