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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

#81

Post by arcanemachiner »

esturk wrote: post #53
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?

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 err on the side of caution for such a scenario.

[1] https://en.m.wikipedia.org/wiki/Per_capita_personal_income_i...

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

#82

Post by ponector »

dopa42365 wrote: post #45
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?

It's possible to do both :)

Possible in theory. But real life shows neither will be done in enough quantities.

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

#83

Post by hanniabu »

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?

I'm assuming they're setting the price so high since insurance will only approve payment of a fraction of that

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

#84

Post by Nasrudith »

downWidOutaFite wrote: post #28

How do the edited genes replace the body's genes?

They technically never do the gene replacement within the body. They take the marrow alter it, grow and produce an external modified new marrow. Chemotherapy wipes out the old bone marrow out and the modified marrow is introduced instead.

As breathtakingly advanced and unprecedented as CRISPR treatment is, the execution is still radical and crude by necessity. Not to diminish the accomplishments but to note that we still have a great deal of room to grow.

Hopefully knowledge will eventually advance so that less extreme and unpleasant methods will take its place. But that would be a tough nut to crack.

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

#85

Post by SoftTalker »

lostlogin wrote: post #69
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.

> which is why this gene has not been bred out of the population over time. Is that why? Or is it just the people with it aren’t sick enough to die before procreating?

Malaria often kills people before they reach the age of being able to procreate.

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

#86

Post by BurningFrog »

lotsofpulp wrote: post #63
soulbadguy wrote: post #61

Earlier quoted context omitted.

> Since the collective probability of rare, but expensive health issues is basically 100%, I would describe it less as insurance and more as wealth redistribution. Humm no... It's just risk amortization...

If it was just that (in the US), then there would be no need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums. The premiums are very explicitly a subsidy from young to old, which I view as a tax by a different name. Except instead of it being based on one’s income/wealth, it is based on age.

What we in the US call "health insurance" is today very different from the textbook definition of insurance.

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

#87

Post by lotsofpulp »

soulbadguy wrote: post #80
Scene_Cast2 wrote: post #72

Earlier quoted context omitted.

Is it risk amortization because we just can't predict certain health issues? Let's suppose that we had a "health oracle" (or something not too far off) to predict medical issues in individuals. How would you structure health insurance in that case?

If we had a health oracle, what would be the point of health insurance ? I think with a 100% health oracle, health inssurance will become more like group buys for negotiating better prices with health providers.

That is why the industry term for health insurers in the US is “managed care organizations” (MCOs).

When you (or your employer) buys a policy from UNH, Elevance, Cigna, CVS, Humana, etc, part of what they pay for is access to the MCOs pricing services. And vetting services to minimize errors/fraud (a process which itself is ridden with errors/fraud).

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

#88

Post by chimeracoder »

jimbob45 wrote: post #36
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?

The Hep C cure was 100k USD when it released in 2014. 10 years later, it’s 25k USD max before insurance. The price you see now will likely shrink in the coming years. Pretty good opportunity for an analysis on CRISPR pricing if you have a well-trafficked blog and are willing to track this for the next five years.

> The Hep C cure was 100k USD when it released in 2014. 10 years later, it’s 25k USD max before insurance.

That's not a great comparison. There was a previous cure for hepatitis C before the first antiviral-based cure 2013, and the initial treatment regiment for the antiviral based regimen was a hybrid of the two, before they settled on a fully antiviral-based treatment.

The reason that the antivirals came down in price so quickly was because so many nearly-identical ones came on the market within a couple of years. That's due to the discovery of a particular protein and corresponding class of inhibitors some years earlier, which was not patented, opening the door for a flood of drugs which are all functionally identical in purpose and mechanism of action, but chemically distinct and eligible for separate patent protections.

That came at a time when political and other pressures made some private insurers more willing to approve treatment (usually after a few rounds of denials and appeals) - but again, with an emphasis on some, because there are large classes of people for whom it is difficult or impossible to get treated for HCV today. (They're just not the ones likely to comment on HN).

Contrast to this treatment, which is for a congenital condition that does not have the same political pressure to address, and for which a significant financial barrier to access is not merely the costs of the drug, but the cost of the associated care (chemotherapy, etc.) which is not included in the quoted price. In addition the patent laws function differently in this case, to the detriment of patients.

The history of hepatitis C and its treatment is fairly idiosyncratic and it would be a mistake to use the price trajectory of HCV antivirals as a predictor for any other treatment.

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

#89

Post by soulbadguy »

lotsofpulp wrote: post #63
soulbadguy wrote: post #61

Earlier quoted context omitted.

> Since the collective probability of rare, but expensive health issues is basically 100%, I would describe it less as insurance and more as wealth redistribution. Humm no... It's just risk amortization...

If it was just that (in the US), then there would be no need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums. The premiums are very explicitly a subsidy from young to old, which I view as a tax by a different name. Except instead of it being based on one’s income/wealth, it is based on age.

> If it was just that (in the US), then there would be non need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums.

I do not follow the point you are making here. The regulation and legislation around health insurance do not change the nature of it.

I think you are assuming that insurers have perfect risk assessments power and thus regulating them should be unnecessary. But they don't and we have to.

> The premiums are very explicitly a subsidy from young to old

You are just repeating your assertions here. I would love some arguments.

> which I view as a tax by a different name. Except instead of it being based on one’s income/wealth, it is based on age.

Sure, as long as we agree that is just your point view and nothing rooted in reality.

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

#90

Post by lotsofpulp »

BurningFrog wrote: post #86
lotsofpulp wrote: post #63

Earlier quoted context omitted.

If it was just that (in the US), then there would be no need to prevent insurers from pricing based on health of the insured. Or legislating a 3x cap on premiums between highest and lowest premium. Or legislating out of pocket maximums. The premiums are very explicitly a subsidy from young to old, which I view as a tax by a different name. Except instead of it being based on one’s income/wealth, it is based on age.

What we in the US call "health insurance" is today very different from the textbook definition of insurance.

I don’t think any developed country in the world has an alternative “health insurance” model. I believe Switzerland and Germany require people to purchase health insurance and it is not priced based on health risk.

Health risks in general are very predictable and very high, especially as one ages.

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