Okay, but Biogen has 9 years to do the confirmatory study and they've indicated that they'll take their sweet, sweet, time with it. Also, it's not like we have 0 evidence: we have two, 4-5 year long studies that show how well it works: and the benefit in one was marginally positive and the effect of aduhelm was negative in the other.

Sarepta's Eteplirsen was in a similar situation: little data to back it up and a very controversial decision. They also had to do confirmatory studies, but it took them 3 years after the approval was done for the study to begin.

They did test this drug for years, the conclusion was that the drug does absolutely nothing for Alzheimer's. A big part of what FDA approval means is that it's been confirmed that the drug does what the makers of the drug claim it does. If that weren't the case, many drugs that have not been approved over past decades would have been.

> Which is reasonable to me. It takes years to test drugs like this and if a patient and her doctor believe this is the best treatment, the FDA shouldn't prevent them from getting access to it.

I can understand this argument as long as Biogen isn't making any money off of these drugs. In fact, they should really be losing money if anything since they are gaining value from the data.

Big Pharma bad, but making drugs is incredibly costly. Especially in Alzheimers, where so many candidates failed. It's incredible someone is even trying after so many failures.

That's true, but I don't really see why the drug companies shouldn't pay those costs.

The second order effects may be that they wouldn't want to even try to develop a drug.

What world do you want to live in? (Rhetorical)

The third order effects may be continued erosion of respect for the federal government's regulatory process. Tread carefully. (And make lots of money while you can.)

And blowing out the drug budget for medicare for a very expensive but futile treatment.

If you say they should pay the costs, they should also reap the rewards, so in that case you have to accept that drugs are really expensive.

The big question is will insurance companies and CMS pay for it, and that's not up to the FDA. That debate is occurring right now and will likely create new precedents.

This right here. People move cross country or even internationally for a chance to be in the 1 hospital who is running a trial for some non-proven drug for their disease

Being able to get the drug wherever you are in the U.S is a good step, and every patient/doctor can decide if they want to try it

> Being able to get the drug wherever you are in the U.S is a good step, and every patient/doctor can decide if they want to try it

FDA approval is for letting companies market a drug to the public, it has nothing to do with whether you as a patient are allowed to take it.

That's incorrect. Biogen can't legally sell you the drug unless it's been approved by the FDA or they use one of the specific exemptions like doing a drug trial.

That's incorrect.

Doctors can (and do) prescribe drugs for off label usages. There's nothing "illegal" about it.

The reason they don't do that more commonly is malpractice lawsuits and insurance companies don't like to cover off label prescriptions.

An excellent example of this: Medical Marijuana. Not FDA approved for almost anything and the only reason it's "illegal" is because of federal DEA classification.

So long as it's not listed as having any therapeutic effects, you can fairly freely buy any non-controlled substance.

For example: https://www.amazon.com/Arsenic-99-9999-Crystalline-Metalloid...

Hang on, so I could totally sell off-patent insulin in America if I wanted to with a “not for therapeutic use” label? How do all those people keeping dying “because they couldn’t afford insulin” then? Feels like someone would capture the cheap end.

Yes, you could.

The issue is getting that off label insulin distributed. Insulin must be refrigerated which really makes distribution difficult. A pharmacy isn't going to store it in their refrigerators and stores aren't going to plop it down next to the milk.

Then, of course, you run into the risk that you get sued anyways by current insulin manufacturers for patent violations (good luck with that!).

Oh, and you also run the risk that someone takes your insulin and sues you for a bad reaction. With FDA approval, there's a much lower legal risk.

And finally, after you setup your insulin product, you simply run the risk that nobody uses it because it's off label and their doctors didn't prescribe it.

Oh, and for certain, your insurance will not cover it at all. So, bunch of risks with minimal rewards. Still completely legal. Can be done doesn't mean will be done.

Insulin is a FDA approved substance with therapeutic properties. How you label it is irrelevant.

You can't sell heroin with a "nutritional supplement"label either.

If a substance has no classification(neither controlled nor therapeutic) however, like water for example; you're free to sell it, to treat dehydration, or put a homeopathy label on it and sell it to cure cancer if you prefer.

lol. You can't order aducanumab on Amazon. The only place it exists in inside Biogen. There's no "off label" here.

Right, but that's not the point. The point is you are claiming that it'd be "illegal" for biogen to sell it. It's not because it's not a controlled substance.

Biogen won't sell it, but that has little to do with the legality of selling it and everything to do with the fact that they want insurance money and to market this.

That's not how that works. If Biogen tried to sell unlabelled bottles of aducanumab, the FDA would come after them in a heartbeat.

Picture the judge in the court case: "So, you developed this new molecule that never existed before, spent years testing it to treat alzheimer's, there's been hundreds of news stories about it, there is no other reason a consumer would buy it, and it costs $50k, but you expect the court to believe you didn't think it was being sold as a treatment?"

Might I again refer you to the fact that you can literally buy arsenic on Amazon.

Would they do it? No. Would it be risky? Yes. Would it be illegal? No.

> The only place it exists in inside Biogen.

You would order it from a compounding pharmacy, would would then do a custom synthesis for you. Or else just make it yourself in your basement.

Why should Biogen be able to sell their drug? It hasn't been shown to work, it is for all intents and purposes at this point in time, snake oil.

Should Biogen be able to continue research and studies, sure, but they shouldn't be able to sell it until they have proof that it actually improves someone's life instead of having some effects on a possible bio-marker.

doesn't this translate into a many year long permission to sell a drug with no evidence that it works?

There is inconclusive evidence that it works.

That's in theory a good scenario for allowing some level of use.

If this practical level of use is good, I don't know.

Given the costs, I don't think this is good enough. Medicare cannot afford to subsidize things are inconclusive especially given how 6 million Americans have Alzheiemrs.

I think a sane system would be where a drug is legal to use, and Medicare can decide it's not worth paying for.

Those should be be independent decisions.

The $50k/year above times 6 million is $300B. For $300,000,000,000, I suspect no one cares whether it works.

What do you mean by this? That sounds like a lot of money and I would think taxpayers would care if such a large amount were to be essentially thrown away.

It certainly sounds like it, yes.

> if a patient and her doctor believe this is the best treatment, the FDA shouldn't prevent them from getting access to it.

I question whether a patient or her doctor is more qualified to make that call than a panel of experts. But we already allow drug trials - those are just measured. And if there are no other treatments, the FDA has had a compassionate use protocol for a long time to let people attempt anything over nothing.

And if the doctor is getting kickbacks?

I was prescribed a very expensive new drug that I couldn’t tolerate. Eliquis, Xarelto.

Medical office went to absurd lengths to keep me on it rather then switching to a cheaper alternative. Too multiple ER visits to get them to switch.

   Kept calling me a liar for saying it had side effects.   

Realized later that they probably made money off it. Would love to know if true.

> Realized later that they probably made money off it. Would love to know if true.

They probably didn't even make a lot of money. Although they paid $18.3 million to doctors in 2018, only nine doctors got over $100k.

Are you in the US? By law, there has been a public database of all pharama dollars to doctors for a bit over a decade. It covers lunches and consulting fees and such. For 2018, ProPublica has a searchable database. Here's Xarelto's results.

Edit: I just realized I forgot to paste in the link. I hope I edit this before you see it: https://projects.propublica.org/docdollars/products/16510

One place to search https://projects.propublica.org/docdollars/.

Disclaimer: not familiar with US medical industry or incentives schemes for docs

> No need for anyone to wear a mask.

-- Many panels of experts in Feb 2020.

And by "can distribute it", do they mean sell? 'Cause selling something you have no reason to believe is fit for purpose can cause problems.

That is, unless a large government agency has you back.