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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

#41

Post by CommanderData »

themaninthedark wrote: post #23
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 .

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?

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

#42

Post by gambiting »

mrtksn wrote: post #30
hummus_bae wrote: post #14

Earlier quoted context omitted.

Another country where nationalized healthcare is a bad idea.

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.

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

#43

Post by csours »

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?

Yes, someone somewhere will be making this decision. It could be insurance, a doctor, nurse, hospital administrator, or family member, or even the affected individual.

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

#44

Post by elil17 »

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…

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 on publicly funded research. We could reduce the length of drug patents. There are many, many options besides “role over and let pharma companies charge $4 million/treatment when it costs them $1 million.”

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

#45

Post by wellanyway »

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.

But caaapiiitaliiism

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

#46

Post by elil17 »

sn0wf1re wrote: post #38
elil17 wrote: post #24

Earlier quoted context omitted.

I agree there needs to be pressure. Why not regulatory pressure?

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.”

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

#47

Post by ifdefdebug »

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…

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.

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

#48

Post by nradov »

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?

In the US, medical insurers are not allowed to deny coverage for chronic or genetic conditions due to the Genetic information Nondiscrimination Act of 2008 (GINA) and the Affordable Care Act of 2010 (Obamacare). Some treatments may require proof of medical necessity, or require that providers and patients try lower cost options first (step therapy).

Rules for life insurers are different and in some circumstances they may deny coverage.

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

#49

Post by [deleted] »

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…

[deleted]

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

#50

Post by timerol »

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 treatment will become cost effective without any change in the treatment or it's cost. The posted article agrees that this is the big open question.

> The benefit of the therapy seemed to last several years, but at this point, "it is not yet clear whether it will persist life-long, and extended follow-up is needed," the EMA noted.

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