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Gene Therapy Was Hailed as a Revolution – Then Came the Bill

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Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#61

Post by Ntrails »

aaronbrethorst wrote: post #20
skwb wrote: post #18

Earlier quoted context omitted.

But due to insurance churn, you don’t want to be footing the bill for your competitor down the road. As an insurance manager, your unit of analysis is not healthcare system or society, but your quarterly bottom line.

Sounds like a great argument for Medicare for All, the NHS, or other nationalized systems.

> Sounds like a great argument for Medicare for All, the NHS, or other nationalized systems.

There's a good chance that NICE won't be willing to fund these treatments at all. Which is in no way a criticism of them (QALY is the just the most sensible semi-objective thing to base these decisions on) - but is worth highlighting when you say "free healthcare for all".

It's only free for the healthcare considered to be good enough value for money.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#62

Post by empath75 »

refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

Letting people die is even cheaper.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#63

Post by rayiner »

fapjacks wrote: post #54
nec4b wrote: post #43

Earlier quoted context omitted.

Could you link to drugs that you have developed and how much they cost?

Can you name a single drug that was developed without public assistance from taxpayers?

90/10 rule. The last 10% is 90% of the work.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#64

Post by refurb »

yread wrote: post #32
refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

From a more general pricing perspective: how should you price your product versus the status quo? Is it really appealing if it's only 10% cheaper? For my SaaS I was aiming more at 50% cheaper. Am I leaving money on the table?

There is a whole specialized field of pricing. The answer is it depends. And even then there are often multiple “correct” strategies.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#65

Post by rayiner »

anfilt wrote: post #3
refurb wrote: post #2

What this article misses is that even with a price tag of more than $1M, many of these therapies will save the system money . They kind of gloss over it when talking about Zolgensma, but the current treatment, Spinraza, costs ~$750K in the first year and $375K every year after. So even by year 2, you've already spent one million and you pay that indefinitely . So if Zolgensma shows 5 years of efficacy and Zolgensma c…

At that point it's probably just cheaper to get a plane ticket to a country that does not patent or enforce a patent for the treatment.

How many life-saving gene therapies have been developed in these countries?

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#66

Post by codeulike »

folli wrote: post #51
codeulike wrote: post #36

Earlier quoted context omitted.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

CRISPR is unrelated to Zolgensma, which uses an Adeno-associated virus to deliver a functioning version of the SMN1 gene.

I know. I was giving an example of modern biotech

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#67

Post by rayiner »

tuxxy wrote: post #8
refurb wrote: post #4

Earlier quoted context omitted.

Unlike small molecules, gene therapy isn't something that somebody can just whip up in a lab. Similar to biologics, there are often some trade secrets involved in the manufacturing that require a lot of time and money to figure out.

How can anyone think this is ethical for lifesaving medical care?

In a market-oriented society, it's unethical to put in place rules that divert capital and talent way from the development of lifesaving medical care. (And the half-ass refrain of accepting a market economy generally, but making an exception for medical care as being "too important to leave to the market" makes no sense. Unless you tamp down on the flow of capital to other industries, designating some areas as "too important" to leave to the market will simply cause capital to flee to, e.g. dating websites, leaving the "too important" industries as ghettos.)

The ethical thing to do is constructing a system that maximizes the amount of lifesaving medical care at minimum cost. Doing that requires acknowledging market forces. Scientists and engineers aren't any more altruistic than anyone else. These days the incoming classes at investment banks are full of physics, biology, and chemistry majors that could easily have gotten a PhD and went to go work for NIH. In the healthcare space, there is at least the potential for making a lot of money by doing a startup with the hopes of getting acquired by one of the big pharma companies. If you take that option away--if developing lifesaving medical care is a field that nets you at best a GS-scale government job--talent and capital are going to go develop dating apps instead.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#68

Post by nradov »

ordu wrote: post #47
Mirioron wrote: post #21

Earlier quoted context omitted.

It's ethical, because as a society we need to look at the long term rather than any specific treatment. It's better for us if private investors invest money into lifesaving treatments, because it means that more of them will be available earlier to people. If investors go and invest in something else then nobody's getting the benefit of the medicine. Let's say we have a disease that kills 1000 people per year. Let's…

I like your comment, it explains a lot. It seems that the system was built the way, that makes a drug research to grow a pie, while in an ideal world where everyone is happy and everything is fair, drug research would shrink the pie: it is the goal of medicine to eliminate all diseases. It means, that all that system is working on self-destruction, but we allow them to get excessive profits now, to make it work. Ther…

Competition does work. You can see that by looking at the average profit margins for drug companies: they aren't particularly high. The problem is that drug development is just really hard and expensive. All the low hanging fruit has already been picked.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#69

Post by nradov »

mikekchar wrote: post #34
refurb wrote: post #13

Earlier quoted context omitted.

Not sure what you mean. Are you saying it’s unethical that it’s hard to make new therapies?

It's interesting to speculate on the "It costs $1 to press the button and $10K for knowing which button to press" kinds of problems. I mean, if you spend several hundred thousand on a medical degree, it is ethical to ignore someone dying if they can't pay you? Personally, I think it is -- nobody should be forced to do something just because they know how. However, I can totally understand the other point of view. Do…

In the US, hospitals are legally required to treat patients in serious condition regardless of ability to pay. The hospital has to at least get the patient stabilized before discharge. Hospitals receive some government funding for that, and then they try to make up the rest of the cost by raising prices for insured patients.

Clinics aren't subject to the same requirement and can legally turn indigent patients away.

Re: Gene Therapy Was Hailed as a Revolution – Then Came the Bill

#70

Post by [deleted] »

dekhn wrote: post #56
codeulike wrote: post #36

Earlier quoted context omitted.

CRISPR's not that hard, this guy did it in his garage. Delivering it to cells is a bit harder, but not that hard unless you're talking brain cells, then the blood-brain barrier causes trouble. https://www.theguardian.com/science/2017/dec/24/josiah-zayne...

anybody with a decent level of biochemical knowledge can do these things in a garage (I built a biolab in my garage and work at a lab that does CRISPR). However, the person you cited is clueless and is just kind of injecting things into his body without any really thought to efficacy or safety. There is a big difference between injecting reagents into a single human, and doing actual medicine.

[deleted]
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