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Comment by xxpor | Hacker News Reader

The world doesn't have unlimited resources. You have to make a call somewhere.

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 measures effectiveness.

If the cost isn’t reflective of real resource/labor consumption, but instead is a rent on IP (which is partially repaying some fixed R&D investment), it’s not so simple.

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.

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.”

From the article: "Libmeldy is approved for use in the European Union and U.K., although the U.K.'s drug price watchdog initially rejected the therapy due to its hefty list price of £2.8 million ($3.4 million at today's exchange rates), BBC News (opens in new tab) reported in 2022. The therapy's manufacturer, Orchard Therapeutics, then offered Libmeldy to the NHS at a significant discount."

So it does seem like they've already discounted it heavily.

"Significant discount" doesn't mean much when they're profiteering.

Do you know that they ended up at a “profiteering” price point, or are you just assuming? And you do have to pay the team of researchers to go on a speculative hunt for a cure for a super low incidence disease, if you want them to do it again in the future. If you then turn around and seize the results, you’re not encouraging them to take the risk next time.

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.

They really aren't that rare, and the reason we don't help all of them is not because of a utilitarian decision not to spend money on public healthcare. If you can get your humanitarianism to move that money from corn subsidies and fighter jets to health spending, more power to you.

> They really aren't that 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.

It didn't occur to me that you were talking about this specific condition, rather than the set of conditions that will be plausibly treated this way.

This isn't actually true. There are thousands of underfunded causes right now, from rare disease research to homeless treatment programs to education of disabled kids.

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.

Doing the procedure has a cost. There must be some pressure to reduce the cost, else it will never reduce.

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.”

And then instead of reading "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment" today, we would have read something like "current regulatory environment precludes research and development expenditures into atidarsagene autotemcel" in some biopharm prospectus a decade ago.

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.

But you can have it both ways because most rare disease research is already funded by governments and nonprofits

This is something people say over and over, I’m not sure of the source.

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.

The USA really doesn't like it when you ignore their patents.

Expect tarrifs, sanctions and trade secrets so you won't be able to reproduce future treatments.

Sounds like a great way for people to just not invest in new therapies.

"Hey, that's a nice life-saving treatment you spent $500M developing. It would be a shame if we just took it from you."

because that is Dirigisme, which we reject for good reasons

This argument ignores the finite resource fact.

No policy decision can change that.

There's certainly enough resources for this and any medical treatment.

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 it. Yes of course the government and the country has enough money to afford it - but NHS is given a fixed budget.

That assume the technology never gets cheaper over time. I would hope it gets better over time as we cure more and more rare diseases.

The price is set to maximize profit. Given so few will benefit, they made the price high since demand is inelastic (up to a point).

That would imply that people are dying right now due to NHS rationing resources, which NHS will never admit to.

Well no, but it could be used to(for example) shorten the ambulance waiting time, and that alone would save more than 1600 people.

If people are dying because of ambulance waiting time, then it sounds like the care is being rationed to me.

No, it would be rationed if you had 100 ambulances and only decided to use 50 for some reason - that's what rationing is. The service is currently at full capacity, but the capacity could always be increased.

I'm pretty sure this has been explicitly stated many many times.

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.

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