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First UK child to receive gene therapy for fatal genetic disorder is now healthy

livescience.com

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Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#51

Post by lazyasciiart »

ifdefdebug wrote: post #47
krona wrote: post #25

Earlier quoted context omitted.

In quality-adjusted life years (QALYs), there is an upper limit to the cost of any intervention because the most you can save from one intervention is one life. If the same money can be used to substantially improve the lives of 100 people with other interventions, then the cost-utility analysis may say a particular intervention is not effective. You might not like the utilitarian approach but this is how the UK meas…

Utilitarian cynism at its best. The humanitarian approach is to save that one live AND improve the other 100 as well. We can afford to do so, because those expensive cases are rare.

They really aren't that rare, and the reason we don't help all of them is not because of a utilitarian decision not to spend money on public healthcare. If you can get your humanitarianism to move that money from corn subsidies and fighter jets to health spending, more power to you.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#52

Post by fshbbdssbbgdd »

krona wrote: post #25
elil17 wrote: post #11

Earlier quoted context omitted.

Governments and non-profits already funded the development of gene therapy. Letting private companies charge money for it and then blocking people who need it from getting it is a policy decision.

In quality-adjusted life years (QALYs), there is an upper limit to the cost of any intervention because the most you can save from one intervention is one life. If the same money can be used to substantially improve the lives of 100 people with other interventions, then the cost-utility analysis may say a particular intervention is not effective. You might not like the utilitarian approach but this is how the UK meas…

If the cost isn’t reflective of real resource/labor consumption, but instead is a rent on IP (which is partially repaying some fixed R&D investment), it’s not so simple.

Let’s suppose a drug company is setting their price to maximize revenue.

Suppose they make the following projections:

They determine that if the treatment price is $10 million above the actual cost of providing the treatment, 5 people will buy it. $50 million total profit.

If the price is set so the profit per treatment is $1 million, 100 people will use it. $100 million total profit.

If the profit per treatment is $100k, 800 people will use it. $80 million total profit.

If the company isn’t factoring in the value of a life saved, they will pick the $1 million price point. If ethicists then just run with that price, they may come to the conclusion that the treatment isn’t cost-effective. However, they are relying on data that’s an output of a process with conflicting values, and that pollutes the result of their calculation. Garbage in, garbage out. The 700 people who didn’t get treated lose out for a pretty bad reason.

We could imagine a policy where the drug companies are mandated to maximize lives saved when setting prices. One might argue that companies won’t develop as many drugs if profits can’t be maximized. We could adjust the policy to subsidize companies for income lost when setting lower prices. Ie. if the drug company picks the $100k price point to save 700 more lives, the government gives them $20M compensation so they can profit like they would have at the $1M price point. That way society spends the same amount of money on this drug, but more lives get saved. I’m sure there’s a lot of challenges in designing a program like that, but the opportunity to save lives makes it seem worthwhile to attempt.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#53

Post by [deleted] »

jiggawatts wrote: post #5

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 .

[deleted]

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#54

Post by pc86 »

elil17 wrote: post #46
sn0wf1re wrote: post #38

Earlier quoted context omitted.

What sort of regulation? Make it cheaper by x% per year or we stop using it? Doesn't sound that dissimilar to "make it cheaper than the total cost of the current treatment or we won't use it".

I’d just change the “or we won’t use it” to “or your patents go away.”

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 making money means, not surprisingly, they will be very hesitant to spend money researching those areas.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#55

Post by Waterluvian »

shadowgovt wrote: post #27
cameronh90 wrote: post #12

Earlier quoted context omitted.

What if the cost was $100 trillion?

Its sticker price is actually $3.8 million. At that price, it could be administered to every child in the UK with the disease at about the cost of 1 year of the UK's defense budget.

The thing that tires me about this old chestnut is that it’s just a shell game and not actually a substantive idea. “Just take money from somewhere else.” It’s akin to saying that we will use the latest AI technology and machine learning to make our value proposition work. It sounds satisfying but it says nothing.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#56

Post by A4ET8a8uTh0 »

gambiting wrote: post #35
ls612 wrote: post #15

Earlier quoted context omitted.

[flagged]

That's such a reductive, dismissive take on the issue it's actually offensive towards everyone in this country trying to make the healthcare system work. The government isn't ran by some cartoon villains, and the resources at NHS's disposal are finite - I certainly don't envy anyone whose job it is to make sure they are allocated in the most efficient way that also saves the most lives.

I will make a short quip here. All cartoon villains I saw appear to be fairly capable administrators. If government was ran by one of those, we would likely see an improvement. I am not sure who is in charge anymore, but I can agree with you that it is not cartoon villains.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#57

Post by linuxftw »

gambiting wrote: post #29
shadowgovt wrote: post #20

Earlier quoted context omitted.

There are, on the high end of the estimate, 1,600 kids in the UK that might have this disease. It's estimated about 5 are born per year. I suspect we can somehow find enough pennies in the couch cushions to get those kids a therapy, especially if it's curative.

The problem is, as always, with allocation of resources. If you are running NHS budgets and these treatments cost £1M each(we don't know what price was agreed in the end, but let's say it's £1M per treatment), that's £1.6 billion to treat 1600 kids. £1.6 billion is a lot of money that can save a lot of more than 1600 people if used for other therapies. It's a horrible choice to make of course, but it's the reality of…

That would imply that people are dying right now due to NHS rationing resources, which NHS will never admit to.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#58

Post by CraigJPerry »

Waterluvian wrote: post #55
shadowgovt wrote: post #27

Earlier quoted context omitted.

Its sticker price is actually $3.8 million. At that price, it could be administered to every child in the UK with the disease at about the cost of 1 year of the UK's defense budget.

The thing that tires me about this old chestnut is that it’s just a shell game and not actually a substantive idea. “Just take money from somewhere else.” It’s akin to saying that we will use the latest AI technology and machine learning to make our value proposition work. It sounds satisfying but it says nothing.

Why would you need to take money from somewhere else? This is misunderstanding how money works for a currency issuer.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#59

Post by meese712 »

bglazer wrote: post #21
SamBam wrote: post #4

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…

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 or radiation to make sure the fixed cells propagate more than the diseased cell line.

Edit: read the study, they do give them the same chemo used for normal transplants.

Re: First UK child to receive gene therapy for fatal genetic disorder is now healthy

#60

Post by janeway »

amelius wrote: post #7
SamBam wrote: post #4

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?

Also, when cells replicate, will the new ones automatically be of the edited type? Or is it not that simple?

That is the goal but not that simple.

In most cases, the original disease-causing stem cells are effectively eliminated by the conditioning regimen (high-dose chemotherapy and/or radiation) used before the (autologous) transplantation, so they do not regenerate and cause further harm to the patient. However, in some cases, residual disease cells may survive the conditioning regimen and persist after transplantation, leading to disease relapse. This is more likely to occur in patients with aggressive or refractory disease or in those who receive a mismatched or haploidentical donor graft. To prevent disease relapse, post-transplantation maintenance therapy, such as immunosuppressive drugs or targeted therapies, may be used to eliminate residual disease cells. Additionally, close monitoring of the patient's blood counts and immune system function can help identify and manage any signs of disease relapse.

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