> 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.
Somewhere in the middle is where most people disagree.
Twenty years ago I had Hodgkins Lymphoma. Obviously the NHS fixed that. This was doubtless quite expensive, it needed a team of experts, expensive chemicals and radiotherapy machinery. But, if they don't they're out a young productive software engineer because Hodgkins kills people, even assuming you're so callous your response is to just immediately put me down like a dog so as to reduce the cost of palliative care or diverting resources from relatives - Whereas since they fixed it that's maybe 30-40 years of economic productivity and ensuing tax income they're getting.
Now, obviously not every such intervention is literally profitable in a financial sense, but I chose my case because even boiled down to simply money this actually can make sense anyway. If you agree it made sense to fix me (and I don't see why you wouldn't) then beyond that it's only about value judgements.
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.
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.
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
The difference in measured in QALYs - quality-adjusted life years. The study attempts to take that into account.
> 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.
Yes, exactly my point. The long-term effects of this treatment are what will make it worthwhile or not. Trying to extrapolate a few years to a full life does not make that an easy exercise.
If only there was a measure that was adjusted for the quality of the year. Some sort of quality-adjusted life year.
Yes, it's not a perfect measure (how could any quantitative measure of a qualitative thing be perfect?).
You can't have it both ways and preventing people from making money means, not surprisingly, they will be very hesitant to spend money researching those areas.
Even if it were true, turning a discovery into a distributable, replicable, intervention is the opposite of a trivial task.
And yes, our lovely regulatory environment makes it enormously expensive to study drugs and treatment and to get them approved. Who knows how many lives have been lost by this.
When people talk about for example university research driving technology, I think the practicalities of the real world … aka business aka living in 4dimensional reality … are inaccessible to them. They elide all these pesky details which represent like 98% of what it takes to actually do anything.
"Hey, that's a nice life-saving treatment you spent $500M developing. It would be a shame if we just took it from you."
Expect tarrifs, sanctions and trade secrets so you won't be able to reproduce future treatments.
You might not like the utilitarian approach but this is how the UK measures effectiveness.
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.”
So it does seem like they've already discounted it heavily.
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.
3,100 cases a year in all of Europe, and 3,600 cases a year in the entire US. That's pretty damn rare really. And it's a genetic condition, which means we should be working to eradicate the problem entirely by screening parents and gene editing ideally making it both rare and increasingly rare from here out.
If you're spending $4 million on this you're either raising taxes again (and government expenditure is already roughly 50% of gdp in most developed European countries, cant go much higher without destroying the thing thats making the taxes) or you're not rehabilitating 5 homeless people, educating 5 special needs kids and discovering a potential new treatment for 1 rare disease.
Let’s suppose a drug company is setting their price to maximize revenue.
Suppose they make the following projections:
They determine that if the treatment price is $10 million above the actual cost of providing the treatment, 5 people will buy it. $50 million total profit.
If the price is set so the profit per treatment is $1 million, 100 people will use it. $100 million total profit.
If the profit per treatment is $100k, 800 people will use it. $80 million total profit.
If the company isn’t factoring in the value of a life saved, they will pick the $1 million price point. If ethicists then just run with that price, they may come to the conclusion that the treatment isn’t cost-effective. However, they are relying on data that’s an output of a process with conflicting values, and that pollutes the result of their calculation. Garbage in, garbage out. The 700 people who didn’t get treated lose out for a pretty bad reason.
We could imagine a policy where the drug companies are mandated to maximize lives saved when setting prices. One might argue that companies won’t develop as many drugs if profits can’t be maximized. We could adjust the policy to subsidize companies for income lost when setting lower prices. Ie. if the drug company picks the $100k price point to save 700 more lives, the government gives them $20M compensation so they can profit like they would have at the $1M price point. That way society spends the same amount of money on this drug, but more lives get saved. I’m sure there’s a lot of challenges in designing a program like that, but the opportunity to save lives makes it seem worthwhile to attempt.
No policy decision can change that.
I suspect we can somehow find enough pennies in the couch cushions to get those kids a therapy, especially if it's curative.
People are dying due to resource rationing and it occasionally makes the news.
Healthcare is expensive and a large proportion of taxes go to it.
That's the same sort of ethical calculus that goes into deciding the distribution of any life-saving resources.
We can always spend more (resources/social capital) to save more lives/years of life. How do you decide where that line gets drawn?
Most medical systems decide it by looking at the cost of treatment (Dollars, organs, risks), and quantifying years of quality life gained. If you'd like that bar raised, increase the tax rate/insurance costs.
'Save every life at any cost' is not compatible with a world where you have decide whether your budget goes on a low-ROI, or a high-ROI intervention. 'First come-first-serve' is not compatible with a world where you are optimizing for overall positive outcomes.
Do you have any alternative guiding principle for where money in the healthcare system should be spent?
Compromises always have to be made because the world does not have infinite resources. Unfortunate, but true.
One way to allocate resources between all our competing needs is by using money.
You can come up with other systems of allocation, however all systems will be unfair and arbitrary in surprising ways. There is always a need to choose between ugly choice A and ugly choice B, because our resources are constrained.
Rules for life insurers are different and in some circumstances they may deny coverage.
If given more money then that value can go up, but the UK economy is in trouble enough as it is with the state taking half of GDP.
You have X amount of dollars. You can spend Y percent on healthcare. How do you spend Y to have the greatest benefit? Save 1 patients with a $300,000 gene therapy, or save 1,000 patients with effective treatment for their diabetes.
Until we have unlimited resources, that trade off will always be made.
Look at the Blue Cross Blue Shield plan for Michigan. It covers a number of gene therapies at a cost of $2.2M for one of them.
Not all European countries pay for it.
https://www.bcbsm.com/amslibs/content/dam/public/employers/d...
The economics would never work out unless premiums were sky high or the insurance is mandatory or across most of your population.
It only works if premiums are spread across a large population so a $2.5M therapy becomes a $1 increase in premium for 2.5M people.
Best of the both worlds.
So, depending on where you are, private healthcare means the government pays their share and you pay the rest through an insurance or directly. Again depending on where you are the insured is often provided by your employer, so if you happen to get ill you can have your treatment in a luxury.
In the UK I earn a decent wage (well more than 80% of people in the UK), and last year I paid less than £3k / $3.5k for healthcare which covered me, my wife, my 2 children. It also covers my 70+ year old mother and my 90+ year old grandmother who don't earn enough to pay income tax.
80% of people pay far less than I do.
What type of health insurance can I buy in the US for $290 a month to cover a family with no co-pays.
>>you have to fund the super expensive good for nothing public one anyway.
My father had battled cancer in Polish oncology hospitals and has received nothing but top tier treatment wherever he went. What's more, NFZ completely funded the treatment with then-experimental Glivec for him, which was billed at around 200k zł/year(in US, a year of Glivec treatment is around $100k). In the course of 8 years of cancer he had to stop working due to the ilness, and yet was still fully covered and received the same treatment as always.
So no, it's not "expensive but good for nothing". It's not expensive compared to private treatment, and it's extremely good if you have a major ilness. I agree that routine treatment and appointments are in shambles, but that's universally true pretty much everywhere in Europe right now, the waiting times in countries with public healthcare systems have inflated into infinity due to a combination of factors. But even in Poland, I wouldn't dare to not pay into the system - it's too good not to pay for it(and I do, in fact, pay for it).
> but that's universally true pretty much everywhere in Europe right now, the waiting times in countries with public healthcare systems have inflated into infinity due to a combination of factor
I have heard that's not the case in Germany and the private appointments are almost not a thing. I could be wrong though.
https://www.icalculator.info/poland.html
Says that people pay 1.25% in health insurance.
Poland in general pays 6% of its GDP on health care. The US spends 3 times that.
If you buy 1600 doses of it at a time they're going to sharpen the pencil for you.
Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply.
It’s no more (or less) complicated than any of the thousands of other fiscal decisions a government makes every year.
There’s a fallacy alluded to in the statement “it’s a very complicated problem” - the allusion is to one of inaction being a safer default choice but nothing about inaction in the face of a complex problem guarantees against making things worse
By qualifying with “complex” problem i mean a problem that’s as complex as choosing what to fund in a national budget. A problem for which no answer can be proven correct. A problem which can only be addressed by taking a considered view on risk. A problem with unresolvable uncertainties.
And if you print money and inflate then the price of goods rises. So sure, you might only pay a bit more for the treatment but now fruits are more expensive and your population gets slightly more unhealthy.
>> >> Inflation caused by excess money supply (NB: not inflation caused by excess money velocity) cannot survive taxation. Taxation destroys money supply.
It depends - in the specific scenario of an incredibly expensive drug for small numbers of patients (and other problems with similar dynamics) then tax might not be the best solution. But in other cases yes.
In this particular case the returns would go to successful investors - not historically a money user class prone to causing inflation (quite the opposite). In this case you may find that you don’t need any intervention - they might take their winnings and go speculate on more investments (great!). It may be that conditions are such that they don’t presently fancy investing, perhaps you could offer them inflation tracking risk free gilts/tbills to park in for now. However you would need to be ready for their choice to go on an inflationary non productive consumption spree. In which case - directed taxation should be applied.
I think a big problem is you don't currently realise just how many different worthy causes are currently underfunded. At a guess you could probably spend a couple of trillion a year in the UK before you've exhausted most of the nice things that could be done. There are sooooo many good things to do and only so many person hours (which is what money pays for mostly) to do them.
If you did scrap the defence budget then the money shouldn't go on treating these kids but instead should be invested in the surgery backlogs, ambulance wait times and the justice system trial backlog.