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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).
You will be bidding for Soylent Green then.
Maybe cockroach coffee is a precursor to us accepting it 😆
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?
Ok
When you patent a new drug (at the point it's "discovered") you typically get 15 years exclusivity on that patent. After that, the world and his wife can churn it out as a "generic" and sell it at just over cost. People investing in Pharma are acutely aware of this, as you would typically see 99% of your sales evaporate literally overnight at the point a generic becomes available. "Blah blah blah, Neurofen...." is usually how this conversation goes, but that's very much an exception, and by-and-large we are talking about high-value hospital-type drugs here.
The expiry of the patent is obviously a good thing, but it does mean that the pharma company has a finite window in order to: recoup the development costs of the drug, recoup the development cost of all the other drugs that went into trials but didn't make it to market (a huge cost), recoup the cost of manufacture/distribution/marketing etc for this drug, pay for all the other non-profit making functions of the pharma company (HR, safety, QA etc), and (yes) make a profit.
In a nutshell, that's why drugs cost so much.
You also have to consider that generally speaking more data is being required in order to be given a license to sell your drug. This means that trials are both taking longer (eating into your sales window), and costing more - both of which result in more to be recouped.
Its obviously highly variable, but going from discovery to market, usually take 9ish years - leaving you only a 6 year window to sell your drug, therefore the cost per dose needs to go up. There are lots of exceptions to this - so called "orphan" drugs, or other various fast-track schemes.
You also need to consider the market for your drug. An ultra-rare disease obviously limits the amount you are going to sell (hence the price goes up), as does only needing one dose vs a sustained course.
There is (obviously?) a degree of commercial strategy when it comes to setting the price of new drugs. As above, it comes down to "how much is this heath outcome worth to you", which is a deeply emotive question, particularly with children. It's NICE that have to make that judgement - an IMO they do a good job.
I wonder what the total lifetime cost of my diabetic care is (medication, appointments, later life complications...). And if you added it all up and delivered it as a bill when I was born, how many people would think it was too expensive?
^ yes quite. This sort of discussion is always good for weeding out the under-cover bastards in the room.
I wonder what the total lifetime cost of my diabetic care is (medication, appointments, later life complications…). And if you added it all up and delivered it as a bill when I was born, how many people would think it was too expensive?
You're easily worth a fiver mate.
I wish there was an ignore option for chewkw that would be worth 1.5mil
That's interesting about the 15 years to get development costs recouped and profit made.
On top of the 'prevent now Vs fix for the rest of life' costs, this now makes sense.
Thank you for the institution that is the NHS and NICE.
That’s interesting about the 15 years to get development costs recouped and profit made.
It’s usually more like 6 years - the 15 year clock starts from when the drug is discovered and patented - the first 9 years or so are spent doing the trials to generate the data in your marketing authorisation/licence to sell the drug
The positive coming from covid is that trials in agreement with regulators are becoming more risk based and with more risk vs reward/outcome decisions on approval, so hopefully we will see drugs come to market faster with hopefully a corresponding reduction in cost (unlikely with pfizer drugs though)
Some of this has already been covered, but just to add some points:
Why does it cost so much? SMA is an ultra rare disease so the potential patient pool is very small, while the development costs will have been much the same as for any other drug. Rare diseases weren't very attractive for pharma until the European and US regulatory agencies introduced incentives to develop drugs for them which allow 10 years market exclusivity, so they have longer to make bank before generics come in. Cynically, it costs so much because that's what pharma have estimated the market will pay - they have teams dedicated to calculating what price they can charge. Ultimately, it's the same as any business - your product is worth what people are willing to pay for it.
Is the price fair? The headline £1.79M isn't what NHS will pay for it, and I wouldn't be surprised if the discount brings it below £1M - it's pretty common for these discounts to be up to 50%. The issue is that other countries use the UK for benchmark pricing, so the companies want a high headline price for that. The NICE process for determining value does have its issues, but I would say it's probably one of the best in the world when it comes to allocating limited resources fairly. The issue is that companies know the threshold for cost-effectiveness (£20-30k per QALY for a non-rare disease and £50-60k for a rare disease), so they of course pitch their drug price to reach the upper end of that threshold. NICE will have pushed back on the modelling assumptions they used to reach that price, and the process for that is pretty thorough - you often see the committee pushing back on very minor modelling points. Ultimately, I would say that if NICE have decided the price is fair, then it actually is fair.
Is Zolgensma worth it? The data are actually pretty impressive - over 2 years you'd expect 100% of untreated infants with SMA to die, and in the trial I think 100% survived at 2 years (might be ~90%). The issues are the small sample size (~20 I think) and the short duration - how do you know these benefits will last? The other issue is what level of function do these infants reach? I know some pts were basically the same as a healthy child in terms of motor milestones at 2 years, but some weren't. How much value is there in extending an infants life if they will never walk or talk? However, I work with lots of different drugs daily and so many of them are just incremental improvements or 'me-too's' that offer little value - I'd honestly say that Zolgensma is a true innovation, and that we should reward innovation.
Obviously the NHS won't be paying that, but, TBH, I don't see this as money spent on a single life, but repayment for the phenomenal research base behind it, and investment in hopefully multiple applications for the technology in future.
Imagine being able to reverse a genetic defect as severe as SMA! Wow. There ain't half been some clever bastards etc.
It’s a large headline figure, but a rounding error in the NHS expenditure budget. Gene therapy using similar technology to the mRNA vaccines is advancing, first in rare diseases and cancer, then in more common diseases like sickle cell anaemia and perhaps haemophilia. Some of the therapies are now remarkable. Take immune cells out of your body, genetically reprogram them to hunt down cancer cells that have been evading your immune system and use as a therapy, for example.
The contentious pricing is normally for more common disease that will present a significant cost burden. Cancer is one of those diseases, but there are others. I’ve just started taking a generic Metered dose inhaler budesonide. Despite working on a successor nearly twenty years ago that’s easier to use, more potent but offered only an incremental benefit. Outside Infectious diseases, cures are very rare but pricing often reflects this.
I joined GSK just as the first ever NICE decision was published. It was a rejection of the influenza medicine Relenza on the grounds that the nhs would be treating colds and people would get better anyway (it’s also used for hospitalised patients). Skip forward to a pandemic and how we’d love an in the medicine cupboard oral antiviral now for just in time symptom prevention. In fact that was the case in 2009 for swine flu.
Why? Why care for other countries? They elected their own government to take care of them so let them be.
Came here expecting a rant about driving offense sentencing. Glad to see a feel-good story bringing out the best in humanity.
or maybe stop wasting money on space exploration, which really dont seem to do much for humanity.
There are two things you're missing here.
1) The space industry drives science advancements. We have tons of things that we would never have had if it weren't for for Mercury / Gemini / Apollo / [insert space programme here].
2) In terms of a given nation's budget, the spend on NASA et al is buttons.