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FDA approves a CRISPR-based medicine for treatment of sickle cell disease

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Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#141

Post by thereisnospork »

coldpie wrote: post #26
SamBam wrote: post #21

> Vertex set the price of Casgevy at $2.2 million > Patients must spend weeks, even months, in the hospital before and after the therapy is administered. Yoiks. So how many actual people are going to be able to get this treatment?

Indeed. The good news is, it actually turns out to be about the same or cheaper than ongoing treatment of a untreated sickle cell: """Each treatment is an individualized “one-off” treatment. For this reason, a single treatment for a single patient is expensive. At present it is estimated that in the UK treatment will cost £1 million or more. In the US the estimated cost is $2 million. That may seem prohibitive, but w…

> it actually turns out to be about the same or cheaper than ongoing treatment of a untreated sickle cell

If I were a betting man I'd wager the house that the above is exactly why it costs what it does. 'Pay 2 million now, or pay 2 million over the rest of the patient's life as they suffer' is a pretty inarguable value proposition.

Of course once patents expire and processes refine prices will come down. The wheel of progress rolls on (more of less) as intended.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#142

Post by robwwilliams »

w0mbat wrote: post #44

The gene that causes sickle cell anaemia actually provides partial immunity to malaria, which is why this gene has not been bred out of the population over time.

Good point and thanks for being on-topic. Humans have three variants of the HBB gene and having sickling mutations in the variant expressed in adult is causal to SC disease.

The FDA-approved treatments reactivate the fetal HBB gene in adults and this change in gene expression control effectively prevents SCD.

Very cool and transformative work. Now we have to get the price tag down from seven figures to four or five figures so that it will be used widely. That may be a few decades. Let’s hope that more efficient alternatives are developed soon.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#143

Post by ajross »

lotsofpulp wrote: post #135
ajross wrote: post #131

Earlier quoted context omitted.

> What I meant, though, is that across a big population’s entire lifetime, there will be a ton of high healthcare cost events. And with technological progress, new treatments will always be coming out. Which is a great thing, just not what is typically thought of as an “insurable risk”. Sorry, how does that follow? Insurance works any time you have a function with predictable average but high variance. Is the total h…

Insurable risk as in charging someone an appropriate premium that is based on their specific expected loss. Property and casualty, term life, etc. Non insurable risk as in charging someone a premium unrelated to their specific expected loss (which is what health “insurance” is).

How exactly are health insurance premiums "unrelated" to care outflows (what you're calling "expected loss")? Are you saying that health insurers books don't balance and that they're losing money (they aren't) or making too much profit (not unless they're criminally hiding it)?

What you're saying doesn't make sense. There's no difference between health insurance and any other insurance in the way it works. You collect reliable and regular premiums from everyone, pay out unreliable/bursty (but statistically very predictable in aggregate!) losses as contracted, and pocket the remainder as profit. And it works.

Really, I don't know what you're talking about here. Health insurance is "expensive" in the US, sure. But it's not failing.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#144

Post by robwwilliams »

ericmay wrote: post #129
ponector wrote: post #37

Earlier quoted context omitted.

I don't agree that it is nothing. 2 millions, if applied properly, could do good for many people. Take ten children from poverty, give ten children chance to get a good education, etc. There is always a some kind of moral dilemma: should you spent millions to try to extend extremely I'll person or help with that money to some healthy poor children?

I understand the dilemma, but many of those same children you are thinking of live in poverty in places such as Africa and with the misfortune of sickle cell disease. If we prevented treatment because the money could be used elsewhere, we likely wouldn’t/won’t develop a drug that we could eventually[1] make cheap enough to cure these kids and give them longer lives too. We can do better! [1] There is a cynical take h…

As mentioned above—this is day 1.

How much did the first human genome sequence cost? (Effectively several billion dollars—now $1000.) How much did the first organ transplant cost? How much did the first electronic computer cost?

Yes, cost will slow widespread use but it will spur the. next wave of innovation—-some motivated by profit, some motivated by social altruism.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#145

Post by maxerickson »

elektor wrote: post #109
huytersd wrote: post #65

Earlier quoted context omitted.

You answered your own question. The taxpayer already pays the same amount for lifetime treatment. This is just going to be the same except the person can lead a completely normal life after this. Also over time this will probably be much, much cheaper than the current lifetime treatment. There’s no reason a lot of the process can’t be easily automated.

No, I did not "answer my own question". These therapies are priced at $2.2 and $3.1 million, much greater than their calculated cost-effective price. And these are not small molecules that can be easily made generic, so not likely to get that much cheaper over time.

Plasmid production is pretty well industrialized. Lots of biotech drugs/treatments ferment plasmids as a step in their process (for example the Covid mRNA vaccines that cost $25).

I imagine an expensive part of this process is incubating the treated stem cells to increase their numbers, and then just the cost of the hospital time while the new cells establish and the patients immune system recovers from the chemotherapy.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#146

Post by mritchie712 »

graphe wrote: post #120
mritchie712 wrote: post #25

Ohalo (the company Dave Friedberg is now CEO of) recently got approval for a potato edited by CRISPR: > Ohalo had two RSRs under consideration this year for its potato, one which focuses on higher concentrations of beta carotene – enhancing the overall health and nutrition value of the potato – and another which results in reduced glucose and fructose content in the potato, which, according to Ohalo, will reduce the…

I would have bred them for more potato protein. It's a very close meat protein substitute by essential amino acids. Might be why it tastes so good. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6245118/ >Of the ten plant-based proteins included in the current analysis, potato protein is the only protein source containing the WHO/FAO/UNU requirements for all essential amino acids. Thus, when consuming potato protein as…

Not quite vegan, but I like cricket protein.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#148

Post by firejake308 »

eszed wrote: post #122
jorlow wrote: post #115

Earlier quoted context omitted.

The article says "patients must undergo a preparatory treatment with a chemotherapy drug to remove any native stem cells that might remain in their bone marrow." It doesn't make much difference to the patient if it's the Lyfgenia itself or the chemo drug, if the chemo drug is a requirement. Right?

Fair enough, but there remains the possibility of finding an alternate, safer chemotherapy drug. Does anyone know if changing the drug would require a new FDA approval for the entire regimen, or could the protocol be easily changed?

It's reasonable to expect small improvements in the risk profile, but I think blood cancer is going to be a side effect for any drug following this basic idea. You will always need some chemo to destroy the defective blood-making stem cells before replacing them with the genetically-modified blood-making stem cells, and any chemo that is strong enough to kill all of the blood-making stem cells in your body is necessarily going to have a risk of damaging healthy cells and turning them into pre-cancer cells. So the risk can be reduced but probably not eliminated.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#149

Post by vidarh »

arcanemachiner wrote: post #81
esturk wrote: post #53

Earlier quoted context omitted.

Such a crass statement. What if you're the patient? Would you spend 2 million to live 30-40 more years? It's so easy to step back and weight the lives of other as if you're making the decision for others.

The median income in America is a little under $40000 per person[1], so that $2.2 million pretty much represents the entire financial income of the average American over a working lifetime (55-60 years). So in essence, you'd be trading the equivalent of one person's entire lifetime of productivity in exchange for the first generation of a radical new medicine whose outcome is unknowable. I don't think it's crass to e…

These people mostly do get treatment now, for decades, involving regular expensive long term hospital stays. So you're trading already expensive treatments that cut their earnings potential drastically both by cutting number of productive years but also due to extensive sick leave.

So if there even is an increase in the total cost of treatments, it's not at all a given it's a a net increase once account for decades of additional working life.

Re: FDA approves a CRISPR-based medicine for treatment of sickle cell disease

#150

Post by smt88 »

mritchie712 wrote: post #146
graphe wrote: post #120

Earlier quoted context omitted.

I would have bred them for more potato protein. It's a very close meat protein substitute by essential amino acids. Might be why it tastes so good. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6245118/ >Of the ten plant-based proteins included in the current analysis, potato protein is the only protein source containing the WHO/FAO/UNU requirements for all essential amino acids. Thus, when consuming potato protein as…

Not quite vegan, but I like cricket protein.

Cricket protein is still insanely expensive though.
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