No, because other gene therapies like Zolgensma are priced at $2.5M for a single dose and selling quite well. There is a huge market for these types of cures.

Article explicitly mentions price is high because there are no recurring costs, so it actually isn't more expensive than other treatments that may cost hundreds of thousands every year.

Iirc economists have estimated that a human life is valued around 3M$ by society (a couple of years ago, probably higher now), the price doesn't necessarily seem too high.

Doesn't it say 10 years? So you'd need a few more during your life

Because resources are scarce, saving people requires resources, and some people require resources that are too much for society to bare.

If losing your patents in a position like this is a possible outcome, it changes the investment calculus. As it stands, there’s still the possibility that administrations or laws will change and governments or insurance companies will come around to wanting to buy it at a price that recovers the investment, or some other company will believe that they can persuade buyers to buy at a worthwhile price and buy the patents or the production rights off of the current owners. Or maybe some other company will profitably deploy a derivative technology and they can sue to recover some of their investment. For all of these reasons the patents and trade secrets are still worth something. If finding yourself in this position means that you are stripped of your patents and receive nothing, that makes drug R&D an even riskier investment proposition. Being stripped of patents by the government after having bankrolled clinical trials because the government refused to pay the price required to recover the investment would be a brazen show of bad faith and a real stab in the back. The patents will expire in a decade or so anyway, do we really need the government to go out of its way to screw these people out of their last possibility of making back some of the money they spent on this socially valuable work? If we think that it’s so important that this treatment be made immediately available to people at a low price, maybe the government should buy the patents early for the several hundred million dollars that they cost to produce?

Cheers mate :)

In the example we’re talking about here in the article, university research generated the treatment itself, and then a pharmaceutical company spent hundred of millions of dollars conducting clinical trials and bringing the drug to market, including wrangling with regulators and lobbying EU politicians directly to get acceptance of the safety and efficacy of the treatment in spite of the small sample size of the trials due to the extreme rarity of people with this disorder.

So the question of who did the initial research and who funded it isn’t really important to the question of whether invalidating patents left and right will negatively impact the rate at which new drugs are brought to market.

If the government wants the company’s patents and distribution rights, at this point I’m sure they’d be willing to sell everything over to the government at cost. Clearly no government is interested in buying the rights for $200M, any more than they are interested in buying doses for $1M each. The company that brought this treatment to market has clearly gotten utterly screwed here, and the nature of the comments on the article suggest that a lot of people here are disappointed that no governments have intervened to screw them even more for the audacity of thinking that they should be compensated for their work.

Perhaps the lesson here is that maybe seizing their parents for not selling their treatment at a loss isn’t actually a bad idea. Maybe there’s no risk of discouraging pharmaceutical companies from developing expensive treatments for niche conditions, since it’s already clearly a terrible idea, and companies already won’t be making the mistake of ever doing it again.

Oh interesting find. Makes sense

Our economic system requires endless amounts of busy work. If that busywork doesn't exist people will compete for a handful of jobs because they cannot comprehend the idea of doing less work per person.

If that busy work consists of keeping people alive and advance medical science it's at least a better use of human potential than working via door dash.

There are not many charities and grants willing to shell out a million dollars to improve one persons quality of life.

You would still have to raise several hundred millions of dollars of funding to reconduct development and clinical trials for the drug after securing the IP.

Clinical trial data and manufacturing know how cant be leveraged from the original approval.

It is incredibly difficult to get investors to fund a second round of development for a product that made it to market and still failed.

You might say this is an opportunity for the government to step in as investor, but they have little interest, knowing that they could use the same money to save many more lives in other areas.

Because it isn't in their interests. They aren't running a charity.

A good plan should never have "and then people act against their own interests" as a step.

Any patent is expired by now. Still nobody wants to make it.

The lie people tell themselves is that because India does it, everyone could do it without disrupting the rate at which new drugs are researched and brought to market.

As I’ve discussed in other comments, this is why pharma companies have to recover their entire investment off of sales in the first world. The rest of the world is never going to buy their drugs at a price that would make the R&D spending worthwhile. This is why companies sell their drugs for much cheaper in countries where incomes are much lower, and also why drugs produced and sold cheaply in other countries generally can’t be legally imported into the United States. It’s also why claims that drug prices could be lowered in the United States by importing them from countries where they are sold more cheaply are fundamentally ridiculous. The reason drug prices are high in the United States and to a lesser extent in Europe is that they are subsidizing the rest of the world by doing substantially the entirety of pharmaceutical R&D spending in the world.

That might be surprisingly feasible.

https://www.resetera.com/threads/guy-cures-himself-of-lactos...

>And let's not forget that this business often operates with sickeningly huge profit margins.

This is grossly misinformed

Looking at the UniQure Tax filings for 2020, They had 37M in revenue, -122M in R&D expenses, and -42M in general expenses, so they were -125M in the hole that year.

https://uniqure.gcs-web.com/static-files/8f960e6d-c40c-474e-...

I think it makes no sense because you are greatly underestimating the cost to produce and market the drug, even after sunk costs. It could easily be many hundreds of thousands of per dose.

> There's a saying that one should not throw good money after bad, in this case it would seem they are refusing good money because it won't cover the bad.

They are a drug development company, marketing their drugs to national government health services and private insurance companies. This is an iterative game, in game theory terms. Caving and selling at a loss just guarantees that counterparties will refuse to buy at the stated price next time too, and wait for them to cave and sell at a loss. Sitting on the drug until the parents expire and losing the entire investment rather than selling at a loss has the benefit of proving seriousness in future price negotiations.

And the article mentions that the ongoing care costs for someone with this disorder is hundreds of thousands of dollars per year. In the years since they refused the $1M price tag in an attempt to drive a hard bargain, the national governments and private insurance companies have no doubt spent more than $1M per patient they refused to buy a dose for.

Hardly seems like planet-destroying centaur levels of implausibility that they may eventually come around to the idea that $1M was actually a perfectly reasonable price, given the circumstances.

> Also tax writeoffs exist, so I'm not sure what that R&D actually cost them?

I think you have some major misunderstandings about what tax writeoffs are and how they work.

I will not disclose the company name.

With purged I mean exactly this. Purged. 80% was low from their point of view. Don't know how many products they had. Couldn't believe it at all.

Business losses offset profit, rather than taxes owed, and though losses can be carried forward for years to offset taxes, a company does at some point need to turn a profit for the tax benefits of its losses to matter at all. The article mentions that the company took investor money for this, and it seems to be a fairly small pharma R&D company, so I doubt they have some pile of other successful drugs that they are offsetting these losses against.

But moreover, losing money for tax purposes only makes sense in a narrow window dictated by tax rate. Leaving aside tricky confounders like government R&D grants, if your corporate tax rate is say, 10%, and you have an investment opportunity that won’t turn a cash profit but will grow the value of your business by 95% of what you invest, it can make sense to make an unprofitable investment and lose 5% on it over realizing the profits, paying taxes, and losing 10% on it. But for this to make sense you have to have an investment opportunity that already very close to breakeven, corporate tax rates aren’t that high. And just throwing money away to count the losses on your taxes never makes sense.

Well, that's my point. They developed it, but no one can make it now even if it's cheap to actually make (not to pay licensing for it). Probably patents and such. Not a big fan of such kind of methodology for medicine where profits trump benefits for people.