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https://en.m.wikipedia.org/wiki/Atidarsagene_autotemcel > The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment." Wow… instead of a lifesaving cure they recommend the treatment of the symptoms until the kid dies because it’s cheaper .
The article was skimpy on the details. Can someone explain how this part works? > The new gene therapy [...] works by inserting into the body working copies of the genes that are faulty in MLD, thus restoring the ability to break down sulfatides. How does the new copy of the gene get into every existing cell that needs it? A virus?
Yes, it's a virus, specifically a lentivirus, which inserts its own genome into the cells DNA, including the therapeutic gene. Looks like they first extract bone marrow from a patient, apply the virus which adds the gene, then they put the "fixed" bone marrow cells back into the patient (probably after killing most existing bone marrow through some really unpleasant procedures). The re-inserted, treated cells then ex…
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Why would you need to take money from somewhere else? This is misunderstanding how money works for a currency issuer.
It's a very complicated problem, but just printing money usually cause inflation. There is no free lunch.
Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply.
It’s no more (or less) complicated than any of the thousands of other fiscal decisions a government makes every year.
There’s a fallacy alluded to in the statement “it’s a very complicated problem” - the allusion is to one of inaction being a safer default choice but nothing about inaction in the face of a complex problem guarantees against making things worse
By qualifying with “complex” problem i mean a problem that’s as complex as choosing what to fund in a national budget. A problem for which no answer can be proven correct. A problem which can only be addressed by taking a considered view on risk. A problem with unresolvable uncertainties.
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In Europe they have these things called planes, it's like a tube you enter and sit for a few hours and once exit you are in a driving distance to American hospitals where you can pay and receive the same treatment as everyone else without nationalised healthcare. Best of the both worlds.
What's more, Europe also has these private hospitals which aren't connected to the national healthcare system and which will provide you with top level care if you are willing to pay, no travel to US necessary. Private health insurance ( gasp! ) is also a thing, should you want to partake in that system.
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I'm confused how this would reduce disease progression compared to a much cheaper allo bone marrow transplant then if they are only modifying hematopoietic stem cells since a BMT is just doing the exact same thing except with someone else's non affected cells. BMTs are a horrific procedure though so this definitely has an advantage in that regard. I would also have to imagine they would have to do myeloablative chemo…
Most recipients don't need to take immunosuppressants at all if they get PBSC or bone marrow transplants. Even if they do, it's short term. Additionally, the allo grafts need to be matched, which if you're non white is not a good success rate. 85%ish of whites get matched, that number gets depressingly low for minorites. On the US registry, only 1 in 400 donors get called. I happen to be one of those donors and a sys…
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In Europe they have these things called planes, it's like a tube you enter and sit for a few hours and once exit you are in a driving distance to American hospitals where you can pay and receive the same treatment as everyone else without nationalised healthcare. Best of the both worlds.
What's more, Europe also has these private hospitals which aren't connected to the national healthcare system and which will provide you with top level care if you are willing to pay, no travel to US necessary. Private health insurance ( gasp! ) is also a thing, should you want to partake in that system.
Earlier quoted context omitted.
I'm confused how this would reduce disease progression compared to a much cheaper allo bone marrow transplant then if they are only modifying hematopoietic stem cells since a BMT is just doing the exact same thing except with someone else's non affected cells. BMTs are a horrific procedure though so this definitely has an advantage in that regard. I would also have to imagine they would have to do myeloablative chemo…
Most recipients don't need to take immunosuppressants at all if they get PBSC or bone marrow transplants. Even if they do, it's short term. Additionally, the allo grafts need to be matched, which if you're non white is not a good success rate. 85%ish of whites get matched, that number gets depressingly low for minorites. On the US registry, only 1 in 400 donors get called. I happen to be one of those donors and a sys…
Hmm...
How does this translate to other places? E.g. can you only match Han in China?
Do Italians and Scots match? Turks and Egyptians?
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Here's the study: https://www.ncpe.ie/wp-content/uploads/2021/04/Libmeldy-Bene... It's worth mentioning that the study in Ireland indicates that the treatment extends life by 14.49 QALYs (average "Total Life-years" moved from 8.92 to 22.74), which is a long way from a cure. If this is truly a cure, and the treated population lives a full life (life expectancy in Ireland is current 82 years, not 23), then this treatme…
They can't possibly know that the drug extends life to "22.74" years, because it has only been approved for use for the last 3 years! This is like asking for 30 years of Kubernetes experience on a job application. Even if the estimate is accurate, there is a massive qualitative difference between slowly dying horribly for 'x' years and living a normal life for 'y' years. You can't just subtract 'x' from 'y' and come…
The difference in measured in QALYs - quality-adjusted life years. The study attempts to take that into account.
> They can't possibly know that the drug extends life to "22.74" years, because it has only been approved for use for the last 3 years! This is like asking for 30 years of Kubernetes experience on a job application.
Yes, exactly my point. The long-term effects of this treatment are what will make it worthwhile or not. Trying to extrapolate a few years to a full life does not make that an easy exercise.