[tptacek]

People are fixated on the price for this drug, but the article itself is pretty clear on the rationale for the price tag: current alternative therapies have a price tag of up to $300k/yr, indefinitely. This drug is a one-time therapy. Presumably, any health system would jump at the chance to replace a $300k/yr recurring charge with a 1-time $1MM fee.

But they don't. Health systems in Europe apparently refuse to pay for this therapy. The reason for that, from reading other articles, appears to be that it is of questionable efficacy. Patients report fewer pancreatitis attacks, but clinical indicators like blood fat levels are apparently unchanged. The tiny market and efficacy concerns might explain why the therapy is no longer available at any price.

[refurb]

Actually, single payers in the EU are much better equipped to pay $1M now to save $300k/yr in perpetuity. All medical spending comes from one big pile.

In the US, not people don’t stay with the same health plan for more than 2-3 years. So why would an insurance company pay $1M when they get 2-3 years of pay back, then the next insurer gets a free ride?

I know there is an MIT prof who is pushing the idea of something like a bond. Some external party pays the $1M and the insurer makes X payments until it’s paid off.

A interesting idea that even the single payers like as they have a more predictable budget.

[tehwebguy]

> In the US, not people don’t stay with the same health plan for more than 2-3 years. So why would an insurance company pay $1M when they get 2-3 years of pay back, then the next insurer gets a free ride?

One of the most compelling arguments for single payer which I’ve somehow never heard before now. Biding time in an effort to pass the buck should not be incentivized.

[tptacek]

Yeah, I'm not saying it doesn't make sense or that the EU wouldn't do it. I'm saying they aren't doing it, because the treatment is of unclear efficacy.

[fyolnish]

But that doesn’t have much to do with the price. Your comment sounded like you were saying the eu was being cheap.

[jstanley]

I thought it sounded like quite the opposite: he's not saying they aren't buying it because they're cheap (he points out that if it worked, and they were trying to be cheap, they would buy it). He's saying they're not buying it because it might not work very well.

[ineedasername]

Except as a result of the price tag, they priced themselves out of the market and any possibility of revenue. That is not a rational. And efficacy isn't really in question: Perfect cure or not, it has a vast increase in quality of life including the ability to have children, avoid pancreatitis, and enjoy alcoholic beverages to name a few. If insurance companies are covering replacement therapy to the tune of $300,000k/year, that would seem a pretty good sweet spot pricing that insurance companies would clamor to approve due to the savings over annual costs.

[refurb]

I don’t think they priced themselves out of a market. Uniqure set the list price at $1M. In Europe each country negotiates their own drug price, so the real price would have been even lower than $1M.

I’m going to speculate that the drug just wasn’t that good, so the demand wasn’t there and the program folded.

I know Germany pays close to $1M for a specific hemophilia therapy and they pay that annually. The price itself wasn’t the issue, this it must have been the drug itself.

[ineedasername]

according to the article, and a few google searches outside of it, the drug was that good. The article's implicit conclusion was that the drug was priced too high, and offers evidence to back that claim. You seem to be assuming a perfect rational market here that has a rational justification, but real markets don't always work that way.

[refurb]

As someone who works in the industry, the article was a 30,000 ft view and didn’t do a good job of digging into the issue.

Regardless, it looks like the drug does reduce pancreatitis attacks, but only by 50%. So it certainly doesn’t “cure” the patient.

The clinical trials were also quite small, so possibly the national payers said “$1M is reasonable if you do X, but you don’t have the data to convince me you can do X.”

[InTheArena]

Here is the problem with this statement - it assumes that the drug is worth the maximum amount that they could extract from the customer (insurance companies, patients and governments for it). Nothing says that they couldn't sell it for less, just that they thought they could get this much for it.

[ctdonath]

A thing is worth what another will pay for it.

[ineedasername]

And no one was willing to pay this much for the drug, but the inexplicably decided no revenue was better than lowering the price.

[tptacek]

It's not "inexplicable". uniQure is a going concern with treatments in the pipeline for Huntingtons, hemophilia, and congestive heart failure. They have a finite amount of resources and are allocating them to projects that they're more optimistic about than this one. Glybera is of questionable efficacy and is very expensive to administer (they're on the hook for long-term monitoring for everyone they dose).

It's not at all hard to see why they'd stop production.

[flatline]

How much does it cost to manufacture the drug, and in such relatively low quantities? The article did not say but it pointed to this as part of their decision.

[ctdonath]

"Pay" goes both ways. The manufacturer wasn't willing to pay for a million dollars by making this treatment.

[NickM]

Maybe caving in and lowering the revenue would encourage future customers for other expensive treatments to boycott high prices and hold off for price drops. Refusing to negotiate and forgoing revenue now could be the rational strategy in the long term.

[obmelvin]

Yeah, but the drug also has weak efficacy. Who cares if it is "only" 100k instead of 1M if it do what it claims?

[grecy]

healthcare should never be one of those things.

[DoreenMichele]

"Your money or your life?" doesn't seem like a wonderfully ethical medical question when aimed at folks who probably cannot afford it even if it is a cure, adds decades to their life and you are willing to take installment payments.

[WhompingWindows]

Interesting post. Hopefully some innovators can come through and figure out a way to make it more cheaply. Even if it's expensive now, if humans exists for 100's of years, then eventually the price will come down when this inventor is toast, right? Either that or people will figure out cheaper, DIY ways to do some of this biological manipulation.

[jpalomaki]

Based on article the price is not at 1M because of high manufacturing costs, but because they want to have return on the investment made during the research and development phase.

[InTheArena]

TO be frank, it's not even that. If you read the article, they never priced it based on the what they invested in it, but rather off the the fact that they could replace something else that was expensive with it.

[tptacek]

It's not even clear that this therapy is locked up because of IPR. If you read uniQure's filings, their patents appear to be for treatments for other conditions (notably Huntingtons), and their protection for the LPLD therapy is under "orphan drug" protection.

[ineedasername]

Yeah, seems ripe for someone to pick this up and charge $100k a dose. Insurance companies would look at that as a deal compared to $300k/year for the alternative replacement therapy.

[DoctorOetker]

another issue I have is with the claim from the article that they constantly have to produce new batches because the old product expires. but with such a small group, it would seem better to first make a comprehensive list of LPLD sufferers, then make a single batch for all of them, and repeat every say 5 years as new cases are found... yeah on average a patient waits 2.5 years...

If there is a long list of rare diseases, a company could specialise in manufacturing doses for rare diseases outside of the classical "order a dose (from continuous production) as a patient is discovered". So they might make a specific rare drug once every X years, but with enough rare drugs they might fill the rest of those years with preparing for and then making and testing single batches of other rare drugs...

[platz]

> alternative therapies have a price tag of up to $300k/yr

So prices should all float to the maximum of the most inferior product, so that consumers do not have a choice?

How would you like that if it happened in your favorite consumer category?

That's like saying computer memory should never get cheaper (there is alternative memory priced at much higher rates!), or that electric vehicles should always be priced at the cost of the initial entrants, despite economies of scale

[tptacek]

What are you talking about? The premise of this product was that it cost a fraction of what existing treatments cost, and was far better. That's the opposite of the dynamic you just described.

[platz]

Even most of the folks described in the article did not have an initial reaction of "It only costs a fraction of existing methods!" Instead, their reaction was that the price was set by forces that had nothing to do with the R&D of the drug, and by that measure, it costs far much more that it otherwise could be. When the patent expires, a generic alternative will likely be made for far cheaper.

[tptacek]

What patent are you referring to? Are you sure it's still under patent? Did you read anything about Glybera other than this story? Want some spoilers?

[platz]

I've only read this story on it.

I assumed the patent information based on other commenters, perhaps those were incorrect.

I never watch the trailers before I go to watch a movie.

[ZeikJT]

Here we are discussing the Real Events the movie was based on when all we've seen are the Movie Adaptation and Metacritic.

[tempestn]

The article talks about turning white, fat-filled blood red, so presumably it would have an effect on blood fat level. Is the issue that humans with this disease have no choice but to remain on extremely strict diets, which does a reasonable job of controlling their blood fat level. This treatment (cure, essentially) would alleviate that, but not necessarily change the levels much, since the patients were already (laboriously) controlling them?

[tptacek]

To learn about the efficacy concerns, you have to go read other articles about the treatment, which will come up right away when you Google it because the withdrawal of this treatment from the market was a big story.