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A million-dollar drug

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Re: A million-dollar drug

#81

Post by tempestn »

tptacek wrote: post #35

People are fixated on the price for this drug, but the article itself is pretty clear on the rationale for the price tag: current alternative therapies have a price tag of up to $300k/yr, indefinitely. This drug is a one-time therapy. Presumably, any health system would jump at the chance to replace a $300k/yr recurring charge with a 1-time $1MM fee. But they don't. Health systems in Europe apparently refuse to pay f…

The article talks about turning white, fat-filled blood red, so presumably it would have an effect on blood fat level. Is the issue that humans with this disease have no choice but to remain on extremely strict diets, which does a reasonable job of controlling their blood fat level. This treatment (cure, essentially) would alleviate that, but not necessarily change the levels much, since the patients were already (laboriously) controlling them?

Re: A million-dollar drug

#83

Post by tptacek »

tempestn wrote: post #81
tptacek wrote: post #35

People are fixated on the price for this drug, but the article itself is pretty clear on the rationale for the price tag: current alternative therapies have a price tag of up to $300k/yr, indefinitely. This drug is a one-time therapy. Presumably, any health system would jump at the chance to replace a $300k/yr recurring charge with a 1-time $1MM fee. But they don't. Health systems in Europe apparently refuse to pay f…

The article talks about turning white, fat-filled blood red, so presumably it would have an effect on blood fat level. Is the issue that humans with this disease have no choice but to remain on extremely strict diets, which does a reasonable job of controlling their blood fat level. This treatment (cure, essentially) would alleviate that, but not necessarily change the levels much, since the patients were already (la…

To learn about the efficacy concerns, you have to go read other articles about the treatment, which will come up right away when you Google it because the withdrawal of this treatment from the market was a big story.

Re: A million-dollar drug

#84

Post by SilasX »

ericflo wrote: post #76
rayiner wrote: post #47

Earlier quoted context omitted.

It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex... . The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which…

> Viewing this after-the-fact leads one astray, into thinking there is some problem with the system > The answer is probably that this drug doesn't make economic sense You don't see it? The problem with the system is that we let economics drive these decisions.

What would it look like for economics not to “drive decisions”? Spend unbounded money to save each life? That doesn’t make sense either.

What decision mechanism do yo prefer, and how would it work differently here? Why would that outcome be better?

Remember, even the much-lauded European health care systems didn’t feel it passed the cost benefit test, and they’re supposed to be the model of “not letting people die for lack of money”.

Rejecting economic thinking doesn’t suddenly solve the problem of opportunity costs and trade offs.

Re: A million-dollar drug

#85

Post by maxander »

skadamou wrote: post #54
rayiner wrote: post #47

Earlier quoted context omitted.

It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex... . The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which…

This is an excellent response and you clearly understand the issue well. I'm curious though, what do you think about the long term effects of continuing to practice gene therapy? Sure, the cost for treating the first few patients will be exorbitant but wouldn't we expect researchers to find ways to bring costs down over time? How will we know if this is even possible if we abandon gene therapy at such an early stage?…

The inherent cost of manufacturing the drug is relatively negligible. The problem is the cost of development and deployment, and the tiny customer (patient) population to spread that cost around to.

A gene therapy for a more prevalent disorder would not have the same problems, or at least not as badly.

Re: A million-dollar drug

#86

Post by maxander »

quotemstr wrote: post #59
wpietri wrote: post #39

Earlier quoted context omitted.

I don't think he understands pricing at all. Market price isn't arrived at via a rational process. It's an inherently social process arrived at by negotiation between producers and consumers. And it's especially weird that he thinks pricing shouldn't be political when his product has a government-granted monopoly and the bulk of its customers will be government-run health-care systems and government-licensed insurers…

> I don't think he understands pricing at all. Market price isn't arrived at via a rational process. It's an inherently social process arrived at by negotiation between producers and consumers. Are you suggesting that prices don't have anything to do with scarcity and are just set arbitrarily by social forces? That doesn't seem quite right.

Well, yes, that’s how supply and demand (supposedly) works. The “correct” price is where the demand, at that price, equals the available supply.

If the product is scarce, that can drive up the price, but only as far as demand is willing to follow- there’s no inherent value in being scarce. “Price” is a social thing, so of course it’s determibed by social forces.

Re: A million-dollar drug

#87

Post by irq11 »

JamesBarney wrote: post #56
rayiner wrote: post #47

Earlier quoted context omitted.

It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex... . The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which…

But of that 100 million dollars the bulk was safety testing due to regulation. So you're right it doesn't make economics sense but for a specific reason. As a society we have to strike a balance between safety and efficiency. And I don't think we've struck the right balance. Many times erroring too much on the side of safety, especially with rarer and more fatal diseases without good current treatments.

You’re focusing on the wrong thing.

Even if you ignored the cost of regulation and development, the company who was producing this drug would need to spend millions of dollars a year on production just to keep a safe supply available. With a pool of a few hundred patients, all of whom are “cured” for a decade, it’s going to be an expensive drug no matter what regulatory system is in place.

Re: A million-dollar drug

#88

Post by sdenton4 »

rayiner wrote: post #47
mabbo wrote: post #24

The point of the patent system is that we are giving inventors a time-limited monopoly on their invention in exchange for them sharing it with the world. We all benefit because we have access to this new invention and soon can make it ourselves, and the inventor has an incentive to promote and sell as much as they can while they own the monopoly. But it's not working here. The patent holder is effectively saying soci…

It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex... . The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which…

To be sure, if you're testing the new platform for a medical technology and can't scrounge up 350 people for your study, there's no faith that you're going to make something that actually works.

It would be far wiser to build a treatment for something where you can get a significant sample size to prove out the platform, and then move into smaller populations from there, building on the newly gained knowledge.

Data speaks, and statistics are how we listen. If you can't do the stats, you shouldn't try to claim an advance.

Re: A million-dollar drug

#89

Post by JunkDNA »

skadamou wrote: post #54
rayiner wrote: post #47

Earlier quoted context omitted.

It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex... . The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which…

This is an excellent response and you clearly understand the issue well. I'm curious though, what do you think about the long term effects of continuing to practice gene therapy? Sure, the cost for treating the first few patients will be exorbitant but wouldn't we expect researchers to find ways to bring costs down over time? How will we know if this is even possible if we abandon gene therapy at such an early stage?…

There’s a bigger issue than gene therapy. Precision therapies that work really well for a very small number of people are going to become increasingly common (whether gene based or otherwise). We are seeing this in cancer right now where there are finally true cures emerging. It’s clear current pricing models are not going to work in a world where you need lots of different therapies to treat small clusters of patients.

Outside of cancer (the only real place where there is a broad clinical application of precision medicine at scale) there’s an estimated 25-30 million people in the US with a rare disease (a disease that affects less than 200,000 people) [1]. There are an estimated 7,000 different rare diseases. That makes rare diseasss more common than cancer in the us which is at about 15 million people [2].

So what if we end up with cancer itself being subdivided into thousands of small diseases (something that is already well underway) in addition to all these “rare” diseases? The current model of developing therapies is just not going to cut it. We need new development methods that are not so cost-intensive and we need new payment models that create enough incentive that individuals and companies take enough risk to identify therapies. It’s a ferociously hard problem.

[1] https://rarediseases.info.nih.gov/diseases/pages/31/faqs-abo...

[2] https://seer.cancer.gov/statfacts/html/all.html

Re: A million-dollar drug

#90

Post by ericflo »

SilasX wrote: post #84
ericflo wrote: post #76

Earlier quoted context omitted.

> Viewing this after-the-fact leads one astray, into thinking there is some problem with the system > The answer is probably that this drug doesn't make economic sense You don't see it? The problem with the system is that we let economics drive these decisions.

What would it look like for economics not to “drive decisions”? Spend unbounded money to save each life? That doesn’t make sense either. What decision mechanism do yo prefer, and how would it work differently here? Why would that outcome be better? Remember, even the much-lauded European health care systems didn’t feel it passed the cost benefit test, and they’re supposed to be the model of “not letting people die fo…

You quoted me, but missed the important word - these decisions. It's a tragedy of the commons type thing, and we can carve out specific solutions to that, as we do for these types of situations. Where the European healthcare bureaucracy draws their specific moral lines has no bearing on my overall argument.
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