7 ms·

Can a wealthy family change the course of a deadly brain disease?

39 points by Snoozus 6mo ago

tylermcgraw 6mo ago
This is the model for rare diseases that wouldn’t be profitable for pharmaceutical companies. Spinal muscular atrophy (sma) is another example that comes to mind.
- yieldcrv 6mo ago
  > diseases that wouldn’t be profitable for pharmaceutical companies I remember when that observation was discredited as a conspiracy theory
  - wat10000 6mo ago
    I’ve never seen that discredited. Are you confusing the obvious fact that they won’t pursue unprofitable drugs with the much more dubious idea that they won’t pursue profitable cures because ongoing treatment is even more profitable?
    - Aurornis 6mo ago
      The dubious idea is that eliminating private medical care systems would open up a world of research into treating very rare conditions with high R&D costs. If this was true, why wouldn't all of the countries with socialized medicine be doing it already?
      - paulryanrogers 6mo ago
        The US already was, and to since extent still does. Same in the UK and other parts of Europe. Government funds a lot of medical R&D. Thank them for the fundamental research that lead to the COVID vaccine.
  - AdamN 6mo ago
    Where and when?
- Incipient 6mo ago
  That's one reason why privatised health is rubbish. "profitable" treatments should be used, in part, to subsidise the cost of unprofitable ones.
  - renewiltord 6mo ago
    [flagged]
    - sokka_h2otribe 6mo ago
      Uhh, you know you could skip the vaccine and just call it a tax..
      - renewiltord 6mo ago
        That wouldn't guarantee subsidization of expensive treatments by cheap ones and therefore is fascist.
  - Aurornis 6mo ago
    No medical system, public or private, has infinite money. There will always be decisions made about which conditions get research and which don't. It's unlikely that a disease this rare would be prioritized by a purely government run system, either. There are too many more common diseases to address first.
    - AdamN 6mo ago
      It's a little bit the opposite. Private groups are focused on profits but there are gov programs to support the rare disease research that would otherwise go unfunded in a pure market system.
    - Incipient 6mo ago
      I mean sure, nothing has infinite money...but better to redirect some profits to other treatments, than to extract them all for shareholders?
- wjxgxey 6mo ago
  pfft just illusion of control theatre for people who are scared of death. Throw in some opportunists exploiting it. Just watch what happens if there are unintended side effects. Its okay to die guys. Everyone does it. The sky doesnt fall.
  - zdragnar 6mo ago
    Dementia is a terrible way to go, both for the people who get it and for their loved ones who are with them. One day, my grandmother forgot English when my uncle was visiting and kept speaking in her native tongue and got so mad because nobody understood her. That was one of the few amusing anecdotes from get decline. The rest are just depressing. Watching your father cry because he went to the hardware store and couldn't remember how to get home and had to ask an employee to call his family for him, for example, was particularly tough.
    - wjxgxey 6mo ago
      You know why that happens? Because the health care system slows natural decay rate of some subsystems (via pills/surgeries etc) while having nothing to offer for other subsystems. So rather than all subsystems decaying together we produce this mismatched state.
      - temp_praneshp 6mo ago
        That's the response you have to the parent's anecdotes? I hope that one day you are not sad and angry anymore.
      - zdc1 6mo ago
        You can't really blame the healthcare system for this. Alzheimer's and Dementia existed before modern medicine. The reality is that many fit, active, and otherwise healthy people will hit their 60s and 70s and will experience cognitive decline and Alzheimer's.
        wjxgxey 6mo ago
- david_shi 6mo ago
  Seems insane that profitability so heavily dictates what is researched and what isn't.
  - silisili 6mo ago
    I don't mean to disagree with you in spirit, but profitability is pretty closely entwined with probability. So companies are chasing solving problems that more people have, even if it's for the wrong reason.
    - ehnto 6mo ago
      As the benefactor of an extremely rare disease, it's not exactly unfair when you look at it from a societal view. If you solve a higher probability problem, you are helping far more people. The real tragedy isn't the allocation of the resources we have spare, it's that so many of our resources are not spare because billionares and corporations have hoarded it. Without changing the percent of allocation, and only changing input resources by capturing it back from billionaires as taxes, we could be helping far more people including super rare diseases.
      - silisili 6mo ago
        Absolutely. And if you take a step back and look at Covid spending, what it was spent on, and how much fraud was involved, it's absolutely maddening that the government isn't instead spending money on solving actual problems its constituents face. We basically just shoveled free money at anyone who claimed to have a business, to no real effect. C'est la vie, I guess.
      - AdamN 6mo ago
        I don't know how much longer it will last but the US government invests significant resources into rare diseases in order to improve outcomes where the normal market wouldn't otherwise support the r&d.
  - s1artibartfast 6mo ago
    I find it makes more sense if you drop the corporate analysis and just think about people. Money motivates them and is why they go into hospitals or research labs instead of staying home with their family or friends.
georgeburdell 6mo ago
I know another family like this. One partner still works, but the other one is essentially a full time advocate for an inherited disease that fewer than 100 people in the world are affected by. I don't think much money is involved, but they've changed the narrative about the disease and some researchers are taking them seriously.
the_wolo 6mo ago
If so, good for them, good for the humanity, but what we actually must do is to ~~expropriate~~ ~~socialize~~ democratize the means of production.
- CMay 6mo ago
  the reason your encouraged approach tends to produce poor results, is that you increase the distance between the decisions that need to be made and the people who understand how to make the decision or whether a decision is even valuable to make. it is basically an unsustainable structure. there's not much value to replacing one structure which you might think is unsustainable with something equally or less sustainable that also produces worse results anyway. another issue is that it can dilute responsibility and someone will take more assertive control anyway which further reduces the quality of decision making. someone still has to enact and enforce the decisions, so whoever does the enacting has to obey and whoever does the enforcing has to enforce the right thing. it's easy to end up with a bunch of people influencing things for their own reasons which have nothing to do with maximizing the production of good results.
- onion2k 6mo ago
  In this case (and probably lots of others) the amount of resources needed to make an impact on the problem means that it would never work if the funding was decided through a democratic process. As soon as a committee of people are deciding to where to put resources they decide to share it between a number of worthy causes, and that means none of them get the bulk of what's available. If you have something that's percieved to be a relatively small problem by people it isn't directly impacting every day, that needs a lot of resources to fix it, then it's never getting done. (Tech-related side note: This is why companies build mountains of tech debt unless there's a former engineer running the show.)
- radu_floricica 6mo ago
  Poe's law? Can't really tell, with the internet these days. Things are so polarized that people talk from and to their tribe and the message is often understood to be obvious. Anyways, in case you're serious: there is a famous thought experiment about healthcare: should a hospital administrator approve a complex and expensive treatment to save a 7 year old girl, with a 100% success probability? Or more to the point: is approving this a "good" act, or a "bad" act? The unintuitive answer is that it depends on the opportunity cost long term, and the math is far from obvious. The quick answer is often "not even wrong" - it simply ignores a lot of facts down the line. The same cash can be used very boringly to do maintenance or to buy a piece of life saving equipment. And it gets very dirty very fast. The easy version is pay for the operation vs buy an MRI machine - in this case you can at least compare apples to apples, if you squint - an MRI machine also saves lives. But if the alternative is renovating a waiting room, you're really off the deep end. Because doing it one time it's an obvious decision: just save the life. And it's not even bad as a general policy: have waiting rooms be a bit dusty, if this means spending more money on treatments. But... how much to cut, exactly? And then you have second order effects: throwing a moderate amount to waiting rooms can make them look a lot better, while having it linger in disrepair can make people actually avoid the hospital (if they can't even paint the walls, why would you trust them with your life?). And all of this is assuming the hospital admin actually has mental bandwidth and latitude to make this decision. In practice, he's looking at the kid's mom when he has to turn her down because the operation money would pay for renovating a whole hospital wing. And the mom is an influencer with a large following. > democratize the means of production This means having humans make such decisions, and even worse, it means a committee or a mob will make such decisions. Zero skin in the game, all vibes and feelings. Some girls will get saved, but I've seen how hospitals look in such a system, and it's not good. To note: the US system may or may not be "democratized", but it managed to have exactly the same flaws - most decisions are taken by humans in a bureaucracy. The government simply wrote the rules then outsourced the bureaucracy and the blame to private corporations.

39 points by Snoozus 6mo ago

This is the model for rare diseases that wouldn’t be profitable for pharmaceutical companies. Spinal muscular atrophy (sma) is another example that comes to mind.

yieldcrv 6mo ago
> diseases that wouldn’t be profitable for pharmaceutical companies I remember when that observation was discredited as a conspiracy theory
- wat10000 6mo ago
  I’ve never seen that discredited. Are you confusing the obvious fact that they won’t pursue unprofitable drugs with the much more dubious idea that they won’t pursue profitable cures because ongoing treatment is even more profitable?
  - Aurornis 6mo ago
    The dubious idea is that eliminating private medical care systems would open up a world of research into treating very rare conditions with high R&D costs. If this was true, why wouldn't all of the countries with socialized medicine be doing it already?
    - paulryanrogers 6mo ago
      The US already was, and to since extent still does. Same in the UK and other parts of Europe. Government funds a lot of medical R&D. Thank them for the fundamental research that lead to the COVID vaccine.
- AdamN 6mo ago
  Where and when?
Incipient 6mo ago
That's one reason why privatised health is rubbish. "profitable" treatments should be used, in part, to subsidise the cost of unprofitable ones.
- renewiltord 6mo ago
  [flagged]
  - sokka_h2otribe 6mo ago
    Uhh, you know you could skip the vaccine and just call it a tax..
    - renewiltord 6mo ago
      That wouldn't guarantee subsidization of expensive treatments by cheap ones and therefore is fascist.
- Aurornis 6mo ago
  No medical system, public or private, has infinite money. There will always be decisions made about which conditions get research and which don't. It's unlikely that a disease this rare would be prioritized by a purely government run system, either. There are too many more common diseases to address first.
  - AdamN 6mo ago
    It's a little bit the opposite. Private groups are focused on profits but there are gov programs to support the rare disease research that would otherwise go unfunded in a pure market system.
  - Incipient 6mo ago
    I mean sure, nothing has infinite money...but better to redirect some profits to other treatments, than to extract them all for shareholders?
wjxgxey 6mo ago
pfft just illusion of control theatre for people who are scared of death. Throw in some opportunists exploiting it. Just watch what happens if there are unintended side effects. Its okay to die guys. Everyone does it. The sky doesnt fall.
- zdragnar 6mo ago
  Dementia is a terrible way to go, both for the people who get it and for their loved ones who are with them. One day, my grandmother forgot English when my uncle was visiting and kept speaking in her native tongue and got so mad because nobody understood her. That was one of the few amusing anecdotes from get decline. The rest are just depressing. Watching your father cry because he went to the hardware store and couldn't remember how to get home and had to ask an employee to call his family for him, for example, was particularly tough.
  - wjxgxey 6mo ago
    You know why that happens? Because the health care system slows natural decay rate of some subsystems (via pills/surgeries etc) while having nothing to offer for other subsystems. So rather than all subsystems decaying together we produce this mismatched state.
    - temp_praneshp 6mo ago
      That's the response you have to the parent's anecdotes? I hope that one day you are not sad and angry anymore.
    - zdc1 6mo ago
      You can't really blame the healthcare system for this. Alzheimer's and Dementia existed before modern medicine. The reality is that many fit, active, and otherwise healthy people will hit their 60s and 70s and will experience cognitive decline and Alzheimer's.
      - wjxgxey 6mo ago
david_shi 6mo ago
Seems insane that profitability so heavily dictates what is researched and what isn't.
- silisili 6mo ago
  I don't mean to disagree with you in spirit, but profitability is pretty closely entwined with probability. So companies are chasing solving problems that more people have, even if it's for the wrong reason.
  - ehnto 6mo ago
    As the benefactor of an extremely rare disease, it's not exactly unfair when you look at it from a societal view. If you solve a higher probability problem, you are helping far more people. The real tragedy isn't the allocation of the resources we have spare, it's that so many of our resources are not spare because billionares and corporations have hoarded it. Without changing the percent of allocation, and only changing input resources by capturing it back from billionaires as taxes, we could be helping far more people including super rare diseases.
    - silisili 6mo ago
      Absolutely. And if you take a step back and look at Covid spending, what it was spent on, and how much fraud was involved, it's absolutely maddening that the government isn't instead spending money on solving actual problems its constituents face. We basically just shoveled free money at anyone who claimed to have a business, to no real effect. C'est la vie, I guess.
    - AdamN 6mo ago
      I don't know how much longer it will last but the US government invests significant resources into rare diseases in order to improve outcomes where the normal market wouldn't otherwise support the r&d.
- s1artibartfast 6mo ago
  I find it makes more sense if you drop the corporate analysis and just think about people. Money motivates them and is why they go into hospitals or research labs instead of staying home with their family or friends.

georgeburdell 6mo ago

I know another family like this. One partner still works, but the other one is essentially a full time advocate for an inherited disease that fewer than 100 people in the world are affected by. I don't think much money is involved, but they've changed the narrative about the disease and some researchers are taking them seriously.

the_wolo 6mo ago

If so, good for them, good for the humanity, but what we actually must do is to ~~expropriate~~ ~~socialize~~ democratize the means of production.

CMay 6mo ago
the reason your encouraged approach tends to produce poor results, is that you increase the distance between the decisions that need to be made and the people who understand how to make the decision or whether a decision is even valuable to make. it is basically an unsustainable structure. there's not much value to replacing one structure which you might think is unsustainable with something equally or less sustainable that also produces worse results anyway. another issue is that it can dilute responsibility and someone will take more assertive control anyway which further reduces the quality of decision making. someone still has to enact and enforce the decisions, so whoever does the enacting has to obey and whoever does the enforcing has to enforce the right thing. it's easy to end up with a bunch of people influencing things for their own reasons which have nothing to do with maximizing the production of good results.
onion2k 6mo ago
In this case (and probably lots of others) the amount of resources needed to make an impact on the problem means that it would never work if the funding was decided through a democratic process. As soon as a committee of people are deciding to where to put resources they decide to share it between a number of worthy causes, and that means none of them get the bulk of what's available. If you have something that's percieved to be a relatively small problem by people it isn't directly impacting every day, that needs a lot of resources to fix it, then it's never getting done. (Tech-related side note: This is why companies build mountains of tech debt unless there's a former engineer running the show.)
radu_floricica 6mo ago
Poe's law? Can't really tell, with the internet these days. Things are so polarized that people talk from and to their tribe and the message is often understood to be obvious. Anyways, in case you're serious: there is a famous thought experiment about healthcare: should a hospital administrator approve a complex and expensive treatment to save a 7 year old girl, with a 100% success probability? Or more to the point: is approving this a "good" act, or a "bad" act? The unintuitive answer is that it depends on the opportunity cost long term, and the math is far from obvious. The quick answer is often "not even wrong" - it simply ignores a lot of facts down the line. The same cash can be used very boringly to do maintenance or to buy a piece of life saving equipment. And it gets very dirty very fast. The easy version is pay for the operation vs buy an MRI machine - in this case you can at least compare apples to apples, if you squint - an MRI machine also saves lives. But if the alternative is renovating a waiting room, you're really off the deep end. Because doing it one time it's an obvious decision: just save the life. And it's not even bad as a general policy: have waiting rooms be a bit dusty, if this means spending more money on treatments. But... how much to cut, exactly? And then you have second order effects: throwing a moderate amount to waiting rooms can make them look a lot better, while having it linger in disrepair can make people actually avoid the hospital (if they can't even paint the walls, why would you trust them with your life?). And all of this is assuming the hospital admin actually has mental bandwidth and latitude to make this decision. In practice, he's looking at the kid's mom when he has to turn her down because the operation money would pay for renovating a whole hospital wing. And the mom is an influencer with a large following. > democratize the means of production This means having humans make such decisions, and even worse, it means a committee or a mob will make such decisions. Zero skin in the game, all vibes and feelings. Some girls will get saved, but I've seen how hospitals look in such a system, and it's not good. To note: the US system may or may not be "democratized", but it managed to have exactly the same flaws - most decisions are taken by humans in a bureaucracy. The government simply wrote the rules then outsourced the bureaucracy and the blame to private corporations.