you can't just simply state that he's healthy without actually seeing his lifespan
You could have read the article to the third word at least, to learn that the child is a girl.
First UK child to receive gene therapy for fatal genetic disorder is now healthy
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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#172Earlier quoted context omitted.
And then instead of reading "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment" today, we would have read something like "current regulatory environment precludes research and development expenditures into atidarsagene autotemcel" in some biopharm prospectus a decade ago. You can't have it both ways and preventing people from maki…
But you can have it both ways because most rare disease research is already funded by governments and nonprofits
This is something people say over and over, I’m not sure of the source.
Even if it were true, turning a discovery into a distributable, replicable, intervention is the opposite of a trivial task.
And yes, our lovely regulatory environment makes it enormously expensive to study drugs and treatment and to get them approved. Who knows how many lives have been lost by this.
When people talk about for example university research driving technology, I think the practicalities of the real world … aka business aka living in 4dimensional reality … are inaccessible to them. They elide all these pesky details which represent like 98% of what it takes to actually do anything.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#173Earlier quoted context omitted.
Well no, but it could be used to(for example) shorten the ambulance waiting time, and that alone would save more than 1600 people.
If people are dying because of ambulance waiting time, then it sounds like the care is being rationed to me.
No, it would be rationed if you had 100 ambulances and only decided to use 50 for some reason - that's what rationing is. The service is currently at full capacity, but the capacity could always be increased.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#174Glad this girl could get the treatment she needed. But I have to question spending millions of pounds on a single dose of a drug at a time when nurses aren't getting payrises in line with inflation and citizens are dying because there's huge waiting list backlogs. Pretty much everybody I know has given up trying to get healthcare if they need it.
Of course, it shouldn't have to be either/or. Unfortunately, the institution (NHS) has been driven into the ground and we're all paying taxes for practically nothing in return.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#175Earlier quoted context omitted.
>> and inflate >> >> Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply.
So you are actually proposing to raise taxes. The problems is that everyone hate that, and will avoid paying taxes and/or vote for the opposition party.
>> So you are actually proposing to raise taxes
It depends - in the specific scenario of an incredibly expensive drug for small numbers of patients (and other problems with similar dynamics) then tax might not be the best solution. But in other cases yes.
In this particular case the returns would go to successful investors - not historically a money user class prone to causing inflation (quite the opposite). In this case you may find that you don’t need any intervention - they might take their winnings and go speculate on more investments (great!). It may be that conditions are such that they don’t presently fancy investing, perhaps you could offer them inflation tracking risk free gilts/tbills to park in for now. However you would need to be ready for their choice to go on an inflationary non productive consumption spree. In which case - directed taxation should be applied.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#176Earlier quoted context omitted.
From the article: "Libmeldy is approved for use in the European Union and U.K., although the U.K.'s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today's exchange rates), BBC News (opens in new tab) reported in 2022. The therapy's manufacturer, Orchard Therapeutics, then offered Libmeldy to the NHS at a significant discount." So it does seem like they'…
"Significant discount" doesn't mean much when they're profiteering.
Do you know that they ended up at a “profiteering” price point, or are you just assuming? And you do have to pay the team of researchers to go on a speculative hunt for a cure for a super low incidence disease, if you want them to do it again in the future. If you then turn around and seize the results, you’re not encouraging them to take the risk next time.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#177Earlier 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…
Matching involves the now simple (if costly) task of comparing diplotypes at immune system loci involved in self recognition, like the MHC.
What's horrific to me is that matching by race is still a thing! These genes are under diversifying selection. That means that they often do not match the surrounding genetic background. That means that racially driven matching is bonkers.
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#178Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#179https://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 .
Another country where nationalized healthcare is a bad idea.
Yes there's no way the UK would have this therapy /s
Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy
#180Earlier quoted context omitted.
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.
Europe consists of many countries, and one of them is more than happy to take 9% / 5% of your income in exchange for one quite crappy public health care system - think whenever you need to see a specialist, you have to pay with your own money anyway (or wait months, years to get an appointment). If you want private health insurance, you have to fund the super expensive good for nothing public one anyway.
Which country?
In the UK I earn a decent wage (well more than 80% of people in the UK), and last year I paid less than £3k / $3.5k for healthcare which covered me, my wife, my 2 children. It also covers my 70+ year old mother and my 90+ year old grandmother who don't earn enough to pay income tax.
80% of people pay far less than I do.
What type of health insurance can I buy in the US for $290 a month to cover a family with no co-pays.