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Really challenging to read about this drug. We as a family benefit from a treatment from NHS equivalent to one of our (good) salaries annually. We're deeply grateful for it.
However £1.79m per dose seems a huge amount - I think I'm more confused about the science of producing something so expensive than concered that we shouldn't spend such an amount on saving young lives.
It's a real tough question. More expensive drugs are amazing for those who benefit but how many more people could be saved if that money was spent on other treatments?
On the other hand, what are the potential cost savings in care needed if that treatment halts the progress of a disease?
Genuinely glad I don't have to make those calls.
It seemed to suggest only a single dose is required per patient, given the expense of healthcare for such patients it probably pays itself back fairly quickly in comparison to treatments they may have otherwise needed.
What price a life?
According to the article ...
The world’s most expensive drug, which treats babies and young children with a rare and often fatal degenerative disorder, will be available this year for the first time on the NHS in England.
Worth it if amongst the babies/young children treated one of them end up as future space explorer etc that leads humanity to greater good.
It's a bit nuts if you start thinking how many lives could be saved in poorer countries with the money used for one dose of that. I know it's not as simple as that, or is it?!
New drugs are always expensive, otherwise no-one would ever invest in research. Give it a decade or two, it'll be free with cornflakes.
What price a life?
Literally the answer is about £20,000-£30,000 per year.
I believe that is the sort of figure that NICE set per Quality Adjusted Life Year. So if you have a disease and a drug can give you an extra year of fully functioning life before you die, they would be pay up to that for your treatment.
When you consider for these children you are buying some 80yrs of extra life, and the NHS would have got a discount too, you can see how it gets justified.
It’s a bit nuts if you start thinking how many lives could be saved in poorer countries with the money used for one dose of that. I know it’s not as simple as that, or is it?!
Why? Why care for other countries? They elected their own government to take care of them so let them be.
7.8 billion world population and you want intervene in their affairs? You want more people on this earth?
Why care for other countries?
Because most of us aren't sociopaths?
Because most of us aren’t sociopaths?
Given who is posting, it's hardly suprising..
Why? Why care for other countries? They elected their own government to take care of them so let them be.
7.8 billion world population and you want intervene in their affairs? You want more people on this earth?
One less of you with that attitude.
I'm ok with the fact that this drug clearly offers a one hit, life transforming, and good value through saved other medical costs.
As I suggested in my post, behind the costs is what's interesting me. Huge development costs, but does the actual drug ongoing cost lots?
Mrs_oab's treatment is costly due to it being an imported, live blood product that requires a lot of work for every 5ml vial. Does a synthetic chemical that this is also cost that amount.
It’s a bit nuts if you start thinking how many lives could be saved in poorer countries with the money used for one dose of that. I know it’s not as simple as that, or is it?!
Agreed when it's giving oldies another 9 months to live in a care home.
Disagree when it's the young, the young everywhere should be prioritized - no matter where they come from.
Literally the answer is about £20,000-£30,000 per year.Literally the answer is about £20,000-£30,000 per year.
That's NICE's rule of thumb but they go higher, sometimes by a lot. Looks suspiciously like the drug co used this when pricing their drug: 50yrs*£30k=£1.5m so justifiable in QALY terms if not necessarily moral ones (as in they could sell it for less and still make healthy profit, but complex area as cos also have to cover R&D costs of drugs that never reach market).
Cleverer people than me have declared it worthwhile which is good enough for me. I'd hate to be the one who decides on things which literally mean life or death.
One less of you with that attitude.
🤣 Don't worry I will not live forever.
Imagine the world population spiral to 20 or 30 billion by that time.
You will have to bid for a tin of sardine. 😆
Cleverer people than me have declared it worthwhile which is good enough for me.
I don't think you've got the hang of the internet.
Imagine the world population spiral to 20 or 30 billion by that time.
Nonsense.
Nonsense.
Consider your/ourselves lucky if you/we don't get to see that. (referring to population)
Does a synthetic chemical that this is also cost that amount.
It's kind of high stakes gambling isn't it? They spend loads on developing lots of stuff, most of which never makes it to market, so they 'need' to make massive profits on the ones that do.
Don’t worry I will not live forever.
Imagine the world population spiral to 20 or 30 billion by that time.
You will have to bid for
a tin of sardine.soylent green
FIFY
The amount negotiated is confidential, but according to the NHS it has been obtained at a substantial discount.
But the NHS isn't paying £1.79m It's obviously still costing a lot of money.
Consider your/ourselves lucky if you/we don’t get to see that. (referring to population)
It won't.
Male fertility rates are plummeting, we'll be lucky to survive as a species at this rate.
Worth it if amongst the babies/young children treated one of them end up as future space explorer etc that leads humanity to greater good.
or maybe stop wasting money on space exploration, which really dont seem to do much for humanity.
then spend all the normally wasted cash on these and other life saving drugs.
Gene therapy treatments like that are both not cheap to create as a drug, but also if you think of the cost of development against the number of potential patients the price becomes clearer.
However a lot of the gene therapy companies are getting into rare diseases as a way of proving the treatments work before moving on to more common diseases so in a way they are a loss leader.
Personally i have no issue with this treatment as if it let's a child a child who likely wouldn't live past 4 live a normal life span then who wouldn't. We have the nhs for that reason.
Zolgensma isn't a traditional drug, it's gene therapy than corrects a genetic defect. It's also like a "one shot treatment" for a terrible disease, spinal muscular atrophy (SMA). SMA affects children and in the most severe form, children only live a few years. Hence if this treatment can give long and mostly normal life then you can see why NICE has passed such an expensive treatment.
Why care for other countries?
I'm trying to work out if this is sarcasm or not, or if the poster is actually that much of a cxxx.
or maybe stop wasting money on space exploration, which really dont seem to do much for humanity.
Space exploration makes all scientists bother to get up in the morning, instead of allowing us to truly believe what we suspect is true - that we're trying to herd a bunch of halfwit cats left of boom - including those of us in healthcare research or pharmaceutical manufacturing.
Space exploration makes all scientists bother to get up in the morning, including those of us in healthcare research or pharmaceutical manufacturing.
great, but flying a 50 billon pound missile to mars dont save lives. good drugs do.
Lots of interesting and useful research comes off the back of NASA work - including environmental knowledge.
You want to save money? Cut the conventional military and keep the nukes, drones and hackers.
Not directly space exploration tech won't save a life, but it will lead to things that probably will.
Plus as said above it gives the incentive to keep exploring
great drugs and cures are made in a lab, not in space.
1. Penicillin – 1942
Penicillin was first developed in 1928, but started to be used in 1942. As the first official antibiotic, it marked a turning point in human history and led the way in the treatment of numerous bacterial diseases. It has been calculated that the antibiotic has saved over 80 million lives and without its discovery and implementation, 75% of people today would not be alive because their ancestors would have succumbed to infection. It has been used to treat a plethora of conditions such as pneumonia and scarlet fever, as well as ear, skin and throat infections. In 2010 over 7.3bn units of penicillin were administered worldwide. However, inappropriate use of the drug has meant that the world is now facing antibiotic resistance, and bacteria are evolving to fight off the drug's effects.
Great medical research is actually done in space as well.

1. Penicillin – 1942
Penicillin was first developed in 1928, but started to be used in 1942. As the first official antibiotic, it marked a turning point in human history and led the way in the treatment of numerous bacterial diseases. It has been calculated that the antibiotic has saved over 80 million lives and without its discovery and implementation, 75% of people today would not be alive because their ancestors would have succumbed to infection. It has been used to treat a plethora of conditions such as pneumonia and scarlet fever, as well as ear, skin and throat infections. In 2010 over 7.3bn units of penicillin were administered worldwide. However, inappropriate use of the drug has meant that the world is now facing antibiotic resistance, and bacteria are evolving to fight off the drug’s effects.
No one GAF about Penicillin anymore, it was a stop gap - we have multidrug resistant bacteria now. You can't do that kind of cheap, easy science anymore.
2. Insulin – 1922
In dividuals with advanced diabetes are unable to produce sufficient amounts of insulin, a hormone involved in the conversion of sugar to energy. Before the advent of insulin in 1922, patients were given a near-starvation diet to ward off symptoms. Insulin is also understood to have paved the way for future hormone replacement therapies and has saved the lives of countless patients diagnosed with diabetes.
Again, low hanging fruit dude. That doesn't prove that NASA/ESA/CNSA/Roscosmos aren't having a significant input to human welfare.
What point are you trying to make with those? Last i could tell space exploration hadn't really started at the time of discovery of penicillin or insulin
A work mate put a post up on Facebook recently saying that his wife with incurable stage 4 cancer had been told by her consultant that she had topped £500k in treatment and that he was aiming for at least 1 million cost. The doctor thought it was worth it and I'm pretty sure her 4 Yr old daughter would too.
Arguing over the cost of these is pointless if nice has approved them.
The costs will drop as technologies improve and evidence increases allowing regulators and scientists to understand more.
Worth it if amongst the babies/young children treated one of them end up as future space explorer etc that leads humanity to greater good.
Or if they just live happy unremarkable lives IMO
+1 AA
Also, I get a warm fuzzy feeling from anything that is essentially a gigantic **** you to the universe.
Science is rock n roll, it's the ultimate rebellion against our own fate.
I’m trying to work out if this is sarcasm or not, or if the poster is actually that much of a cxxx.
Have you not met before?
Worth it if amongst the babies/young children treated one of them end up as future space explorer etc that leads humanity to greater good.
Or if they just live happy unremarkable lives IMO
agree with this. well worth it.
The perceived value seems expensive because it fits in a vial. If it was some sort of complex robotic procedure controlled by CAT/MRI imaging with a team of surgeons and all the associated expert support that cost 50k a pop and needed done every 3-4 years probably wouldn’t seem that expensive (but is about the same in total, potentially at lower risk to the patient).
I’m trying to work out if this is sarcasm or not, or if the poster is actually that much of a cxxx.
Have you not met before?
You will be bidding for Soylent Green then. 🤣
Always want to intervene in other countries' affairs no wonder they don't like you (Developed nations).