18 ms·

A million-dollar drug

298 points by rpledge 8y ago

charlesism 8y ago
"Well, one pharma company has the patent, and we need to let them charge what they think the market will bear. Sorry LPLD carriers, you have to die now." Apparently, this is the best the world can manage in 2018.
- miguelrochefort 8y ago
  I don't understand how anyone can think patents are a good idea.
  - jmcgough 8y ago
    Not a fan of it either, but how do we incentivize companies to research and create new drugs if they can't make back the huge cost of clinical trials?
    - carbocation 8y ago
      What if governments paid for the clinical trials?
      - NikolaeVarius 8y ago
        The article has a section that talks about the difficulty of getting the drug even approved for testing by governments in the first place.
      - sneak 8y ago
        The government is the whole reason the clinical trials are required before patients can be treated with a working drug. They are the cause of the problem, not the solution.
      - longerthoughts 8y ago
        The government does frequently fund the discovery of new drugs. The problem is that the return on that investment is usually abysmal and the taxpayer ends up not only paying to fund the drug’s discovery, but also paying absurdly high prices to buy it from pharmaceutical companies who passed some or all of their risk onto taxpayers. One example: https://abcnews.go.com/WNT/YourMoney/story?id=129651 https://abcnews.go.com/WNT/YourMoney/story?id=129651
        carbocation 8y ago
        Of course. Most of basic science in the US is government funded. I'm wondering what the OP thinks about removing the barrier that they identified (private corps paying for the trial vs gov paying for the trial) and how that would affect their thinking.
        umanwizard 8y ago
        If you read the article, the drug was in fact discovered at UBC, which is a publicly funded university. But it turns out that discovering the drug wasn't the expensive part. The expensive part was privately funded.
    - latch 8y ago
      Did you read the article? The company that charged that absurd price acquired the patent, they had nothing to do with developing it.
      - mabbo 8y ago
        While I don't disagree with your point, the company that bought the patent paid a lot of money for it from the company that developed it. Patents being exchangeable is not necessarily a bad thing.
    - miguelrochefort 8y ago
      How do you incentivize environmental research? How are we supposed to find solutions to climate change if they can't be patented? Copyrights are just as bad as patents, and musicians/writers shouldn't make people pay for their music/books. We should start giving money to causes we support, whether it's art, medical research, software projects, etc. It's a mistake to think we should only give money if we're forced to by law (through pricing something that's not scarce).
  - worldsayshi 8y ago
    There should at the very least be a law that states that a patented technology should be made available in reasonable ways to make the patent valid.
    - umanwizard 8y ago
      Would you rather have this drug exist and be patented, or never have existed at all?
      - zaptheimpaler 8y ago
        I see you arguing that things went just the way they should have all over this thread. This point in particular is completely wrong. The two doctors who made the drug do not even hold the patent and basically gave it away for free to the company trying to distribute it. So yes, if not for the patent this drug would definitely still exist, and probably be available to people who actually freaking need it.
        umanwizard 8y ago
        > I see you arguing that things went just the way they should have all over this thread. Actually, I never said that anywhere. I think what happened is basically a no-op. Without a profit motive, the drug would never have been developed, and therefore not available to anyone, and we'd be in the same boat we're in anyway. What I do think is one of two things should happen: Either (1) we decide that the drug is worth it, in which case health systems should pay the owner of this drug fairly for their investment. Or (2) it's not worth it, in which case we're no worse off than we would have been without this company, other than the fact that they wasted a bunch of their money. (As an aside, note that I said we should pay the owner, fairly for their invention, not the original developer who voluntarily gave up the rights. This is an important distinction as the ability to voluntarily transfer property rights is pretty central to the whole concept. Imagine if after you bought a car, anyone could take it, because you're not the original manufacturer. It's not hard to see the path from this society to one where nobody bothers making cars.) > The two doctors who made the drug do not even hold the patent and basically gave it away for free to the company trying to distribute it. Those two doctors did the least expensive part of the whole operation. The more expensive part was funded privately. If you want to fix this you can either have the government fund that expensive part, or you can have it done privately and pay the owners what they want. > if not for the patent this drug would definitely still exist, and probably be available to people who actually freaking need it Sure, maybe the doctors still would have done the research, but there would be no money either for the clinical trials, and so manufacturing and distributing the drug would be illegal.
  - charlesism 8y ago
    I don't have a problem with the idea of patents. I just think we could be a whole lot more pragmatic about them. They're supposed to incentivize useful behavior.
    - miguelrochefort 8y ago
      Who should decide what's a useful behavior? What's a fair price? What's a reasonable lroduction/distribution strategy? I think the free market should decide, not the government and its enforced monopolies.
  - umanwizard 8y ago
    Reading the article has convinced me that drug patents in particular are a great idea. Isn't it wonderful that we live in a world where futuristic new treatments like this can be developed? Yes it's true that people can't get the drug now, but at least someday it might be available. Without patents, who would you find to work for free developing something like this?
    - miguelrochefort 8y ago
      > Isn't it wonderful that we live in a world where futuristic new treatments like this can be developed? This futuristic new treatment can no longer be developed or produced by ANYONE in the world, without the consent of this company. Before the patent, anyone could. Do you think that's not a problem? We'll incentivize researchers the same way we'll do with musicians and writers in a post-copyright society. People who think this disease is important will crowdfund the research, and those who want to receive experimental and personalized treatment will pay for it. How do you think we should incentivize things like environmental research? Is our knowledge about climate change patentable? What if someone found a cure?
      - umanwizard 8y ago
        > Before the patent, anyone could Anyone could, but nobody did, or would have without either a profit motive or massive state funding. > Do you think that's not a problem? Well it’s certainly not the most ideal thing you can imagine, but I don’t see a better way in a capitalist society to incentivize medical research. Do you? > People who think this disease is important will crowdfund the research I’m not aware of any crowdfunding campaign that has raised hundreds of millions of dollars. > How do you think we should incentivize things like environmental research? It should be funded by the state > What if someone found a cure? That’d be awesome. We should pay them incredibly handsomely for their work.
- cratermoon 8y ago
  Late-stage capitalism in a nutshell.
- lisper 8y ago
  The last sentence of the article sums it up pretty well: "If it's not commercially viable to produce a certain therapy, unfortunately, in our Western society, it does not happen." And it is far from clear that this is not as it should be, because it's not just LPLD carriers about whom tear-terking stories can be told. $1M can be deployed in other ways that could potentially save more lives. Instead of "Sorry LPLD carriers, you have to die now", it might be, "Sorry, 100 poor African children without access to clean water, you have to continue to die of dysentery so we can save this one LPLD carrier."
  - thomastjeffery 8y ago
    Except $1 millon is not the entire economy. It's easy to hide resources by equating them to their monetary value. The fact is that there exist many millions of dollars, and the way each dollar is spent does not determine the way another dollar is spent.
    - lisper 8y ago
      So what? Dollars are still proxies for finite resources, and there are only three possible ways to procure them: their owners can give them voluntarily, they can be forcibly seized by the government through taxation, or the government can print them, which produces inflation and effectively seizes a little bit from everyone who holds dollars (essentially a tax on savings). If you can't get someone to voluntarily spend $1M to save a life, which of the other two processes do you propose to use?
    - tptacek 8y ago
      This isn't complicated. Say there are 1,000 LPLD patients in North America (a wild overestimate). Then treating them with this therapy costs us $1Bn. If we limit ourselves to health care problems, is $1Bn better spent on these 1,000 LPLD patients, or on increasing penetration of existing therapies for other conditions? It seems likely to be the latter. To believe LPLD therapy objectively deserves the allocation you're talking about, you have to believe that there aren't other cohorts of patients that are currently underserved. But we know that isn't true.
  - 8y ago
ttul 8y ago
This is what we call “market failure”.
gfodor 8y ago
in society, we have another large, one-time purchase people often make in which there are special financial arrangements and rules: buying a home. it seems to me that gene therapies (high priced, one-time use drugs) are going to warrant alternative financial instruments. there's absolutely no reason this drug should have failed when the amortized cost over 10 years is less than an inferior product. regardless of the other questions this is a pure market failure by the financial industry. edit: the downvotes probably assume I mean the patient is going to pay out of pocket. of course not. the insurance companies would be paying. the only inferiority between gene therapies and non-cures is their pricing structure. we invented a solution to this problem thousands of years ago: debt.
- wrong_variable 8y ago
  The problem is with a house / car / TV / phone you can always reposes it.
  - nradov 8y ago
    We have loans for education even though lenders can't repossess knowledge.
  - gfodor 8y ago
    It just backs out to interest rates commensurate with the risk. Lack of collateral doesn't mean you can't get a loan. Perhaps the economics wouldn't work, but it sure sounds like nobody tried in this case. What it would boil down to is if the insurance company expected a larger net cost on a traditional drug regimen for the life of the patient, vs servicing a loan at the market rate over N years. Interest rates are still very low so in general debt is seeking out risk.
    - ThrustVectoring 8y ago
      Insurance companies don't cover total health costs over the life of the patient. They cover the next month of health costs, and a certain percentage of their customers churn afterwards. For insurance companies, providing treatment that lasts a longer amount of time can often be a terrible business decision - if the customer switches insurance providers afterwards, then they're essentially subsidizing the insurance company they switched to. This more commonly happens with prescription quantities. Medication compliance is far better when people can pick up a three-month supply of medication. Insurance companies want patients to pick up one month supply instead, so that they aren't paying for two months of medication for every patient who switches insurance companies.
apsec112 8y ago
This seems bizarre to me: Van Deventer says the company never considered lowering the price. "Why would we? Pricing shouldn't be a political decision. It should be a rational decision based on merits and value." If no one will pay $1 million, your revenue is $0. Assuming the drug costs much less than $1 million to make, surely any revenue number is better than $0?
- simion314 8y ago
  What I think is happening is that the number of affected people is so low and the cost of producing this small number is very large and the company does not want a profit but a BIG profit.
  - worldsayshi 8y ago
    If a company does not make use of a patent they should at least loose their right to it.
    - torpfactory 8y ago
      Especially for lifesaving medical patents. If the drug isn’t being produced, or the price is too high (I don’t know how this would be defined), governments should be empowered to seize the IP and solicit bids to produce the invention. We move heaven and earth to save lives in natural disasters, why do we move so slowly with certain medical conditions?
      - umanwizard 8y ago
        Then nobody would ever bother investing in new drug development. A better model would be for the state to fund this research and have the results be public domain.
        donarb 8y ago
        Unfortunately, this happens all the time, but not for the public good. Research universities use patent portfolios to increase their revenue by licensing inventions to companies that can manufacture the product. For example in 2014 NYU earned $214M in IP licensing and nationally led the period from 2004-2010.
rbultje 8y ago
As sad as this story is, the silver lining is that patents expire, and 20 years from the patent filing point (probably 5-10 years from now?), patients will be able to be affordably cured. It sucks but is better than nothing.
- pierrebai 8y ago
  The story is clear: the problem is not the patent itself but the cost of passing regulation hurdles and maintaining a plant producing the drug when there is suck low demand. No one will invest in this even after the patent expires. The best the patients could hope is to group togethr to get some done produced?
  - whydoineedthis 8y ago
    Regulation hurdles were met, and the cost of a plant is overblown. Most specialty pharmacy are made in small labs with a few pharmacy techs, and they can make other things in between this drug. I think the issue is that of pharma alows the price to drop on this drug, they lose negotiations on other drugs. They don't want a precedence to be set where they drop thier price if insurance companies refuse to pay.
dssu 8y ago
Video for article https://www.youtube.com/watch?v=rBSv0deIVzM https://www.youtube.com/watch?v=rBSv0deIVzM
jl2718 8y ago
Does anybody know which virus vector was used?
- rfinney 8y ago
  The adeno-associated virus serotype 1 (AAV1) viral vector delivers an intact copy of the human lipoprotein lipase (LPL) gene to muscle cells. source: https://en.wikipedia.org/wiki/Alipogene_tiparvovec https://en.wikipedia.org/wiki/Alipogene_tiparvovec
mabbo 8y ago
The point of the patent system is that we are giving inventors a time-limited monopoly on their invention in exchange for them sharing it with the world. We all benefit because we have access to this new invention and soon can make it ourselves, and the inventor has an incentive to promote and sell as much as they can while they own the monopoly. But it's not working here. The patent holder is effectively saying society isn't allowed to have the invention. They're using a legal means meant to share the knowledge with the world to instead horde it away from the rest of us. When the system doesn't work, the system must be improved. How can we incentivize inventors to not do this?
- ams6110 8y ago
  If a patent holder is not producing the patented thing, and not licensing it to someone else who is, the patent should expire. Kind of like a trademark that is not defended. Seems that would fix the problem?
  - d4l3k 8y ago
    It still wouldn't get the drug to the people who need it. Most of the cost involved is in getting it approved by the authorities for human use. There's nothing stopping people from manufacturing the drug for personal use, other than that it's likely extremely hard to make.
    - ineedasername 8y ago
      In this case it's already been approved for human use. So that's a non issue. But yes, I'm sure that is a hurdle for plenty of other pharmaceutical research
  - tptacek 8y ago
    No, not here.
  - ekianjo 8y ago
    for drugs you usually patent them about 10 years before you can have them on the market. That would be not very compatible with the current requirements of regulatory bodies.
ken 8y ago
Will other scientific or technological advances (like gene therapy drugs for more common disorders) cause the price of this drug to come down in the future? Say, if gene therapy became common, there wouldn't be as much red tape for approving this particular one, and there might be machines or research that could be shared with other drugs to amortize the cost?
DoctorOetker 8y ago
>"You need to maintain the factory, you need to do the paperwork, you need to test the product, you need to make new product batches all the time because product expires," he said. I'm pretty confident LPLD sufferers around the world would be prepared to move to the LPLD hot spot in Quebec, and work part time in the factory under proper supervision?
- gus_massa 8y ago
  You can't put a bunch of untrained people in a drug factory and expect them to make a good job. You need some people with a Chemistry/Biology/Pharmaceutical degree, or some kind of specialization, or many years working in the industry. Specially if it is a cutting edge factory with a technology that is being invented now and changes every month. Whatever that can be automated so anyone can make it without knowledge is probably automated. It's like thinking that the Supreme court can fire all the technical staff, and the judges themselves can administer the web and mail server (perhaps with a little of help from some experts that come only once in a while).
tptacek 8y ago
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.
- WhompingWindows 8y ago
  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.
  - tptacek 8y ago
    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 8y ago
      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 8y ago
        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...
Mvandenbergh 8y ago
NICE, which makes value for money decisions for the NHS, uses a threshold of £25k per Quality Adjusted Life Year for assessing cost effectiveness of treatments. At that threshold, to pay for an £800k drug treatment you'd need to give someone 32 extra years of life (or 64 years at double the quality of life and so on). This is enough to pay that kind of amount for life saving gene therapies, especially if given to young children but not enough to treat something that can be largely managed through diet control. Since the majority of the world's patients are in Quebec, I don't understand why the provincial government doesn't cut a deal for this drug. I'm sure they'd rather sell it $250k a dose to a nice big patient population than at $1m to nobody. Quebec has a lot of pricing power here as the only large potential buyer.
- freeone3000 8y ago
  RAMQ [Quebec Health] does negotiate separately from the rest of Canada... but they don't have the money. They don't have the money for top-line cancer treatments, and they so severely underpaid optometrists that the optometrist union opted out entirely. There is very little chance that even at $250K a dose (which is an imaginary figure! the sellers have said they will not reduce the price at all!) a negotiation could be reached.
- xxpor 8y ago
  The fact that women can't have children without the drug, but apparently can with it adds a new complication to the QALY calculation I think.
dev_dull 8y ago
> The problem was the price. I thought that drug patents were basically illegal in India. It seems there’s a huge market there for this kind of thing. An abandoned drug with a huge price tag. Also I’m not terribly worried. The patent will wear off eventually and then anyone can make it.
whydoineedthis 8y ago
TBH, it sounds like the govt regulation for the drug to even get approved are what drove the cost up. The product was essentially made before pharma even had to lift a finger, but they made them jump through a lot of hoops to get it to market. That's the $100mm they are trying to recoup. Also, I think a lot of folks are missing that the disease is not necessarily lethal when diet is controlled. Just saying, that's all.
drannex 8y ago
The article is fascinating, but the format with the one sentence at a time system is incredibly hard to read and digest
- Raymonf 8y ago
  On the contrary, I find that easier to read! Except for the font, that's just way too light.
cletus 8y ago
So I was expecting another story about some IP troll buying the rights to a drug with a monopoly and then jacking up the price (like the whole EpiPen fiasco) but this wasn't that. There's an old Chris Rock bit about Big Pharma where he says there'll never be another cure for HIV like there was for polio because there's no money in that. The money is in getting you to the next stop. It's poignant because this isn't a theoretical scenario. Gilead recently was downgraded on declining revenues because they're wiping out the disease (Hepatitis C IIRC?). So, back to Glybera. There are plenty of low-incidence diseases that are treated by expensive drugs to manage them. This is a lifelong commitment. Covering such drugs in company health plans can significantly increase the per-member costs. If you have a drug that essentially cures the drug in one dose shouldn't that be weighed against the lifelong cost of covering a regime to manage the disease that is inferior? Multiply that by the disease being quite rare and sure, you end up with a $1 million price tag. As further evidence for how screwed up the US health insurance system is: companies enroll in plans for their members typically for a period of a year or maybe a few years. Let's say your drug's price of $1m compares favourably to $100k/year for 40 years to manage the disease. How can a company who might only be covering the employees for 1-3 years be expected to cover that higher cost? To be clear, this is further evidence of how stupid the US model is. In a single payer model this particular concern goes away. How many rare genetic disorders are out there where $1 million per patient for an essentially complete cure isn't a bargain compared to the cost of managing the disease? Probably a lot. Is it fair to decry such expensive drugs just on their price tag without looking at the facts? Probably not but I bet you it will happen.
- ken 8y ago
  > Let's say your drug's price of $1m compares favourably to $100k/year for 40 years to manage the disease. How can a company who might only be covering the employees for 1-3 years be expected to cover that higher cost? Yes, it's essentially a Prisoner's Dilemma. If every insurance company agreed to pay for this drug, the total cost of treatment overall would be lower (and the patients would be healthier and happier). If my company decides to pay it and no other insurance company does, then when the patient leaves our network (in less than 10 years) and joins another insurance network, my company will have paid the cost but can't reap the financial benefits. There's two major differences between this scenario and classic PD, though. First, companies are allowed to communicate. We could conceivably get everybody in a room together and come up with some agreement by which everybody agrees to pay for this drug, even if patients switch insurers. Everybody wins. Patients are healthier, and insurers save money. Second, it's not a one-time decision. An insurer can change their mind from "no" to "yes" at any time. So it's more like "iterated PD", with an indefinite number of rounds. In that game, interestingly, there is no strictly dominant strategy, and altruism tends to do better in practice! So maybe you don't even need an agreement. > To be clear, this is further evidence of how stupid the US model is. In a single payer model this particular concern goes away. How do you figure? The article points out that they also had trouble convincing European governments to pay for it. You can't get Glybera anywhere in Europe or Canada today, either. The US model is arguably a poor one, but this isn't a good example of that, because every other country in the world failed at Glybera, too.
DoreenMichele 8y ago
This is kind of a common issue with genetic disorders. When I was active on cystic fibrosis lists, sometimes articles were posted that said stuff like "It costs an average of $100k annually to treat CF" and parents would discuss it and go "That sounds low to me" and some older patient would chime in and say "Last year, my drug prescriptions alone were $100k. That's not counting the cost of doctor's visits and hospitalizations." (Estimates in articles vary wildly. I searched briefly and just trying to find something that makes sense for like two minutes just makes me tired. So if you want to jump up and dispute that with some googled up article, I don't feel like arguing with you about it. I've already told you actual people living with the condition read those articles and go "Where on earth are they getting these ridiculous low ball figures???") So then they come out with some very expensive drug that costs like a quarter million or more annually and it only treats around five percent or so of patients with CF. The price on Wikipedia [1] is currently listed at over $300k annually. [1] https://en.wikipedia.org/wiki/Ivacaftor https://en.wikipedia.org/wiki/Ivacaftor And then who can afford that? If you have been sick your whole life, you probably don't have a lot of savings or a lot of ability to come up with big bucks, etc. You are probably deeply in debt and not making much money. I don't know the answer. Conventional medicine doesn't really work for people with genetic disorders. It tends to be crazy expensive and also merely mitigates things somewhat. The standard expectation is that you will suffer a bit less and maybe live a bit longer, but you won't ever really be well. So such people understandably want a cure. That's the holy grail for folks coping with genetic disorders. I think there are potentially other avenues for some portion of people with genetic disorders. But I don't currently have the words for that and I get tired of being ganged up on by boatloads of internet strangers who want to inform me that I'm imagining things and my entire life experience is a hallucination and I don't actually know nothing about genetic anything. So let's just leave it at "I have this opinion and maybe it provides a way out of this trap and maybe it doesn't." Because the current approach of inventing incredibly expensive drugs that might provide a cure is essentially failing as a method. And all other approaches are so awful it's why such things get labeled "dread diseases" -- because what it does to your life is so terrible that if a doctor were not prescribing it, it would be in violation of the Geneva Convention. It's just not humane.
hummingurban 8y ago
To me, this is akin to a game company raising the price of a game. The costs are already sunk. The only thing that counts is setting a price on copies of that game. Those who pirate copies of the game incur to loss of revenue because those people would've never been able to pay for it. It becomes a question of projecting the maker's morals onto those who did not pitch to the coffer instead enjoying the game like a dirty thief. This is crazy, the only loss to the manufacturer is the uncomfortable idea people are enjoying or benefiting from your creation for free. Yet there are developers who happily contribute to open source projects and games with little to no recognition or reward. Likewise with drug manufacturing, it's the same idea. It's illegal to copy a pharmaceutical drug and take it yourself and others without the approval of the patent holder. Yet, it is the patent holding party that determines how many licensed copies it is willing to sell. Thus, they are able to take the moral high ground in pricing this drug, but it's the same principle as above-pony up for my idea, shame on you for benefitting from it and not contributing to my wealth. I really do think this company is out of touch with reality. All in all, I feel like this only earns them bad press, especially after Martin Shrekli debacle. There's also a potential for a lawsuit, based on human rights I'm sure.
apo 8y ago
Glybera was never sold in North America and was available in Europe for just two years, beginning in 2015 The patent will expire within a few years, setting the stage for generic manufacturers to step in, but the article doesn't mention it. In the US, patent protection extends for 20 years after the earliest filing date for the application on which the grant is based. There can be adjustments to the term if the grant was delayed. https://en.wikipedia.org/wiki/Term_of_patent https://en.wikipedia.org/wiki/Term_of_patent The article is fuzzy about the timeline of events, bu has this quote: The stunning results of the mouse experiments were featured on the cover of the journal Human Gene Therapy in September 2004. The cover image showed how, week by week, the mouse blood changed from milky white to a clear, transparent red, illustrating just how effectively the gene therapy was working. So it seems like a good first guess to put the patent filing at or before 2004. If so, then the Glybera patent would expire sometime after 2024. At that point, generic manufacturers can step in to sell their version of the product, assuming regulatory approval.
- refurb 8y ago
  As you can see from the lack of biosimilars in the US, manufacturing a biological is a hell of a lot more complex and expensive than doing it for a small molecule. And in fact, since this is version 1.0, I’ll bet a better version comes out before any generic company launches.
  - akvadrako 8y ago
    If they come out with a new version, they must be expecting it to sell in a quantity that makes the investment worth it. So maybe the pricing worry is just a temporary concern.
umanwizard 8y ago
> "Why would we? Pricing shouldn't be a political decision. It should be a rational decision based on merits and values," he said. "Hundreds of millions of investor money has gone into the company, and if there is no return for those investments, there will be no new drugs because nobody's going to do that in the future, right?" As terrible as it sounds, this logic seems sound to me. If we think having this type of drug is worth the > $100MM it took to develop, then health systems should either fund the $100MM research, or they should let private companies do it and pay them fairly for their work. $1MM for something so expensive to develop with so few possible users doesn't seem crazy to me. If the $100MM is in fact not worth it, then, well, you get the situation we have now. I'm sure a lot of people will read this article and think the owners/funders of the drugs are the bad guys. But nobody can be expected to work for free.
- eloff 8y ago
  Yeah, people just look at the price and think that's terrible, but it's a business not a charity. It's up to the government to step up if people really deem this to be a problem worth solving. With their tax dollars. I bet a lot of critics would go quiet at the point they actually have to pony up.
- wiz21c 8y ago
  There another way to read that sentence : >>> Hundreds of millions of investor money has gone into the company, and if there is no return for those investments, Considering that there are patients who needs it, you can divide the price by 10, this way you make sure everybody will pay and the only sacrifice you make is your ROI to be on a 10x longer period. The problem with investors is that they want tons of benefits and in a very short time. They could as well sell the IP if they don't use it anymore. I understand that this is business, but here I see at as "if we fail, then everybody will fail".
GeekyBear 8y ago
Getting a drug approved for use is an expensive proposition, but that doesn't mean that it is impossible for a nonprofit organization to step in and get the job done when the target treatment group is too small or too poor to make it worthwhile for those with a profit motive. The Drugs for Neglected Diseases Initiative, a nonprofit which grew out of Doctors Without Borders, just received approval for a pill that successfully treats African Sleeping Sickness. >Ultimately, the drive for approval cost $63 million and involved clinical trials including 750 patients in Congo and the Central African Republic. Two million villagers were screened. The costs were paid by seven European countries, the Bill and Melinda Gates Foundation, Doctors Without Borders and other donors. https://www.nytimes.com/2018/11/16/health/sleeping-sickness-africa-cure.html https://www.nytimes.com/2018/11/16/health/sleeping-sickness-...
cubano 8y ago
This article brings up a thought experiment I've been pondering for a good while now... So what would you charge for a drug that guaranteed you would live 30 extra years after you spent 20 million to develop it? For an added twist, let's say it's NOT FDA approved but you have solid clinical evidence that it's efficacy was solid and can your "street cred" (whatever that means in biotech) allows you access to the world's most successful (ie richest) people. I have my answer...what's yours?
- InTheArena 8y ago
  This is exactly the reason we have governments. Theoretically in a liberal democracy, we don't allow people to do actions that enrich themselves at unreasonable societal cost. Allowing only the rich to have access to longer life is one of those basic things that governments are supposed to be here.
- glass_of_water 8y ago
  That's a really interesting thought experiment, and I don't really know what my answer is. The following choices assume that more people living an extra 30 years is a good thing ethically, and that there are no devastating effects environmentally or otherwise. The following choices also assume that you'll always have a monopoly on the production of the drug. 1. Assuming that the supply is practically unlimited, an altruistic actor should probably give it away for as close to free as possible. A purely selfish actor should probably try to sell to each individual for as much as they can afford (though this can be hard pricing strategy to enact). I suppose you might actually want to charge less than that even as a purely selfish actor, since you wouldn't want to somehow destroy the world economy by making everyone really poor except for yourself (would this actually destroy the economy though? I'm not sure... probably not relevant for the purposes of this thought experiment). 2. Assuming supply is limited, a purely altruistic actor will have to choose the most fitting recipients. This is a really tough choice. How do you define the most fitting recipients? Those who are most likely to contribute to the betterment of humanity with their extra 30 years? It seems like an almost impossible task to pick out who these people would be. A purely selfish actor should charge the richest as much as they're willing to pay. If it were me and supply were unlimited, I'd like to think that I'd sell it for as little as possible but enough to still never have to worry about money again. Making $10 million in profit would probably be more than enough (though if you can make more and still help everyone, that'd be even better). If supply were limited, I don't know what I would do. One thing I just realized though, is that even if supply were unlimited, if you had this godly power, you might choose to withhold it those you deem unworthy, which is a whole other ethical can of worms. I'm guessing this kind of deviates from the intent of your thought experiment though. What's your answer?
mirimir 8y ago
> But it's the only way scientific discoveries ever get to patients, because universities don't make drugs. Why not? If there are so very few patients, a lab could easily produce enough for them.
- ineedasername 8y ago
  Yeah, they had to make some quantity to run their trials. presumably it cost much less than $1mil/dose or they'd not have research funding at their level
  - mirimir 8y ago
    Indeed. A lot less than $1million per dose, I bet. I understand that there are regulatory issues. But it seems pretty clear that the standard pharma industry model doesn't work for drugs like this.
iamleppert 8y ago
I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing to me that we can manufacture and program viral particles to target specific cells. In my case, my treatment is a non-nucleonic technique that didn't repair the actual chromosomal hereditary defect but inserted a working gene, additional and freely available genetic material into my liver cells. It's amazing that the body can just recognize this extra bit of code and just start producing the factor. The engineer in me is also impressed they can make so many copies of the engineered virus. It appears to be working. My factor levels have steadily increased and I’m now no longer a severe or even moderate hemophiliac. I’m their first patient to have achieved these results for hemophilia A severe and it puts me well into the therapeutic range. It really feels like I have been given a new body, it’s indescribable. For the first time in my life, I’m pain free. It’s a weird feeling. Pain, mostly from bleeds into my joints, has been my constant companion. I was never able to do sports as a kid and had to avoid many other activities that would put me at risk. I have mixed feelings about all of this. On one hand I’m extraordinarily grateful, and the other side I see what a huge disadvantage I’ve been at compared to others for all of my life. I thought this day would never come, I had resigned myself to my limitations but now they are gone in a matter of weeks through the miracle of a medical experiment. My old medication, a replacement clotting factor that was synthetically produced in a lab, worked but not nearly as well as this. Not to mention the fact it cost over $100,000 per month and I had to give myself an IV every other day. It's truly been a life changing experience for me, and I hope that gene therapy becomes widely available to others. Also, the thing that was a milestone in this case was that the Hemophilia A factor VIII gene has been notoriously difficult to create a genetic therapy for because of the complexity of the actual gene itself. On the scale of what the body produces, its one of the more complicated proteins. So it basically means many other genetic conditions will be able to be treated with and addressed with similar technology. Such an exciting time to be alive, and yes there are amazing things happening right now despite all the bad news you may hear!
- homero 8y ago
anon2775 8y ago
If it works but it's not economically-viable: the moral and ethical choice is to open-source it or give it to a non-profit medical foundation. It sucks to have a rare disease because there isn't enough profit to recoup r&d; this is where non-profits, NGOs and GSEs should advance medicine where big pharma trades lives for profits.
timka 8y ago
That's probably the greatest example of the issue that Open Longevity was going to solve by combining patient organization with ICO funding to conduct clinical trials without profit driven big pharma. Unfortunately, that didn't happen…
platz 8y ago
> Pricing shouldn't be a political decision. It should be a rational decision based on merits and values," he said. The money quote. A rational decision? Maybe. Exactly what merits? Exactly which values? "Hundreds of millions of investor money has gone into the company" But none of that led to the development of the drug.
ada1981 8y ago
What’s up with this drug now? Sounds like it’s patents and IP protection should be up. I’d imagine you could get a single dose manufactured 1 off for $100k by a lab.

298 points by rpledge 8y ago

"Well, one pharma company has the patent, and we need to let them charge what they think the market will bear. Sorry LPLD carriers, you have to die now." Apparently, this is the best the world can manage in 2018.

miguelrochefort 8y ago
I don't understand how anyone can think patents are a good idea.
- jmcgough 8y ago
  Not a fan of it either, but how do we incentivize companies to research and create new drugs if they can't make back the huge cost of clinical trials?
  - carbocation 8y ago
    What if governments paid for the clinical trials?
    - NikolaeVarius 8y ago
      The article has a section that talks about the difficulty of getting the drug even approved for testing by governments in the first place.
    - sneak 8y ago
      The government is the whole reason the clinical trials are required before patients can be treated with a working drug. They are the cause of the problem, not the solution.
    - longerthoughts 8y ago
      The government does frequently fund the discovery of new drugs. The problem is that the return on that investment is usually abysmal and the taxpayer ends up not only paying to fund the drug’s discovery, but also paying absurdly high prices to buy it from pharmaceutical companies who passed some or all of their risk onto taxpayers. One example: https://abcnews.go.com/WNT/YourMoney/story?id=129651 https://abcnews.go.com/WNT/YourMoney/story?id=129651
      - carbocation 8y ago
        Of course. Most of basic science in the US is government funded. I'm wondering what the OP thinks about removing the barrier that they identified (private corps paying for the trial vs gov paying for the trial) and how that would affect their thinking.
      - umanwizard 8y ago
        If you read the article, the drug was in fact discovered at UBC, which is a publicly funded university. But it turns out that discovering the drug wasn't the expensive part. The expensive part was privately funded.
  - latch 8y ago
    Did you read the article? The company that charged that absurd price acquired the patent, they had nothing to do with developing it.
    - mabbo 8y ago
      While I don't disagree with your point, the company that bought the patent paid a lot of money for it from the company that developed it. Patents being exchangeable is not necessarily a bad thing.
  - miguelrochefort 8y ago
    How do you incentivize environmental research? How are we supposed to find solutions to climate change if they can't be patented? Copyrights are just as bad as patents, and musicians/writers shouldn't make people pay for their music/books. We should start giving money to causes we support, whether it's art, medical research, software projects, etc. It's a mistake to think we should only give money if we're forced to by law (through pricing something that's not scarce).
- worldsayshi 8y ago
  There should at the very least be a law that states that a patented technology should be made available in reasonable ways to make the patent valid.
  - umanwizard 8y ago
    Would you rather have this drug exist and be patented, or never have existed at all?
    - zaptheimpaler 8y ago
      I see you arguing that things went just the way they should have all over this thread. This point in particular is completely wrong. The two doctors who made the drug do not even hold the patent and basically gave it away for free to the company trying to distribute it. So yes, if not for the patent this drug would definitely still exist, and probably be available to people who actually freaking need it.
      - umanwizard 8y ago
        > I see you arguing that things went just the way they should have all over this thread. Actually, I never said that anywhere. I think what happened is basically a no-op. Without a profit motive, the drug would never have been developed, and therefore not available to anyone, and we'd be in the same boat we're in anyway. What I do think is one of two things should happen: Either (1) we decide that the drug is worth it, in which case health systems should pay the owner of this drug fairly for their investment. Or (2) it's not worth it, in which case we're no worse off than we would have been without this company, other than the fact that they wasted a bunch of their money. (As an aside, note that I said we should pay the owner, fairly for their invention, not the original developer who voluntarily gave up the rights. This is an important distinction as the ability to voluntarily transfer property rights is pretty central to the whole concept. Imagine if after you bought a car, anyone could take it, because you're not the original manufacturer. It's not hard to see the path from this society to one where nobody bothers making cars.) > The two doctors who made the drug do not even hold the patent and basically gave it away for free to the company trying to distribute it. Those two doctors did the least expensive part of the whole operation. The more expensive part was funded privately. If you want to fix this you can either have the government fund that expensive part, or you can have it done privately and pay the owners what they want. > if not for the patent this drug would definitely still exist, and probably be available to people who actually freaking need it Sure, maybe the doctors still would have done the research, but there would be no money either for the clinical trials, and so manufacturing and distributing the drug would be illegal.
- charlesism 8y ago
  I don't have a problem with the idea of patents. I just think we could be a whole lot more pragmatic about them. They're supposed to incentivize useful behavior.
  - miguelrochefort 8y ago
    Who should decide what's a useful behavior? What's a fair price? What's a reasonable lroduction/distribution strategy? I think the free market should decide, not the government and its enforced monopolies.
- umanwizard 8y ago
  Reading the article has convinced me that drug patents in particular are a great idea. Isn't it wonderful that we live in a world where futuristic new treatments like this can be developed? Yes it's true that people can't get the drug now, but at least someday it might be available. Without patents, who would you find to work for free developing something like this?
  - miguelrochefort 8y ago
    > Isn't it wonderful that we live in a world where futuristic new treatments like this can be developed? This futuristic new treatment can no longer be developed or produced by ANYONE in the world, without the consent of this company. Before the patent, anyone could. Do you think that's not a problem? We'll incentivize researchers the same way we'll do with musicians and writers in a post-copyright society. People who think this disease is important will crowdfund the research, and those who want to receive experimental and personalized treatment will pay for it. How do you think we should incentivize things like environmental research? Is our knowledge about climate change patentable? What if someone found a cure?
    - umanwizard 8y ago
      > Before the patent, anyone could Anyone could, but nobody did, or would have without either a profit motive or massive state funding. > Do you think that's not a problem? Well it’s certainly not the most ideal thing you can imagine, but I don’t see a better way in a capitalist society to incentivize medical research. Do you? > People who think this disease is important will crowdfund the research I’m not aware of any crowdfunding campaign that has raised hundreds of millions of dollars. > How do you think we should incentivize things like environmental research? It should be funded by the state > What if someone found a cure? That’d be awesome. We should pay them incredibly handsomely for their work.
cratermoon 8y ago
Late-stage capitalism in a nutshell.
lisper 8y ago
The last sentence of the article sums it up pretty well: "If it's not commercially viable to produce a certain therapy, unfortunately, in our Western society, it does not happen." And it is far from clear that this is not as it should be, because it's not just LPLD carriers about whom tear-terking stories can be told. $1M can be deployed in other ways that could potentially save more lives. Instead of "Sorry LPLD carriers, you have to die now", it might be, "Sorry, 100 poor African children without access to clean water, you have to continue to die of dysentery so we can save this one LPLD carrier."
- thomastjeffery 8y ago
  Except $1 millon is not the entire economy. It's easy to hide resources by equating them to their monetary value. The fact is that there exist many millions of dollars, and the way each dollar is spent does not determine the way another dollar is spent.
  - lisper 8y ago
    So what? Dollars are still proxies for finite resources, and there are only three possible ways to procure them: their owners can give them voluntarily, they can be forcibly seized by the government through taxation, or the government can print them, which produces inflation and effectively seizes a little bit from everyone who holds dollars (essentially a tax on savings). If you can't get someone to voluntarily spend $1M to save a life, which of the other two processes do you propose to use?
  - tptacek 8y ago
    This isn't complicated. Say there are 1,000 LPLD patients in North America (a wild overestimate). Then treating them with this therapy costs us $1Bn. If we limit ourselves to health care problems, is $1Bn better spent on these 1,000 LPLD patients, or on increasing penetration of existing therapies for other conditions? It seems likely to be the latter. To believe LPLD therapy objectively deserves the allocation you're talking about, you have to believe that there aren't other cohorts of patients that are currently underserved. But we know that isn't true.
- 8y ago

ttul 8y ago

This is what we call “market failure”.

gfodor 8y ago

in society, we have another large, one-time purchase people often make in which there are special financial arrangements and rules: buying a home. it seems to me that gene therapies (high priced, one-time use drugs) are going to warrant alternative financial instruments. there's absolutely no reason this drug should have failed when the amortized cost over 10 years is less than an inferior product. regardless of the other questions this is a pure market failure by the financial industry. edit: the downvotes probably assume I mean the patient is going to pay out of pocket. of course not. the insurance companies would be paying. the only inferiority between gene therapies and non-cures is their pricing structure. we invented a solution to this problem thousands of years ago: debt.

wrong_variable 8y ago
The problem is with a house / car / TV / phone you can always reposes it.
- nradov 8y ago
  We have loans for education even though lenders can't repossess knowledge.
- gfodor 8y ago
  It just backs out to interest rates commensurate with the risk. Lack of collateral doesn't mean you can't get a loan. Perhaps the economics wouldn't work, but it sure sounds like nobody tried in this case. What it would boil down to is if the insurance company expected a larger net cost on a traditional drug regimen for the life of the patient, vs servicing a loan at the market rate over N years. Interest rates are still very low so in general debt is seeking out risk.
  - ThrustVectoring 8y ago
    Insurance companies don't cover total health costs over the life of the patient. They cover the next month of health costs, and a certain percentage of their customers churn afterwards. For insurance companies, providing treatment that lasts a longer amount of time can often be a terrible business decision - if the customer switches insurance providers afterwards, then they're essentially subsidizing the insurance company they switched to. This more commonly happens with prescription quantities. Medication compliance is far better when people can pick up a three-month supply of medication. Insurance companies want patients to pick up one month supply instead, so that they aren't paying for two months of medication for every patient who switches insurance companies.

apsec112 8y ago

This seems bizarre to me: Van Deventer says the company never considered lowering the price. "Why would we? Pricing shouldn't be a political decision. It should be a rational decision based on merits and value." If no one will pay $1 million, your revenue is $0. Assuming the drug costs much less than $1 million to make, surely any revenue number is better than $0?

simion314 8y ago
What I think is happening is that the number of affected people is so low and the cost of producing this small number is very large and the company does not want a profit but a BIG profit.
- worldsayshi 8y ago
  If a company does not make use of a patent they should at least loose their right to it.
  - torpfactory 8y ago
    Especially for lifesaving medical patents. If the drug isn’t being produced, or the price is too high (I don’t know how this would be defined), governments should be empowered to seize the IP and solicit bids to produce the invention. We move heaven and earth to save lives in natural disasters, why do we move so slowly with certain medical conditions?
    - umanwizard 8y ago
      Then nobody would ever bother investing in new drug development. A better model would be for the state to fund this research and have the results be public domain.
      - donarb 8y ago
        Unfortunately, this happens all the time, but not for the public good. Research universities use patent portfolios to increase their revenue by licensing inventions to companies that can manufacture the product. For example in 2014 NYU earned $214M in IP licensing and nationally led the period from 2004-2010.

rbultje 8y ago

As sad as this story is, the silver lining is that patents expire, and 20 years from the patent filing point (probably 5-10 years from now?), patients will be able to be affordably cured. It sucks but is better than nothing.

pierrebai 8y ago
The story is clear: the problem is not the patent itself but the cost of passing regulation hurdles and maintaining a plant producing the drug when there is suck low demand. No one will invest in this even after the patent expires. The best the patients could hope is to group togethr to get some done produced?
- whydoineedthis 8y ago
  Regulation hurdles were met, and the cost of a plant is overblown. Most specialty pharmacy are made in small labs with a few pharmacy techs, and they can make other things in between this drug. I think the issue is that of pharma alows the price to drop on this drug, they lose negotiations on other drugs. They don't want a precedence to be set where they drop thier price if insurance companies refuse to pay.

dssu 8y ago

Video for article https://www.youtube.com/watch?v=rBSv0deIVzM https://www.youtube.com/watch?v=rBSv0deIVzM

jl2718 8y ago

Does anybody know which virus vector was used?

rfinney 8y ago
The adeno-associated virus serotype 1 (AAV1) viral vector delivers an intact copy of the human lipoprotein lipase (LPL) gene to muscle cells. source: https://en.wikipedia.org/wiki/Alipogene_tiparvovec https://en.wikipedia.org/wiki/Alipogene_tiparvovec

mabbo 8y ago

The point of the patent system is that we are giving inventors a time-limited monopoly on their invention in exchange for them sharing it with the world. We all benefit because we have access to this new invention and soon can make it ourselves, and the inventor has an incentive to promote and sell as much as they can while they own the monopoly. But it's not working here. The patent holder is effectively saying society isn't allowed to have the invention. They're using a legal means meant to share the knowledge with the world to instead horde it away from the rest of us. When the system doesn't work, the system must be improved. How can we incentivize inventors to not do this?

ams6110 8y ago
If a patent holder is not producing the patented thing, and not licensing it to someone else who is, the patent should expire. Kind of like a trademark that is not defended. Seems that would fix the problem?
- d4l3k 8y ago
  It still wouldn't get the drug to the people who need it. Most of the cost involved is in getting it approved by the authorities for human use. There's nothing stopping people from manufacturing the drug for personal use, other than that it's likely extremely hard to make.
  - ineedasername 8y ago
    In this case it's already been approved for human use. So that's a non issue. But yes, I'm sure that is a hurdle for plenty of other pharmaceutical research
- tptacek 8y ago
  No, not here.
- ekianjo 8y ago
  for drugs you usually patent them about 10 years before you can have them on the market. That would be not very compatible with the current requirements of regulatory bodies.

ken 8y ago

Will other scientific or technological advances (like gene therapy drugs for more common disorders) cause the price of this drug to come down in the future? Say, if gene therapy became common, there wouldn't be as much red tape for approving this particular one, and there might be machines or research that could be shared with other drugs to amortize the cost?

DoctorOetker 8y ago

>"You need to maintain the factory, you need to do the paperwork, you need to test the product, you need to make new product batches all the time because product expires," he said. I'm pretty confident LPLD sufferers around the world would be prepared to move to the LPLD hot spot in Quebec, and work part time in the factory under proper supervision?

gus_massa 8y ago
You can't put a bunch of untrained people in a drug factory and expect them to make a good job. You need some people with a Chemistry/Biology/Pharmaceutical degree, or some kind of specialization, or many years working in the industry. Specially if it is a cutting edge factory with a technology that is being invented now and changes every month. Whatever that can be automated so anyone can make it without knowledge is probably automated. It's like thinking that the Supreme court can fire all the technical staff, and the judges themselves can administer the web and mail server (perhaps with a little of help from some experts that come only once in a while).

tptacek 8y ago

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.

WhompingWindows 8y ago
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.
- tptacek 8y ago
  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 8y ago
    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 8y ago
      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...

Mvandenbergh 8y ago

NICE, which makes value for money decisions for the NHS, uses a threshold of £25k per Quality Adjusted Life Year for assessing cost effectiveness of treatments. At that threshold, to pay for an £800k drug treatment you'd need to give someone 32 extra years of life (or 64 years at double the quality of life and so on). This is enough to pay that kind of amount for life saving gene therapies, especially if given to young children but not enough to treat something that can be largely managed through diet control. Since the majority of the world's patients are in Quebec, I don't understand why the provincial government doesn't cut a deal for this drug. I'm sure they'd rather sell it $250k a dose to a nice big patient population than at $1m to nobody. Quebec has a lot of pricing power here as the only large potential buyer.

freeone3000 8y ago
RAMQ [Quebec Health] does negotiate separately from the rest of Canada... but they don't have the money. They don't have the money for top-line cancer treatments, and they so severely underpaid optometrists that the optometrist union opted out entirely. There is very little chance that even at $250K a dose (which is an imaginary figure! the sellers have said they will not reduce the price at all!) a negotiation could be reached.
xxpor 8y ago
The fact that women can't have children without the drug, but apparently can with it adds a new complication to the QALY calculation I think.

dev_dull 8y ago

> The problem was the price. I thought that drug patents were basically illegal in India. It seems there’s a huge market there for this kind of thing. An abandoned drug with a huge price tag. Also I’m not terribly worried. The patent will wear off eventually and then anyone can make it.

whydoineedthis 8y ago

TBH, it sounds like the govt regulation for the drug to even get approved are what drove the cost up. The product was essentially made before pharma even had to lift a finger, but they made them jump through a lot of hoops to get it to market. That's the $100mm they are trying to recoup. Also, I think a lot of folks are missing that the disease is not necessarily lethal when diet is controlled. Just saying, that's all.

drannex 8y ago

The article is fascinating, but the format with the one sentence at a time system is incredibly hard to read and digest

Raymonf 8y ago
On the contrary, I find that easier to read! Except for the font, that's just way too light.

cletus 8y ago

So I was expecting another story about some IP troll buying the rights to a drug with a monopoly and then jacking up the price (like the whole EpiPen fiasco) but this wasn't that. There's an old Chris Rock bit about Big Pharma where he says there'll never be another cure for HIV like there was for polio because there's no money in that. The money is in getting you to the next stop. It's poignant because this isn't a theoretical scenario. Gilead recently was downgraded on declining revenues because they're wiping out the disease (Hepatitis C IIRC?). So, back to Glybera. There are plenty of low-incidence diseases that are treated by expensive drugs to manage them. This is a lifelong commitment. Covering such drugs in company health plans can significantly increase the per-member costs. If you have a drug that essentially cures the drug in one dose shouldn't that be weighed against the lifelong cost of covering a regime to manage the disease that is inferior? Multiply that by the disease being quite rare and sure, you end up with a $1 million price tag. As further evidence for how screwed up the US health insurance system is: companies enroll in plans for their members typically for a period of a year or maybe a few years. Let's say your drug's price of $1m compares favourably to $100k/year for 40 years to manage the disease. How can a company who might only be covering the employees for 1-3 years be expected to cover that higher cost? To be clear, this is further evidence of how stupid the US model is. In a single payer model this particular concern goes away. How many rare genetic disorders are out there where $1 million per patient for an essentially complete cure isn't a bargain compared to the cost of managing the disease? Probably a lot. Is it fair to decry such expensive drugs just on their price tag without looking at the facts? Probably not but I bet you it will happen.

ken 8y ago
> Let's say your drug's price of $1m compares favourably to $100k/year for 40 years to manage the disease. How can a company who might only be covering the employees for 1-3 years be expected to cover that higher cost? Yes, it's essentially a Prisoner's Dilemma. If every insurance company agreed to pay for this drug, the total cost of treatment overall would be lower (and the patients would be healthier and happier). If my company decides to pay it and no other insurance company does, then when the patient leaves our network (in less than 10 years) and joins another insurance network, my company will have paid the cost but can't reap the financial benefits. There's two major differences between this scenario and classic PD, though. First, companies are allowed to communicate. We could conceivably get everybody in a room together and come up with some agreement by which everybody agrees to pay for this drug, even if patients switch insurers. Everybody wins. Patients are healthier, and insurers save money. Second, it's not a one-time decision. An insurer can change their mind from "no" to "yes" at any time. So it's more like "iterated PD", with an indefinite number of rounds. In that game, interestingly, there is no strictly dominant strategy, and altruism tends to do better in practice! So maybe you don't even need an agreement. > To be clear, this is further evidence of how stupid the US model is. In a single payer model this particular concern goes away. How do you figure? The article points out that they also had trouble convincing European governments to pay for it. You can't get Glybera anywhere in Europe or Canada today, either. The US model is arguably a poor one, but this isn't a good example of that, because every other country in the world failed at Glybera, too.

DoreenMichele 8y ago

This is kind of a common issue with genetic disorders. When I was active on cystic fibrosis lists, sometimes articles were posted that said stuff like "It costs an average of $100k annually to treat CF" and parents would discuss it and go "That sounds low to me" and some older patient would chime in and say "Last year, my drug prescriptions alone were $100k. That's not counting the cost of doctor's visits and hospitalizations." (Estimates in articles vary wildly. I searched briefly and just trying to find something that makes sense for like two minutes just makes me tired. So if you want to jump up and dispute that with some googled up article, I don't feel like arguing with you about it. I've already told you actual people living with the condition read those articles and go "Where on earth are they getting these ridiculous low ball figures???") So then they come out with some very expensive drug that costs like a quarter million or more annually and it only treats around five percent or so of patients with CF. The price on Wikipedia [1] is currently listed at over $300k annually. [1] https://en.wikipedia.org/wiki/Ivacaftor https://en.wikipedia.org/wiki/Ivacaftor And then who can afford that? If you have been sick your whole life, you probably don't have a lot of savings or a lot of ability to come up with big bucks, etc. You are probably deeply in debt and not making much money. I don't know the answer. Conventional medicine doesn't really work for people with genetic disorders. It tends to be crazy expensive and also merely mitigates things somewhat. The standard expectation is that you will suffer a bit less and maybe live a bit longer, but you won't ever really be well. So such people understandably want a cure. That's the holy grail for folks coping with genetic disorders. I think there are potentially other avenues for some portion of people with genetic disorders. But I don't currently have the words for that and I get tired of being ganged up on by boatloads of internet strangers who want to inform me that I'm imagining things and my entire life experience is a hallucination and I don't actually know nothing about genetic anything. So let's just leave it at "I have this opinion and maybe it provides a way out of this trap and maybe it doesn't." Because the current approach of inventing incredibly expensive drugs that might provide a cure is essentially failing as a method. And all other approaches are so awful it's why such things get labeled "dread diseases" -- because what it does to your life is so terrible that if a doctor were not prescribing it, it would be in violation of the Geneva Convention. It's just not humane.

hummingurban 8y ago

To me, this is akin to a game company raising the price of a game. The costs are already sunk. The only thing that counts is setting a price on copies of that game. Those who pirate copies of the game incur to loss of revenue because those people would've never been able to pay for it. It becomes a question of projecting the maker's morals onto those who did not pitch to the coffer instead enjoying the game like a dirty thief. This is crazy, the only loss to the manufacturer is the uncomfortable idea people are enjoying or benefiting from your creation for free. Yet there are developers who happily contribute to open source projects and games with little to no recognition or reward. Likewise with drug manufacturing, it's the same idea. It's illegal to copy a pharmaceutical drug and take it yourself and others without the approval of the patent holder. Yet, it is the patent holding party that determines how many licensed copies it is willing to sell. Thus, they are able to take the moral high ground in pricing this drug, but it's the same principle as above-pony up for my idea, shame on you for benefitting from it and not contributing to my wealth. I really do think this company is out of touch with reality. All in all, I feel like this only earns them bad press, especially after Martin Shrekli debacle. There's also a potential for a lawsuit, based on human rights I'm sure.

apo 8y ago

Glybera was never sold in North America and was available in Europe for just two years, beginning in 2015 The patent will expire within a few years, setting the stage for generic manufacturers to step in, but the article doesn't mention it. In the US, patent protection extends for 20 years after the earliest filing date for the application on which the grant is based. There can be adjustments to the term if the grant was delayed. https://en.wikipedia.org/wiki/Term_of_patent https://en.wikipedia.org/wiki/Term_of_patent The article is fuzzy about the timeline of events, bu has this quote: The stunning results of the mouse experiments were featured on the cover of the journal Human Gene Therapy in September 2004. The cover image showed how, week by week, the mouse blood changed from milky white to a clear, transparent red, illustrating just how effectively the gene therapy was working. So it seems like a good first guess to put the patent filing at or before 2004. If so, then the Glybera patent would expire sometime after 2024. At that point, generic manufacturers can step in to sell their version of the product, assuming regulatory approval.

refurb 8y ago
As you can see from the lack of biosimilars in the US, manufacturing a biological is a hell of a lot more complex and expensive than doing it for a small molecule. And in fact, since this is version 1.0, I’ll bet a better version comes out before any generic company launches.
- akvadrako 8y ago
  If they come out with a new version, they must be expecting it to sell in a quantity that makes the investment worth it. So maybe the pricing worry is just a temporary concern.

umanwizard 8y ago

> "Why would we? Pricing shouldn't be a political decision. It should be a rational decision based on merits and values," he said. "Hundreds of millions of investor money has gone into the company, and if there is no return for those investments, there will be no new drugs because nobody's going to do that in the future, right?" As terrible as it sounds, this logic seems sound to me. If we think having this type of drug is worth the > $100MM it took to develop, then health systems should either fund the $100MM research, or they should let private companies do it and pay them fairly for their work. $1MM for something so expensive to develop with so few possible users doesn't seem crazy to me. If the $100MM is in fact not worth it, then, well, you get the situation we have now. I'm sure a lot of people will read this article and think the owners/funders of the drugs are the bad guys. But nobody can be expected to work for free.

eloff 8y ago
Yeah, people just look at the price and think that's terrible, but it's a business not a charity. It's up to the government to step up if people really deem this to be a problem worth solving. With their tax dollars. I bet a lot of critics would go quiet at the point they actually have to pony up.
wiz21c 8y ago
There another way to read that sentence : >>> Hundreds of millions of investor money has gone into the company, and if there is no return for those investments, Considering that there are patients who needs it, you can divide the price by 10, this way you make sure everybody will pay and the only sacrifice you make is your ROI to be on a 10x longer period. The problem with investors is that they want tons of benefits and in a very short time. They could as well sell the IP if they don't use it anymore. I understand that this is business, but here I see at as "if we fail, then everybody will fail".

GeekyBear 8y ago

Getting a drug approved for use is an expensive proposition, but that doesn't mean that it is impossible for a nonprofit organization to step in and get the job done when the target treatment group is too small or too poor to make it worthwhile for those with a profit motive. The Drugs for Neglected Diseases Initiative, a nonprofit which grew out of Doctors Without Borders, just received approval for a pill that successfully treats African Sleeping Sickness. >Ultimately, the drive for approval cost $63 million and involved clinical trials including 750 patients in Congo and the Central African Republic. Two million villagers were screened. The costs were paid by seven European countries, the Bill and Melinda Gates Foundation, Doctors Without Borders and other donors. https://www.nytimes.com/2018/11/16/health/sleeping-sickness-africa-cure.html https://www.nytimes.com/2018/11/16/health/sleeping-sickness-...

cubano 8y ago

This article brings up a thought experiment I've been pondering for a good while now... So what would you charge for a drug that guaranteed you would live 30 extra years after you spent 20 million to develop it? For an added twist, let's say it's NOT FDA approved but you have solid clinical evidence that it's efficacy was solid and can your "street cred" (whatever that means in biotech) allows you access to the world's most successful (ie richest) people. I have my answer...what's yours?

InTheArena 8y ago
This is exactly the reason we have governments. Theoretically in a liberal democracy, we don't allow people to do actions that enrich themselves at unreasonable societal cost. Allowing only the rich to have access to longer life is one of those basic things that governments are supposed to be here.
glass_of_water 8y ago
That's a really interesting thought experiment, and I don't really know what my answer is. The following choices assume that more people living an extra 30 years is a good thing ethically, and that there are no devastating effects environmentally or otherwise. The following choices also assume that you'll always have a monopoly on the production of the drug. 1. Assuming that the supply is practically unlimited, an altruistic actor should probably give it away for as close to free as possible. A purely selfish actor should probably try to sell to each individual for as much as they can afford (though this can be hard pricing strategy to enact). I suppose you might actually want to charge less than that even as a purely selfish actor, since you wouldn't want to somehow destroy the world economy by making everyone really poor except for yourself (would this actually destroy the economy though? I'm not sure... probably not relevant for the purposes of this thought experiment). 2. Assuming supply is limited, a purely altruistic actor will have to choose the most fitting recipients. This is a really tough choice. How do you define the most fitting recipients? Those who are most likely to contribute to the betterment of humanity with their extra 30 years? It seems like an almost impossible task to pick out who these people would be. A purely selfish actor should charge the richest as much as they're willing to pay. If it were me and supply were unlimited, I'd like to think that I'd sell it for as little as possible but enough to still never have to worry about money again. Making $10 million in profit would probably be more than enough (though if you can make more and still help everyone, that'd be even better). If supply were limited, I don't know what I would do. One thing I just realized though, is that even if supply were unlimited, if you had this godly power, you might choose to withhold it those you deem unworthy, which is a whole other ethical can of worms. I'm guessing this kind of deviates from the intent of your thought experiment though. What's your answer?

mirimir 8y ago

> But it's the only way scientific discoveries ever get to patients, because universities don't make drugs. Why not? If there are so very few patients, a lab could easily produce enough for them.

ineedasername 8y ago
Yeah, they had to make some quantity to run their trials. presumably it cost much less than $1mil/dose or they'd not have research funding at their level
- mirimir 8y ago
  Indeed. A lot less than $1million per dose, I bet. I understand that there are regulatory issues. But it seems pretty clear that the standard pharma industry model doesn't work for drugs like this.

iamleppert 8y ago

I was recently one of the first recipients of gene therapy for my condition. I have Hemophilia A, which is a bleeding disorder that results in bleeding into joints, muscle and soft tissue due to lack of a naturally occurring clotting factor that my body can't produce due to a defective gene. I received my dose of 30 billion viral particles about 13 weeks ago as part of a phase II clinical trial at UCSF. It is amazing to me that we can manufacture and program viral particles to target specific cells. In my case, my treatment is a non-nucleonic technique that didn't repair the actual chromosomal hereditary defect but inserted a working gene, additional and freely available genetic material into my liver cells. It's amazing that the body can just recognize this extra bit of code and just start producing the factor. The engineer in me is also impressed they can make so many copies of the engineered virus. It appears to be working. My factor levels have steadily increased and I’m now no longer a severe or even moderate hemophiliac. I’m their first patient to have achieved these results for hemophilia A severe and it puts me well into the therapeutic range. It really feels like I have been given a new body, it’s indescribable. For the first time in my life, I’m pain free. It’s a weird feeling. Pain, mostly from bleeds into my joints, has been my constant companion. I was never able to do sports as a kid and had to avoid many other activities that would put me at risk. I have mixed feelings about all of this. On one hand I’m extraordinarily grateful, and the other side I see what a huge disadvantage I’ve been at compared to others for all of my life. I thought this day would never come, I had resigned myself to my limitations but now they are gone in a matter of weeks through the miracle of a medical experiment. My old medication, a replacement clotting factor that was synthetically produced in a lab, worked but not nearly as well as this. Not to mention the fact it cost over $100,000 per month and I had to give myself an IV every other day. It's truly been a life changing experience for me, and I hope that gene therapy becomes widely available to others. Also, the thing that was a milestone in this case was that the Hemophilia A factor VIII gene has been notoriously difficult to create a genetic therapy for because of the complexity of the actual gene itself. On the scale of what the body produces, its one of the more complicated proteins. So it basically means many other genetic conditions will be able to be treated with and addressed with similar technology. Such an exciting time to be alive, and yes there are amazing things happening right now despite all the bad news you may hear!

homero 8y ago

anon2775 8y ago

If it works but it's not economically-viable: the moral and ethical choice is to open-source it or give it to a non-profit medical foundation. It sucks to have a rare disease because there isn't enough profit to recoup r&d; this is where non-profits, NGOs and GSEs should advance medicine where big pharma trades lives for profits.

timka 8y ago

That's probably the greatest example of the issue that Open Longevity was going to solve by combining patient organization with ICO funding to conduct clinical trials without profit driven big pharma. Unfortunately, that didn't happen…

platz 8y ago

> Pricing shouldn't be a political decision. It should be a rational decision based on merits and values," he said. The money quote. A rational decision? Maybe. Exactly what merits? Exactly which values? "Hundreds of millions of investor money has gone into the company" But none of that led to the development of the drug.

ada1981 8y ago

What’s up with this drug now? Sounds like it’s patents and IP protection should be up. I’d imagine you could get a single dose manufactured 1 off for $100k by a lab.