ALS Ice Bucket Challenge funded drug "Relyvrio" gets FDA approval

Originally published at: ALS Ice Bucket Challenge funded drug "Relyvrio" gets FDA approval | Boing Boing


Any of us that have watched ALS take the life out’a another human being know how important this is.


There is also a fairly new drug for people with cystic fibrosis, Trifacta, which is making a huge difference; people who expected to die around thirty, may live a normal lifespan.


I lost my father-in-law to ALS. But I definitely understand why this approval is so controversial. There isn’t yet stong evidence that this drug is effective, and such evidence won’t exist until an ongoing trial with 600 patients is finished in 2024, yet the FDA is going to allow this company to start charging $158k/year for this treatment starting now.

Enrolling a lot of willing, informed patients into trials right now would be great, as long as the trials are no cost or at least low cost. But you can’t always justify charging that kind of money for an unproven treatment just because you have desperate patients willing to try anything because they have no other options.

This part from the linked article sure raises some red flags to me, at least regarding the motivations of the drug company:

So if the big trial turns out to be negative they might voluntarily pull the drug off the market, if that’s ok with their investors. But no mention on whether they would consider refunding any of that substantial amount of money to patients if it turns out to be a dud.


ALS is a terrible fucking disease. Its the kind I would take a long camping trip and not come back from. I DID do the ice bucket challenge. I was pretty funny when I did it. I ended up donating some as well, though at the time i was pretty broke.


The drug is believed to slow the progression of the disease, which is great.
Claiming that an ALS drug works might suggest to some that it can cure, halt, or prevent the disease.

My understanding is that it’s hard to predict the rate of progression of the disease in any particular patient. When my uncle was diagnosed, he was told he probably had 6 months to 2 years to live, yet he survived much longer, like 10 or 12 years. Maybe he was an outlier, or maybe it’s just really hard to predict how the disease will progress. Either way, it seems like proving the effectiveness of a drug that might give patients 5 or 6 more months of life would be challenging without a relatively large pool to get statistically significant results.


That’s a great idea, but if I demanded that as part of my contractual agreement in a trial, I think they would find a reason to not let me in.

ALS is a horrible affliction that I hope this drug will provide some extra agency to the afflicted, and offer more choices where there were far too few in the past. I’m unsure of the efficacy of this drug, but I am certain that for some, a pill prescribed can work wonders on the power of positivity and faith strong enough to transcend beyond the miraculous, back to the vaunted air of full remission.

On the $158k/year price, I have to recognize that I’m constantly asking: “Is this a lot to you?!?” as prices rise and now I’m that old grandpa-to-gen-alpha; yelling about “back in my day, a Coke cost 99 cents.” I have to check myself because I’ll lose auctions for stuff simply because my personal CPI is out of date due to extensive shopping online. Gone are the days when I would wander up and down each aisle, mentally cataloging where everything was, only to be undone when the new guy was put to work and now the international section is all jacked up, again. The days of empty grocery stores is gone too, where I never feel that empty airport-butter flies in your stomach feeling like pre-Rona late-night grocery store trips used to elicit.

That’s a lot unless you’re a member of the 1%. Maybe the 0.5%. The median wages in the US are just over $50k right now, so that’s over 3 year’s wages for most people. For one year of medicine. You either have to be filthy rich or have outstanding insurance to afford that.


It’s also about two orders of magnitude more expensive than Riluzole, which is the only other FDA-approved drug to treat ALS. That one only extends life by about 3 months but at least it had a lot more data behind it than this new drug.


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