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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

#71

Post by jiggawatts »

timerol wrote: post #50
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 .

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 up with a delta and compute based on that.

Reminds me of the studies that showed that Tamiflu is ineffective because it only reduced the duration of influenza symptoms by 1/2 a day on average. Yes, that's true, I've taken it myself and the symptoms continued for about the normal period of time. But it reduces the severity massively. It's like a light switch. The most severe flu suddenly turns into the mildest of mild cold-like symptoms in a matter of hours. But... that's hard to measure objectively, so it is not recommended because the according to a metric that doesn't matter it doesn't work.

The statistics and metrics in medical papers are woeful, which is why it's commonly accepted that 1/2 of all medical research is false.

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

#72

Post by ericd »

elil17 wrote: post #44
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…

I do like the utilitarian approach, I just think sometimes you need to look outside the box of do A or don’t do A. If the only options were pay for the treatment or don’t, then Ireland might be making the right call by not paying for it. Ireland could simply let a local company violate the drug patents. A country like the UK could impose conditions such as profit caps on pharmaceutical companies who base their work o…

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've already discounted it heavily.

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

#73

Post by piyh »

meese712 wrote: post #59
bglazer wrote: post #21

Earlier quoted context omitted.

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…

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 system where I'm not needed is a better one. The best part is no part.

https://ashpublications.org/blood/article/104/12/3501/89040/...

>Previous studies have shown that 30% to 70% of all patients surviving beyond 100 days after HCT require treatment for chronic GHVD,2,4-6 often for more than 2 years.

Re: a better system is where you don't need donors:

https://www.cbsnews.com/newyork/news/l-i-woman-dies-after-ma...

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

#75

Post by vondur »

CommanderData wrote: post #41
themaninthedark wrote: post #23

Earlier quoted context omitted.

And this in turn leads people to be against national health care as they are worried that the government will be the one making the decision that it is cheaper to let people die rather then treat them.

Don't some insurers outright deny cover for chronic or genetic conditions?

People tend to react differently when the learn it's a government bureaucrat making decisions on healthcare. However, people will also howl when they find out it the decision from a cruel penny pinching CEO. These are really difficult problems. How much is too much to save a life?

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

#77

Post by meese712 »

piyh wrote: post #73
meese712 wrote: post #59

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…

I was getting at the article giving the impression this somehow cured the disease vs a BMT just slowing progression. I know the whole transplant thing sucks. I've had two transplants, thank you for being on the list :).

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

#78

Post by louthy »

jiggawatts wrote: post #71
timerol wrote: post #50

Earlier quoted context omitted.

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 statistics and metrics in medical papers are woeful

Ok, this probably needs some extraordinary evidence …

> which is why it's commonly accepted that 1/2 of all medical research is false.

The irony

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

#80

Post by [deleted] »

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?

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