
FDA Approves Radicava, First New ALS1 Therapy in 22 Years - alexee
https://alsnewstoday.com/2017/05/08/fda-approves-radicava-first-new-als-therapy-in-20-years-and-cause-for-hope/
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hornd
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.

~~~
threecheese
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.

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JPLeRouzic
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"

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caycep
"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

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easilyBored
_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.

~~~
makomk
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.

~~~
semi-extrinsic
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).

~~~
DanBC
English prescribing is open data. The answer might be here:
[https://openprescribing.net/](https://openprescribing.net/)

Is Humira the same as adalimumab?

[https://openprescribing.net/chemical/1001030S0/](https://openprescribing.net/chemical/1001030S0/)

[https://openprescribing.net/analyse/#org=CCG&numIds=1001030S...](https://openprescribing.net/analyse/#org=CCG&numIds=1001030S0&denomIds=10.1.3&selectedTab=summary)

~~~
semi-extrinsic
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?

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baq
> 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.

~~~
epmaybe
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?

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quickben
How does this works over there in US? Government or private insurance covers
it?

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comex
Private insurance, unless you're on Medicare.

~~~
maxerickson
Medicaid may cover it and the company is providing it at no charge to people
that are uninsured and meet some other requirements
([http://web.alsa.org/site/PageNavigator/alsa_radicava_faq.htm...](http://web.alsa.org/site/PageNavigator/alsa_radicava_faq.html)
)

~~~
afarrell
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.

~~~
maxerickson
So what's your point?

I was speaking to there being other potential mechanisms to obtain the drug,
not glorifying the US health system.

~~~
afarrell
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.

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caycep
probably better to post the paper:
[https://www.ncbi.nlm.nih.gov/pubmed/28522181](https://www.ncbi.nlm.nih.gov/pubmed/28522181)
Paywall, tho. Someone with university access could probably help more than I.

The stuff is called edaravone. Radicava is the name that was made up by some
marketing intern.

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ekianjo
> 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.

~~~
satai
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.

Source, please?

~~~
ekianjo
Are you kidding me ? Are you assuming I don't know what I am commenting about
?

[http://www.cancer.net/navigating-cancer-care/how-cancer-
trea...](http://www.cancer.net/navigating-cancer-care/how-cancer-
treated/clinical-trials/placebos-cancer-clinical-trials)

"In past years, it was generally not necessary or possible to use placebos in
cancer clinical trials."

~~~
carbocation
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.

~~~
ekianjo
> 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

> In the absence of any effective therapy

