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.
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.
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.
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.
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.
A metric other than profitability seems like a terrible target for private research which (outside of a charity or cause-driven org) needs to justify its expenses.
In the US alone, we have dozens of grants, programs, and funding sources for things like orphan/rare diseases.
Those seem like pretty good ideas of the kind of targets we should have. But as you mention, those seem to only be considered for cause driven places or charities.
For a private company, QOL for someone doesn’t pay the researcher, laboratory chemical supplier, or lab’s landlord. Money does.
You won’t get a cure or treatment if you can’t discover it, get it approved, or distribute it, all of which costs (a lot) of money.
Inevitably, the bigger the quality of life improvement or more significant the impact = more money, so those measures are already indirectly considered.
It’s only incredibly rare diseases that aren’t receiving active research efforts.
The reason why it's less profitable is because it will help less people. If profitability didn't dictate what is researched, widespread diseases would get less researched and rare diseases - more researched, which would be a net negative.
IMO the issue isn't discovery and research, it's development. Unless companies foresee a good return for buying/licensing/etc rights to treatments, discovered drugs with potential just sit there.
What sucks is when drugs are deliberately not brought to market, but kept in portfolios, because it might impact sales of other existing cashcows. For example, Gilead has a history of staggering the release of new drugs only once their patents expire for similar drugs they already have on the market.
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.
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.
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?
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?
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.
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.
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.
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.
They hit their 60s and 70s because the health care system is good at fixing certain physical issues not bugs accumulating in the brain. The brain just like your OS cant just keep getting patched forever. So currently people just keep patching older wearing out hardware without any software upgrades available.
That would happen even if there was no medicine at all. It's not like in the natural world disease and dying is smooth. Individual systems fall apart and then the rest of the organism dies slowly or quickly.
As much as I love this forum, the one thing I learned to never say is that it's normal and even good that people die (usually on threads about people trying to live forever).
I've never received such hateful responses on any other topic.
Keep saying it. They will get used to it. Just like the earth is round. Thats how they "learn" most things in the first place. Not by discovering it by themseleves, but through repeating what the majority around them say.
There is no we. You can talk about what you accept or dont accept for yourself. Not for anyone else unless you take responsibility for their life if something bad happens. And currently when some unintended side effect happens or quality of life degenates guess what the health care system does? Just gives you more bills and asks you to talk to their lawyers.
Its very easy to talk about "we" have to do this and that. But the test is always who takes responsibility after things dont go the way they think.