Just looking from the outside the Aduhelm approval just seems kinda insane. Approving a drug that failed its primary trials based on a biomarker that is under serious doubt just doesn't make any sense. And it's not like this is a harmless drug, it does have quite serious potential side effects.
> People bitch when the FDA says “no” and then turn around and bitch when the FDA says “yes”.
The FDA’s purpose is to vet medical claims, and if the data does not support the claim, then the FDA should not give it a stamp of approval. Whether or not nebulous people complain about approval or denial is irrelevant, only the reasons for approving or denying are relevant.
Many critiques I've seen were based the premise that FDA approval would force (public and private) insurers to cover Aduhelm. This decision by CMS indicates that such concerns may have been misguided: it's possible to have FDA accelerated approval without forcing taxpayers to cover the treatment cost.
To be clear, it's possible that the blowback around the FDA decision led to CMS's decision, so I'm not critiquing the critiques. Just keeping in mind that this seems like a pretty ideal outcome.
The way I understand it the claim that Biogen was making was that Aduhelm could reduce the amount of plaque in the brain. The thing that hasn’t been proven yet is the relationship between this plaque and Alzheimers.
The only reason people care about reducing the amount of plaque in the brain is the belief that that ameliorates Alzheimer's. This was of course the primary intended endpoint of Biogen's trials, but (like other plaque-reducing drugs) the fact that it hasn't ameliorated Alzheimer's has caused many to abandon that belief.
Right, but if the FDA's job is to verify the claims made by Biogen then it shouldn't be a surprise that it was approved. Have there been any studies that show that plaque-reducing drugs don't ameliorate Alzheimer's? or is there just no evidence to support it yet?
The FDA scientific advisory committee rejected Biogen's claims in a unanimous vote. (Note that Biogen actually claimed in its application that aduhelm ameliorates Alzheimer's). The history of the aduhelm approval process just has a stench about it; you can see earlier posts by this author on the topic (linked to in the original article) to see some of that come out.
> Have there been any studies that show that plaque-reducing drugs don't ameliorate Alzheimer's?
There are, I believe, about a dozen drugs that tried to reduce plaques that have all failed to ameliorate Alzheimer's. And several of them despite actually demonstrating reduction of plaques.
Approving a drug based on a theory that it might help is not a good approach. If you're asking for approval to sell a drug to the public you need more than a hope. Some degree of solid proof that there are better outcomes with it than without seems a bare minimum threshold and Biogen couldn't clear it.
I think there are a lot of people who think the FDA should not get in the way of business. this makes it very hard for them to do their job objectively.
"People bitch when the FDA says “no” and then turn around and bitch when the FDA says “yes”."
that's true for most preventative professions. You get either criticized if you spend money and nothing happens or you get criticized when something happens. People have trouble with risks that may or may not materialize.
Approving drugs based on wishful thinking isn't how the process is supposed to work, but that is what happened here. It is still pretty unclear whether the amyloid hypothesis is true and whether reducing amyloid would actually treat the cause of Alzheimers.
The trials for this drug failed, they were stopped because it didn't work. And then they picked some data out of that and decided it reduced amyloids, so we just approve it based on that even without a clear signals that patients actually benefit.
> Approving drugs based on wishful thinking isn't how the process is supposed to work…
This is not entirely correct. There is relatively recent FDA guidance addressing the balance of pre market and post market data collection.[1] I am aware the title of the guidance says devices, but note that the center for biologics evaluation and research (CBER) is on the guidance.
That being said, my opinion is that this decision by FDA is an abomination — the second extremely questionable approval on Woodcock’s watch — and never should have happened.
Do they, though? I mean, I don't live in the US, but when some regulatory body forbids the sale of something medicinal my tendency is to have a sigh of relief about being spared something dangerous - and that's based on the assumption that regulation is too lax.
Of course there are exceptions to this rule, e.g. how governments have mostly prevented the use of cannabis for medicinal purposes because of the plant's psychoactive effects, but still... who actually bitches about FDA disapprovals?
Pre-medicare people have the impression medicare is cheap. But I pay $615 a month because I make more than $91,000 a year, which triggers an income surcharge. My premiums increased $784 (whole year) since last year.
I assume official inflation figures will always be lower than the growth in my expenses. It is the only possibility due to demographic changes causing greater demand for labor relative to supply, and will allow the government to fulfill its debt obligations (at least nominally).
Basically an obfuscated tax, especially for higher earners.
Free-market folks could argue: what's the point of the FDA here? The organizations that bear the cost of the medicine have decided that there isn't enough evidence of benefit from Aduhelm and further clinical evidence is required.
Perhaps the FDA's involvement should stop at Phase 2? Allow the free market to decide whether the clinical trial and efficacy results are sufficiently positive. The current setup encourages Pharma companies to design trials for the purpose of gaming the FDA.
As I understand, Medicare (Part B) pays "market rate" for this drug and cannot act as an independent payer and negotiate, yet, they seem as the primary market? This market price would be set by the secondary, and much smaller, market of private payers.
They aren’t the primary market. Except for drugs used in hospitalizations and certain other settings, Medicare itself doesn’t cover drugs. Drug coverage is under Part D plans offered through private insurers, who do negotiate prices. The goal is to have a many-to-many market. When you have a one buyer to many seller market, you open yourselves up to monopsony, which can drive prices below the efficient level.
My point (a view shared by the article and many others) is that the FDA decided Aduhelm is safe but did a terrible job of deciding that it is effective. Safety is also not absolute. All medicines have adverse effects, safety is then relative to the benefits of the medicine.
Accelerated approvals are just that - accelerated and don't require the same standard of proof of efficacy. Just like Avastin for Breast Cancer - it got accelerated approval, turned out it didn't work, so approval was recinded.
The goal is to give patients access to potentially effective medications. They do this because people bitch when the FDA says "no".
“this is safe and doesn’t work” is batshit insane; aduhelm never should have been approved, but at least a fail-safe worked and it’ll never be widely used.
A sad thing is the FDA dragged its feet on approving the covid vaccine for all age groups, taking 14 months. And vaccine makers/CDC had hard data vaccination would cut the 10,000 weekly death toll by 85%.
Here they approve a costly drug with almost no track record. FOLLOW THE MONEY!
