I realize this doesn't add anything to the conversation, but as someone whose mom passed away from ALS, I welcome anything that can remotely improve this terrible disease. Watching her go from walking to barely able to lift a glass in six months was devastating and I'd have given much for 33% more able-bodied time with her.
Me too. I almost can't keep it together reading this. 33%!! I do wish it's mechanism was known, hopefully anyway it will open up the door for better treatments.
I feel for you. Just two days ago I learned that an old friend of mine, one whom I haven't seen in many years, has been suffering from ALS recently. I hope to see her soon.
I have some problem to understand why this is presented as a therapy (may be I do not understand what it implies) when the paper (or I may have found the wrong paper) states:
"Edaravone showed efficacy in a small subset of people with ALS who met criteria identified in post-hoc analysis of a previous phase 3 study, showing a significantly smaller decline of ALSFRS-R score compared with placebo. There is no indication that edaravone might be effective in a wider population of patients with ALS who do not meet the criteria"
Another paper is quite skeptical:
"Edaravone: a new treatment for ALS on the horizon?
Orla Hardiman, , Leonard H van den Berg"
"therapy" means something you do to someone to try and change things. It may be a good therapy or crappy therapy, but it falls under the definition of therapy. Kind of like that Calvin Trillin line about a particular restaurant being debated as "good decor or bad decor but you can't say there's no decor..."
Overall, I'm always skeptical about treatments that focuses on "reducing oxidative damage"...because if you think about it, it doesn't really focus specifically on the disease itself. Oxidative damage happens to everyone daily, just by breathing air.
There's a bit of viewgraph engineering too, because the treatment group declined by 5-ish points, and the control group by 7 ish points out of a 48 point scale, they say "33% improvement"....
That being said, I suppose because it does something more than riluzole and doesn't kill anyone more than a typical chemo therapy, the company managed to get the FDA to Ok it...TBH, I probably should be more familiar w/ the criteria with which the FDA OKs or doesn't OK something
According to the ALS Association, the treatment’s list price is $1,000 per infusion, or about $146,000 annually, and it is expected to be available for use by August
A whole new class of drugs is reaching these prices. "The insurance" doesn't really pay; those xx% increases every year in premiums do.
Yeah. Other countries generally solve this by not covering these treatments; for example there's no way this would be available on the NHS at this price. Somehow I suspect this would be a political non-starter in the US through.
How many people with some sort of inflammation are on anti-TNF-alpha aka. Humira (for arthritis, psoriasis, intestinal inflammation diseases, etc.) in the UK? It's a treatment covered by the NHS. While "only" £10,000 per patient per year, so an order of magnitude cheaper than what is discussed here, it's typically prescribed for orders of magnitude longer (as in several decades).
Yup, adalimumab is another name (naming of biologicals is weird). That data suggests about 500 people in the UK are on Humira; a bit lower than I expected, but I gather they only prescribe it as a drug of last resort.
However, if you click to get map data in the second link you give it's really weird. There's basically no subscription in most of England, but very high in a few small pockets, and not in any sensible pattern (e.g. if you scroll back a couple of months there's none in all of London, and these items are prescribed in a very regular way). Maybe this search somehow doesn't get all the data?
No, a huge portion of the US is on board with the idea that the government shouldn't be covering any medical care.
Apparently they don't even want the federal government having standards for acceptable insurance plans (but they want a federal law saying that the standards set by any state can be used to market plans in all states).
> According to the ALS Association, the treatment’s list price is $1,000 per infusion, or about $146,000 annually, and it is expected to be available for use by August.
$146k/year for being able to live a better life, or maybe at all. right up there with cancer treatments according to my ethical standards.
Would it be better for them to just not make the new treatment?
Stuff like this doesn't scale in the way we are used to. There are only 30,000 ALS patients in the US, and this drug only benefits a subset of those people. And it costs tens if not hundreds of millions of dollars to develop. How exactly should you price it?
Now you can say the government should pay for it. And I agree that as a general matter the government should pay for healthcare. But that's not really a workable solution here either. At that point you've got to ask the difficult but necessary question: should the government be investing that much money to help maybe a few thousand people? Imagine how many poor kids whose lives you could change with that money instead.
I don't claim to understand this aspect of healthcare very well. But I've definitely observed that the list price of medications keeps on going up, insurance keeps on paying for it, and we keep on having higher premiums. Who wins? Who loses?
Wikipedia suggests it's very expensive, but the molecule looks simple enough. I imagine there would be a prominent gray-market of nootraceuticals in no time.
How much paperwork does it take to prove you meet those requirements and what is the cognitive/time cost of that paperwork?
Gentoo is free-as-in-speech, but way way too expensive for most people. Given that the US healthcare system is bewildering even to middle-class people who have relatives that work in healthcare, the cognitive costs of something like this might be considerable.
To tell the person asking "How does this works in the US?" That even if there theoretically are means to obtain the drug for the uninsured, it might be practically out-of-reach.
> Results from the six-month Japanese clinical trial — in which 137 patients were randomized to receive either Radicava or placebo
Giving placebo to ALS patients? How is that allowed ? They should be able to tell already what is the course of a typical ALS patient without using such methods. For cancer drugs there are no placebo used since there is extensive survival data available to prove whether or not a drug actually makes any difference.
You have to get rid of placebo effect. It can be pretty strong in an (supposedly) advanced drug tests.
>For cancer drugs there are no placebo used since there is extensive survival data available to prove whether or not a drug actually makes any difference.
Placebo isn't used because there are treatments that are effective (even if the effect is small). Therefore, the placebo comparison is (for many cases) unethical. The appropriate comparison is to the standard therapy, not to no therapy at all.
If there is no therapy that is known to be effective for a particular illness, then placebo control remains appropriate.
> If there is no therapy that is known to be effective for a particular illness, then placebo control remains appropriate.
For cancer there's almost always some kind of combination that gives some results (even if not great). So your statement proves what I said.
> Use of placebo controls may be justified in these situations:
> To prove effectiveness of a new treatment for diseases with high placebo response rates
> In conditions that alternately become worse or better, have spontaneous remissions (the disappearance of the signs and symptoms of cancer, but not necessarily the entire disease), or have an uncertain and unpredictable course
> When existing therapies are minimally effective or have serious side effects
