Fabry Patients Sue Genzyme Over Drug Shortage; After NIH Refuses To Allow Others To Make Fabrazyme
from the people-are-dying dept
One of the more horrifying stories of patents costing lives out there is the situation with Genzyme and Fabrazyme. As we detailed, not enough of the drug is being made allegedly due to manufacturing errors by Genzyme (for which it was fined $175 million by the FDA), and its causing serious health problems -- and even death -- to the sufferers of Fabry disease, who are caught in the middle of this. The patients asked the NIH to allow others to make the drug, via a "march-in" order, so that there would be enough supply, but the NIH refused, basically because it said that the patients (you know, the people actually suffering) couldn't make such a request. The end result, of course, is that lives remain at risk. Genzyme is apparently now rationing the drug, only giving 50% of the recommended dosage, despite a study suggesting that such reduced dosages are a problem:Neither Genzyme nor Mt. Sinai has tested whether the reduced dosage is either safe or beneficial for treating Fabry disease. In fact, in November 2010, The European Medical Agency determined that the lowered dose of Fabrazyme resulted in patients having more strokes, heart attacks, renal disease and other symptoms including severe GI distress and pain and burning in their extremities (neuropathy). It also determined that the lowered dose actually accelerated the course of the disease in some patients. Europe recommended in October 2010 that all patients be given a full dose of Fabrazyme instead of the lowered dose. Genzyme rejected the European assessment and continued to provide only unapproved low doses to patients in the U.S.The lawyer for some of those patients, Allen Black, alerts us to the news that his clients have now sued Genzyme and Mt. Sinai Medical School over the continuing shortages (pdf). Mt. Sinai is the actual patent holder, with an exclusive license to Genzyme. The patients are arguing for "compensation and punitive damages for lack of adequate treatment and Genzyme's failure to take adequate precautions including keeping a reserve inventory and providing second sources of manufacturing."
Part of the lawsuit is based on negligence claims -- and given the FDA fine, perhaps there's a basis for a negligence claim. The latter part of the case may be more interesting, however. I'm not sure how much of a chance it actually has, though I certainly sympathize with the plaintiffs. At a high level, it may be tough to show that Genzyme has any legal obligation to make enough of the drug (a moral obligation may be a different story). However, the lawyers make a very key point concerning patent law in making this legal argument: that the Bayh-Dole Act actually gives Genzyme a legal obligation.
The Bayh-Dole Act was designed to help universities better commercialize research in order to get those innovations out to market. For a variety of reasons, I've argued that the law has failed significantly in this regard, but these patients are picking up on a key part of Bayh-Dole, which is that the law was put in place to prevent patentees from denying citizens access to publicly funded inventions. It thus may require that certain standards be met when it comes to federally funded inventions -- which Fabrazyme certainly is. The law specifically says that it was put in place to "protect the public against nonuse or unreasonable use of inventions." And since the plaintiffs feel that this is the case here, they're arguing that they're now able to recover damages (though you'd have to imagine they'd prefer full doses). If that argument succeeds, it could lead to a very interesting series of follow-up cases concerning other federally-funded inventions.
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Filed Under: bayh-dole, fabrazyme, fabry disease, patents, rationing
Companies: genzyme, mt. sinai
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It would seems that producing this drug is difficult and expensive. The FDA has fined them, and forced them onto a "timetable" for changing their production methods. While it isn't clear, I would suspect that the modified production methods include significantly more post production inspections and more frequent production downtimes as machines are inspected and the line cleaned.
The Bayh-Dole Act is intended to keep medicines and developments from being held unused. That is not the case here. There is production, the school has fulfilled it's requirement to allow the development to be brought to market. The production is more difficult, and so only a certain amount can be made. But the trigger for Bayh-Dole is "non-use or unreasonable use", the plaintiffs have a pretty big hill to climb. The FDA has gotten involved and defined reasonable in this case, and Bayh-Dole doesn't look like it applies against FDA, which would be third or fourth party here. The school did what it needed to do, it signed a commercial agreement to produce the drug. Genzyme appears to have brought the drug to market, and the FDA has, through fines and setting a "timetable" defined the production process.
Further, if they do find a way to force another manufacture to be added (forcing another licensee), that would very likely create a whole bunch of legal problems between the school and Genzyme, who almost certainly signed an exclusive production deal. It could potentially lower the value to all parties of any such future contract, as there would be a legal trap door that third parties could use to break the agreement if they don't like how the drug is made or sold. That seems a pretty big risk, no?
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Not of you are the one that actually needs the medicine to live.
They could always grant a short-term license to another manufacturer to make more of the medicine until stocks are adequate.
What's amazing, and maybe this is hindsight, is why there aren't ANY safeguards to let the patent holders out of their exclusive agreements if there isn't enough product being made, like now.
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This isn't about feelings of "like" or "dislike", this is about lives "living" or "dying".
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When you break the financial cycle that includes payment to the developer, you remove the financial support that makes the research happen. Since you know this drug isn't being licensed for $1 total, that money would be put back into the school and it's research programs.
Would the research have happened without funding? What about the "next drug" that is currently being paid for with the money generated by this contract? If that contract was nullified by decree, would it change the way these contracts are done in the future, such that it would bring less money to the developer, and perhaps make it harder to finance future developments?
Also, is there any indication that anyone would want to get involved in producing something that is obviously difficult to make, all without getting any assurance of being able to stay in the marketplace? At what price would it be sold? Would the next story be about "overpriced meds"?
The question is always there: What would they do if the drug wasn't produced at all?
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Why couldn't Genzyme go back to Mt. Sinai get an expanded license allowing them to "sub-license" for limited production runs. In other words, Genzyme gets to offer the ability to other companies to manufacture the drug. They maintain their exclusivity since they're still in control of the overall license, they have the ability to revoke sub-licenses or define them in a way such that they expire.
This is one of the biggest complaints some people have with how the patent system works for medicine. It's been reduced to what can make a profit, not what can help make people healthy. In fact, there is zero interest in making people well because then there would be no need to purchase expensive medicines.
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As a note, Mt. Sinai and Genzyme are currently suing the only other manufacturer of a treatment for Fabry disease (Shire’s Replagal) in Germany and Sweden, even though there is a world-wide shortage of Fabrazyme. The amount of money they wish to recover against Shire is substantial.
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The current company can still manufacture the drug, but now they've got to compete - based on prior failures they'll have a reputational hill to climb, but they're not precluded from operating.
There should be consequences for this kind of crap. Serious, hardcore, let's get real penalties for putting people's lives at risk over patent/license/who did what when where's my cut bullshit minutiae.
Really, would it kill them to exist at the mercy of such a system? Yes? How's that feel, then?
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Welcome to slippery slope, USA. What you propose it to basically add an asterix on the patent and copyright system that says "if someone complains, we can take your rights away". That would basically drive a stake though the heart of the system, and render it meaningless. I suspect hat it what you would want.
Patient risk is only one issue here. What were these patients doing before this medication was developed? Without a valid and functioning patent system, would this medicine have come into being so soon? Or would it have come 30 or 50 years from now, such that all the current patients would be dead anyway?
What you are proposing is an extreme short term and narrow gain for long term pain. That isn't going to work out.
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FTFY
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This same law could be used to lower ridiculous drug prices if profit margins are set too high.
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eminent domain
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Re: eminent domain
So, if IP is subject, could a government transfer ownership of IP from a resident/citizen, to a bigger company, claiming that the business can make better use of the product, generate more jobs, etc?
I can't imagine that would be abused...
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Re: eminent domain
BTW, the exercise of march-in rights would not be based upon eminent domain, but on contract. Hence, there would be no requirement for the government to pay "just compensation" as mandated by the Fifth Amendment when private property is taken for public use when march-in rights are employed.
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People are Dying
Sometimes laws are stupid, this is a good example. Make the drug, save the people. After all, I did help pay for that drug, so should I not get a say?
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Something that gets lost here...
-C
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As written about in your first post on this topic, the govt. didn't allow other companies to step in and make the drug because Genzyme would have their production facility back online before anyone else would have been able to begin production. Genzyme had to fix problems, others would have to go through the approval process from the start, which would take about 2 years.
Genzyme testified they should be back up and running within a year. Its not like Genzyme doesn't want to produce it, they have to fix problems before they can being producing it again.
This is an orphan drug, meaning it doesn't affect many people, which means typically no one wants to produce it. This is why these types of drugs gain special consideration from the govt.
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Tell us it isn't so!
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From just last week:
http://www.msnbc.msn.com/id/41994697/ns/health-pregnancy/
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Nice logic there!
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You are not addressing the topic at hand yourself, so at best, welcome pot. Or more realistically, hello troll.
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Wow, you guys are actually sorta engaging in journalism, or at least in being among the first to get the news from a related (though sometimes one sided) source and then relaying it. Who needs newspapers anymore?
and to say that this isn't journalism because the source came to you I think isn't true, journalists often do report on information that related parties have given them and they perhaps add their opinions and criticisms so that the story won't be one sided (though, in this day and age, mainstream media journalism is often very one sided).
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In order to discuss what is associated with this matter, it does help to look at the NIH's reasons.
They can be found at:
http://freepdfhosting.com/d30ee703c3.pdf
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Why would the NIH need another company to go through years of testing on a drug that has, presumably, since it's already gone to manufacture and sale, been tested already?
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repercussions
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