9 ms·
The million-dollar drug (2018)
- d136o 5y agoIt seems to me that it’s not just the price of a single treatment that is too high, but also that the patients are fully recovered and don’t have to pay a recurring annual subscription fee to live normal lives. Or is that too cynical?
- refurb 5y agoNo, because other gene therapies like Zolgensma are priced at $2.5M for a single dose and selling quite well. There is a huge market for these types of cures.
- DoreenMichele 5y agoOr is that too cynical? My, you innocent child, that you have to ask this. (I have a genetic disorder commonly treated with one of those drugs that cost $300k annually.) Edit: I was going to delete this in short order but now that it's flagged, I'm leaving it. People with genetic disorders get all kinds of hatred from the world and medical system for just wanting to be healthy and how dare we say that online.
- isoprophlex 5y agoDiscussing the value of a human life, and figuring out how much the many are willing to sacrifice in order to treat the needy few, isn't something that should be out of bounds. I don't think the voices of the few should be silenced. Its uncomfortable maybe, but I think not a discussion to avoid.
- deleted 5y ago[deleted]
- imtringued 5y agoOur economic system requires endless amounts of busy work. If that busywork doesn't exist people will compete for a handful of jobs because they cannot comprehend the idea of doing less work per person. If that busy work consists of keeping people alive and advance medical science it's at least a better use of human potential than working via door dash.
- kungito 5y agoDoesn't it say 10 years? So you'd need a few more during your life
- 14 5y agoNo that is not what they implied just that so far it has only been 10 years. These people are still healthy but the company can't say it lasts 30 years because no one has lived long enough to see if it does. It may last indefinitely.
- dodobirdlord 5y agoIt is in fact too cynical. The company that wanted to sell this drug isn’t the same as the companies selling recurring treatment to people with this disorder. Your cynicism has blinded you to the fact that the marketers of this drug do not have any economic motivation to do what you are suggesting. Their economic motivation is to sell the tiny number of doses required to treat everyone in the first world (2,000-3,000) at a price high enough to recover their investment and make a profit.
- mmazing 5y agoWhy not look for funding from the many sources of grants and charities that might not care about economic motivation? Your comment seems very defeatist. Actually this whole thread seems defeatist.
- s1artibartfast 5y agoThere are not many charities and grants willing to shell out a million dollars to improve one persons quality of life.
- kkjjkgjjgg 5y agoArticle explicitly mentions price is high because there are no recurring costs, so it actually isn't more expensive than other treatments that may cost hundreds of thousands every year. Iirc economists have estimated that a human life is valued around 3M$ by society (a couple of years ago, probably higher now), the price doesn't necessarily seem too high.
- at_compile_time 5y agoHow long does the patent last? It should be made available as a generic drug when the exclusivity period elapses, right?
- folli 5y agoThey usually last around 10 years with some options to extend for another 5-10 years. By the way, that's one of the reasons drugs are so expensive: pharma co. needs to recoup the costs before the patents expire.
- BizarroLand 5y agoThe flip side of patent expiration is that a rival company would need the infrastructure and capability of manufacturing the drug. For something like Aspirin, that's not a high bar to clear, an avid experimenter could probably do that in their basement, but for gene therapy drugs I feel confident in saying that it's orders of magnitude higher. Then again, they could kickstarter it and say that if they get X orders or raise Y money they'll do it, make and sell the 20-30k doses for everyone and then close up shop with a healthy profit.
- WalterGR 5y agoFYI, the drug is (trade name) Glybera (generic) alipogene tiparvovec. Wikipedia: https://en.wikipedia.org/wiki/Alipogene_tiparvovec https://en.wikipedia.org/wiki/Alipogene_tiparvovec From the submitted article: "...The first drug ever approved that can fix a faulty gene. It's called Glybera, and it can treat a painful and potentially deadly genetic disorder with a single dose — a genuine made-in-Canada medical breakthrough..."
- nefitty 5y ago"... Her blood becomes thick and white with fat particles that can destroy her pancreas. All her life, Turcotte has had to follow a strict diet. She can't eat cheese or chocolate or any food that contains fat. And she can't drink even a drop of alcohol." Jesus.
- grp000 5y agoBetter than not knowing what's killing you.
- DoreenMichele 5y agoI wish I knew why these patients can't have alcohol. I mean the biological mechanism. What the relationship is between misprocessing fat and alcohol being deadly.
- tsol 5y agoNot sure exactly, but considering alcohol messes with hormonal signaling I'm guessing it throws something out of balance Edit: Looked up the wiki article, here is their explanation; >Additional measures are avoidance of agents known to increase endogenous triglyceride levels, such as alcohol
- DoreenMichele 5y agoThx. https://pubmed.ncbi.nlm.nih.gov/23511381/ https://pubmed.ncbi.nlm.nih.gov/23511381/ Probably more pertinent: Alcohol changes the structure of the liver cells and slows down the liver’s ability to process fats. This is the second way that triglycerides begin to accumulate and the levels rise. This is also the beginning of fatty liver disease. https://findpulse.com/what-liquor-raises-your-triglycerides-the-least/ https://findpulse.com/what-liquor-raises-your-triglycerides-...
- tsol 5y agoOh interesting find. Makes sense
- d136o 5y agoI am equally curious. Fatty liver can be a consequence to high alcohol consumption, but people can also have NAFLD. My guess (IANADr) is that alcohol leads to excessive build up of fat in their bodies. NAFLD is also highly correlated with diabetes, so I wonder if there could be something to learn from studying this or related genes.
- deleted 5y ago[deleted]
- exhilaration 5y agoI'm confused, they say it was priced at $1 million because that was what they felt the drug was worth compared to the ongoing therapies needed to keep these patients alive. Ok fine. But once it became clear no one was going to pay that price, wasn't there some lower price that would still earn them a profit?
- joe_the_user 5y agoIt's a matter of principle. Perverse principle but principle: "Why would we? [lower the price] 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?"" Which is to say that you have an investor group that expects to either be paid or they'll take their medicine away and the public be damned. Or perhaps a perverse process. The system is how you get new, actually amazing drugs. The system is also how actual health care has become really terrible in the US in particular.
- nine_k 5y agoBuying the rights on drugs like this, and more important expensive drugs, an license them under a freedom-protecting license (similar to GPL) could be a major application of charity funds.
- s1artibartfast 5y agoYou would still have to raise several hundred millions of dollars of funding to reconduct development and clinical trials for the drug after securing the IP. Clinical trial data and manufacturing know how cant be leveraged from the original approval. It is incredibly difficult to get investors to fund a second round of development for a product that made it to market and still failed. You might say this is an opportunity for the government to step in as investor, but they have little interest, knowing that they could use the same money to save many more lives in other areas.
- 5y ago
- DantesKite 5y agoI'm surprised how well this essay was written. It was short and terse where it had to be.
- jagger27 5y agoI can’t imagine how hard it must be for the dozens of people out there suffering from this rare ailment knowing a perfect cure/treatment is possible but not worth it for the shareholders.
- 14 5y agoIn a world with nothing to loose I am surprised one of those people doesn't go John Q.
- epicureanideal 5y agoOr alternatively, try to hire some chemistry grad students to make some.
- DoreenMichele 5y agoThat might be surprisingly feasible. https://www.resetera.com/threads/guy-cures-himself-of-lactose-intolerance-via-home-made-virus-biotech.23013/ https://www.resetera.com/threads/guy-cures-himself-of-lactos...
- jfoster 5y agoIf he can do this at home, why hasn't a pharmaceutical company made something equivalent for the incredibly large population of lactose intolerant people around the world?
- DoreenMichele 5y agoIt wasn't done at home. It was done in a lab by someone who majored in biology for the express purpose of finding a cure for his lactose intolerance. It's been a bit since I watched the two videos* he made, so I don't remember the details. But it was the first time I ever felt anything positive about gene therapy. I'm too used to seeing people like me treated like Frankenstein's monster so people can do cool science and not care about the suffering of their human guinea pigs. But most likely the short version is that he was only using this on himself. He didn't need to get FDA approval or do clinical trials. That's an enormously frustrating process for the folks with incurable conditions who just tear their hair out over decisions to deny approval because something bad might happen down the road. We tend to feel like "Let me live long enough to have those problems, you ass!!!!" But those processes exist for a reason. (Look up Thalidomide if you care to know how wrong things can go.) Maybe someday there will be a readily available gene therapy for lactose intolerance. Maybe his work will be the starting point for it. * He has made a lot more than two videos but there are two specifically about this gene therapy he administered to himself.
- stefan_ 5y agoNever mind non-practicing entities for software patents, but surely for patents on medicine keeping them valid when no one is making any is entirely bogus and not deserving of legal protection?
- dodobirdlord 5y agoIf you invalidate people’s patents for trying to sell their drug at a price high enough to recover R&D costs, they’ll just stop doing R&D for any drugs that will have niche target populations.
- carlmr 5y agoThis isn't the same though, this is invalidating patents for drugs that have been developed that no one is producing anymore. Patent protection makes sense to inventivize development and production. In this case development already happened and only production stopped. So it doesn't make sense to uphold this patent.
- dodobirdlord 5y agoIf losing your patents in a position like this is a possible outcome, it changes the investment calculus. As it stands, there’s still the possibility that administrations or laws will change and governments or insurance companies will come around to wanting to buy it at a price that recovers the investment, or some other company will believe that they can persuade buyers to buy at a worthwhile price and buy the patents or the production rights off of the current owners. Or maybe some other company will profitably deploy a derivative technology and they can sue to recover some of their investment. For all of these reasons the patents and trade secrets are still worth something. If finding yourself in this position means that you are stripped of your patents and receive nothing, that makes drug R&D an even riskier investment proposition. Being stripped of patents by the government after having bankrolled clinical trials because the government refused to pay the price required to recover the investment would be a brazen show of bad faith and a real stab in the back. The patents will expire in a decade or so anyway, do we really need the government to go out of its way to screw these people out of their last possibility of making back some of the money they spent on this socially valuable work? If we think that it’s so important that this treatment be made immediately available to people at a low price, maybe the government should buy the patents early for the several hundred million dollars that they cost to produce?
- flipbrad 5y ago"Use it or lose it" / compulsory licensing doesn't seem like a terribly bad idea sometimes.
- dodobirdlord 5y agoThat would probably just result in keeping a dose around on a shelf somewhere in case anyone wants to pay $1M for it.
- shmerl 5y agoPrice should be based on cost of production, not on how often the drug is needed. So stupid not to make it available because someone was crazy to price it that high.
- dodobirdlord 5y agoA drug with high cost of production that’s not needed very often will cost a lot of money per dose. The article mentions that hundreds of millions of dollars were spent on R&D and clinical trials, and maybe a couple thousand people in the first world have this disorder. Development costs have to be recouped entirely off of first world sales, since the rest of the world isn’t going to ever pay any appreciable amount of money for a drug, so even if they could sell this to everyone in their target market the breakeven cost seems like it would be about $100,000. Since they’re not going to be able to sell to everyone (there are limits on what various governments and private insurance will pay for medical treatment), and since ongoing care for this disorder costs hundreds of thousands of dollars a year, $1M/dose seems like a perfectly reasonable price.
- shmerl 5y agoHow much does it cost now, after all the R&D and tests are completed? I doubt they need millions to actually produce the drug once they know how to and that it works. So they basically prefer not to produce it at all, than to sell it at affordable prices. It makes no sense to me.
- s1artibartfast 5y agoI think it makes no sense because you are greatly underestimating the cost to produce and market the drug, even after sunk costs. It could easily be many hundreds of thousands of per dose.
- shmerl 5y agoMay be, but probably not a million. So they overpriced it and ended up not making it available at all. Also, I don't think marketing is a big issue for such drug which is the only available option for affected people.
- refurb 5y agoIt doesn't make for a very compelling news story, but the real reason why Glybera failed is because it's not that great of a drug and it loses effect over time and you can't redose with the same AAV vector. They also relied a surrogate endpoint versus actual clinical measures (as those were to confounded by the small sample size and patient variability). It just barely got approved by the EMA and based on feedback the company didn't bother to pursue FDA approval.
- deleted 5y ago[deleted]
- lotsofpulp 5y agoThanks for the info, much more helpful than the usual outrage and cynicism.
- legulere 5y agoSimilar drug still on the market: https://en.m.wikipedia.org/wiki/Onasemnogene_abeparvovec https://en.m.wikipedia.org/wiki/Onasemnogene_abeparvovec
- BizarroLand 5y agoSo they solved the problem of paying one million dollars with an already developed drug by making a new drug that costs... $2.125 million. "Novartis' Zolgensma, a one-time gene therapy for spinal muscular atrophy tops the list with a price tag of $2.125 million." https://www.biospace.com/article/gene-therapy-zolgensma-tops-goodrx-list-of-10-most-expensive-drugs/ https://www.biospace.com/article/gene-therapy-zolgensma-tops...
- thinkloop 5y agoEveryone is blaming the company or the patent system or general greed, but really there is an extremely simple solution: pay the $1M. In Canada and Europe it's medicare, ie taxes, ie the general population that would pay for it, but they refused. In the US it's insurance companies, ie the general population (as insurance is just socialized losses), that refused. If people are uninsured then force the state, ie everyone who is feeling so bad about it, to pay for it. It's odd that being the genius inventors also means that you now also have to be the only ones to pay for it. This isn't a story of exorbitant profits - even at a million a dose they would barely make their money back. It makes no sense to isolate the group of people who are actually working on the thing to be the only ones to pay for it. By extension, this story demonstrates how we price life. What if the cure was a billion dollars, is that worth society paying? What about a trillion? At one point a limit is reached, and it seems that one million is one of those limits that we all quietly agree upon.
- ak39 5y agoFascinating story and the use of virus to repair genetic anomalies. What other uses can there be of similar conditions? And why aren't we hearing more of it (if the approach is viable)?
- theskypirate 5y agoSociety itself is collectively responsible for this failure, not the patent owners. Funding could have been provided through charity, or through legislation. But this was not done, and instead development of this drug was left up to profit seeking investors. It isn't the investors' duty to bankrupt themselves to produce this drug. They tried to find a way to get this produced through the private sector, and they found that it was impossible. It should instead be handled through the public sector, and it is the general public who are ultimately responsible.