Fabry Patients Sue Genzyme Over Drug Shortage; After NIH Refuses To Allow Others To Make Fabrazyme

from the people-are-dying dept

Thu, Mar 17th 2011 7:22am — Mike Masnick

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

37 Comments

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Marcus Carab (profile), 17 Mar 2011 @ 6:43am

I don't really believe in "evil" - but sometimes pharmaceutical manufacturers make me question that conviction...
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Anonymous Coward, 17 Mar 2011 @ 7:43am

While on the surface it seems pretty simple, there are a couple of things here that just need to be covered.

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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Spaceboy (profile), 17 Mar 2011 @ 7:56am

Re:
"That seems a pretty big risk, no?"

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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Don, 17 Mar 2011 @ 7:59am

In the case where drugs are needed urgently and demand can't be met there should be a "medical patent marshall law" that would invalidate a patent until demands are met and patients are essentially out of the woods.

This same law could be used to lower ridiculous drug prices if profit margins are set too high.
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CompTroll, 17 Mar 2011 @ 8:13am

eminent domain
So, is intellectual property subject to eminent domain? They keep likening it to tangible assets, so it would stand to reason that the Govt. should to seize a patent for the common wealth.
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Anonymous Coward, 17 Mar 2011 @ 8:23am

Re: Re:
Just for fun, can you please give me a legal definition of "enough"?
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Derek (profile), 17 Mar 2011 @ 8:24am

People are Dying
Laws are a set of rules, Patents are part of the grand set of laws we have. Once a law, or part of a law (patent), starts doing things that hurt others or are just morally wrong. It should not be a law anymore.

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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David Collado (profile), 17 Mar 2011 @ 8:26am

Re:
"...to break the agreement if they don't like how the drug is made or sold."

This isn't about feelings of "like" or "dislike", this is about lives "living" or "dying".
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Ali, 17 Mar 2011 @ 8:48am

First of all Genzyme did not bring the drug to market. I am patient of Genzyme's bread winner Cerazyme. Both drugs where funded primarily by NINDS under the premise of orphan drug act. Genzyme was created because the government can not produce and distribute a pharmaceutical drug. Now that Genzyme is a company and has investors and apparently morons running the show they have contaminated the drugs which they alone are responsible for. They should be sued for this simple fact that they in fact also distributed contaminated drug to patients negligently until the federal government stepped in and told them they could not. The government also bears some responsibility in the fact that they did not monitor the company they choose to hand the drug they developed to and instead gave them free reign. I know because I was there in 5th floor of the Clinical Center when the drug was called Ceradase. So I say sue there asses off.
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Anonymous Coward, 17 Mar 2011 @ 8:52am

Re: Re: Re:
Enough what? People that suffered or died due to pointless robo-lawyering?
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Christopher (profile), 17 Mar 2011 @ 9:09am

Something that gets lost here...
... is that pharmaceutical companies are really, really bad at manufacturing. Marketing, great. Sales, great. Even discovery, great. But actually making the drug? Ridiculously inept. Manufacture should be something you outsource immediately to companies where SCO are actually well-implemented and driven to success by the pressure of competition. Teva, for example, would be a much better manufacturer for most drugs than Merck, J&J, Pfizer, etc. Without the high-margins that big pharmas take for granted, Teva (purely as an example) has to make its money by actually being very, very good at manufacturing.

-C
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Chuck Norris' Enemy (deceased) (profile), 17 Mar 2011 @ 9:17am

Re:
So, ladies and gentleman, we see that, once again, the government gets involved and totally screws things up. At least, sufferers of Fabry can get 50% of the dose they need. That is up from zero.
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Anonymous Coward, 17 Mar 2011 @ 9:49am

So much misinformation in this article. Maybe not misinformation, but your focus is slanted.

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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Anonymous Coward, 17 Mar 2011 @ 9:50am

"The lawyer for some of those patients, Allen Black, alerts us to the news"

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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Anonymous Coward, 17 Mar 2011 @ 9:55am

Re: eminent domain
that is the scariest thing I've heard this year. As the government moves more and more into the corporate pocket, will there be a ruling that allows eminent domain tranfers of IP? I know that in some cities, they can take property that they believe is "underutilized" and transfer rights to a new developer, who can build *things* that are good for the local economy/city, like strip malls and parking lots.

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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Berenerd (profile), 17 Mar 2011 @ 9:56am

Re: Re: Re:
Enough = Sufficient supply so that those who need the medication can get the medication when needed and in the amount needed as deemed by doctors.
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Anonymous Coward, 17 Mar 2011 @ 10:16am

Re: Re:
You have to consider the alternatives: Drug not made at all.

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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Mike C. (profile), 17 Mar 2011 @ 10:16am

Re: Re: Re:
While not necessarily a "legal" definition, I think most would agree that failing to product sufficient quantities to prevent patient DEATH (you know - that generally irrevocable event that causes a person to become an ex-person) might qualify as "not enough".
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Mike C. (profile), 17 Mar 2011 @ 10:22am

Re: Re: Re:
Since we're taking it in unrealistic directions, how about another one:

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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Allen, 17 Mar 2011 @ 10:29am

Re: Re: Re:
To address Anonymous Coward, innovation and ensuring a reliable drug supply go hand in hand� you must accomplish both to save lives. Obviously, a child that is denied access to a life-saving drug by a shortage will die just as surely as if a drug company never invested in the first place. Nobody should have to choose between a having a reliable drug supply and promoting innovation. They are simply not mutually exclusive.

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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Anonymous Coward, 17 Mar 2011 @ 10:42am

Re: Re: Re:
Here's another question: the gov't. giveth, it can taketh away, so how about the current monopolist gets stripped of that monopoly due to proven poor stewardship? Throw it out to competitors or any co. willing to fulfill the obligations that were not met by the current company?

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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Killer_Tofu (profile), 17 Mar 2011 @ 10:59am

Re: Re:
From the article though it should be noted that not getting a full dose could actually be more harmful than getting none at all. Maybe they didn't take the time to study what would happen if the company the would license the patent to would be screwing over those in need of the drug.
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Anonymous Coward, 17 Mar 2011 @ 11:28am

Re: eminent domain
Short answer is "yes".

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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Anonymous Coward, 17 Mar 2011 @ 11:36am

Re: Re: Re: Re:
so how about the current monopolist gets stripped of that monopoly due to proven poor stewardship?

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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Anonymous Coward, 17 Mar 2011 @ 11:38am

Re: Re: eminent domain
Worse, imagine a company that feels pressured to bring product to market, but in their rush to avoid eminent domain screws up the product, and ends up killing people? Medicine isn't an area where you want companies to rush to avoid some arbitrary system of "snatch and grab". It is one of those incredibly poorly thought out ideas.
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Anonymous Coward, 17 Mar 2011 @ 11:51am

Re:
So what you are saying is that Mike once again didn't get enough information, and appears to have run off to slam the "greedy pharma companies" and the patent system without realize what all was involved?

Tell us it isn't so!
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Anonymous Coward, 17 Mar 2011 @ 12:00pm

I made an earlier post that for some reason never appeared here.

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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Any Mouse (profile), 17 Mar 2011 @ 12:45pm

Re: Re: Re: Re: Re:
'What you propose it to basically add an asterisk on the patent and copyright system that says "if PEOPLE DIE DUE TO NEGLIGENCE IN PRODUCTION, we can take your rights away".'

FTFY
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Anonymous Coward, 17 Mar 2011 @ 1:30pm

Re: Re: Re: Re: Re: Re:
Thanks for pointing that out. :) I had included the word 'proven' for a reason, and it was quoted but ignored.
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Anonymous Coward, 17 Mar 2011 @ 1:36pm

Re: Re:
Yeah, 'cause it's such a huge leap to make 'n all.

From just last week:

http://www.msnbc.msn.com/id/41994697/ns/health-pregnancy/
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nonanymous, 17 Mar 2011 @ 5:37pm

Re: Re:
So what you are saying is that Mike once again didn't get enough information, and appears to have run off to slam the "greedy pharma companies" and the patent system without realize what all was involved?

You are not addressing the topic at hand yourself, so at best, welcome pot. Or more realistically, hello troll.
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Anonymous Coward, 17 Mar 2011 @ 9:06pm

Re:
I don't know much about this stuff - except that it makes me angry that people are made to feel frightened, hopeless, and abandoned on top of being or close to someone seriously ill - but something there seemed weird to me.

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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The eejit (profile), 18 Mar 2011 @ 1:17am

Re: Re:
For the dollars. In America it's always about the dollars. At least, if you're a Tea Partier, it is.
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Anonymous Coward, 18 Mar 2011 @ 7:31am

Re: Re: Re:
I have to laugh. What you are saying is that Mike can be judgemental of the pharma industry because of what another player in the game did. By your logic, the US should be locking up all the black people because some of them have committed crimes.

Nice logic there!
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Anonymous Coward, 18 Mar 2011 @ 4:21pm

Re: Re: Re: Re:
"Nice logic there!" he said, after making a false analogy, equating an obscure blogger's opinions with the actions of a government. He was minimizing the human side as best he could, understandably, as it allowed him not to care. He'd likely never met someone with the condition. Few people had. The concept of not being able to sweat had never occurred to him, nor the idea of running a fever because his body was unable to regulate its temperature. Maybe he'd felt a storm coming in an old sports injury or some creeping arthritis in a few joints, but never had every joint in his body screaming at him with nerve pain. Perhaps if he had, and felt the joy of being able to exercise for once, relishing every bead that dripped off his forehead; to walk, even run, on a spring day without having to dunk his head in a tub of ice water afterward; to finally play sports with the other boys, though in his mid-20s; and then had that slowly, creepingly stolen away again by a company's incompetence, he'd be less willing to advance a monetarist agenda at the expense of others' well-being.
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patent litigation, 20 Mar 2011 @ 4:39pm

repercussions
I agree that this case could have significant future repercussions for patent litigation, in regard to Bayh-Dole. I hope you will keep us updated on its progress.
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Shine (profile), 3 Mar 2012 @ 12:41am

They obviously are not concerned about the people..their concern is only what they can get from the people. So sad.
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