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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.