File this under 'if at you first you don't succeed...' After being rejected last month by the National Institutes of Health in a bid to override the patent on a Genzyme drug that is in perpetually short supply, a pair of Fabry's disease patients are now petitioning the FDA to insist that overseas stock of the med is first made available to US citizens.
The move comes amid ongoing difficulties Genzyme has encountered in producing sufficient supplies of Fabrazyme due to severe manufacturing problems that began in 2009. These prompted a $175 million fine, a consent decree and a takeover bid from Sanofi-Aventis. Existing patients, meanwhile, are subject to inadequate rationing and newly diagnosed patients are unable to receive any meds.
To rectify the situation, some patients last summer asked the US Department of Health and Human Services last summer to override the use patents held by Genzyme on its med, which is the only such treatment currently available for a rare, inherited disorder that causes kidney and heart problems. Last month, though, the NIH turned them down (back stories here and here).
Now, the patients want the FDA to level a playing field in which they say 62 percent of Fabrazyme made in the US is allocated to overseas patients, even though those patients have access to an alternative, Replagal, which is made by Shire Pharmaceuticals. While the European Medicines Agency recommended Fabry's patients switch drugs, Shire is not currently seeking approval to sell Replagal in the US.
"The result is the tragically ironic situation that there is no drug shortage overseas," Allen Black, the lawyer for the patients, writes us. "There have already been three deaths reported in the US under the rationing, but none reported overseas. Even more bizarre, is that the FDA is importing Replagal to replace the Fabrazyme that was shipped overseas in the first place. However to receive treatment, the patients must show 'critical medical need' on an individual basis."
And so the petition sent to the FDA asks the agency to allocate Fabrazyme first to US patients and, in the event the request isn't granted, that patients be allowed to present their case for medical need before Fabrazyme is sent overseas (you can read the petition here). Is this reasonable? What do you think the FDA should do?
Should the FDA Grant This Request?
- Yes (87%, 92 Votes)
- No (13%, 14 Votes)
Total Voters: 106
17 Comments
I don't own stock in Genzyme, but I sure don't understand why the stockholders don't rise up and toss the entire management team of Genzyme out on their collective ears. The manufacturing problems have gone on for YEARS! And the argument that "these proteins are hard to make" is starting to lose steam as it appears Shire is managing to accomplish the very same feat.
Thanks for posting this Ed. I hope that it helps vulnerable patients access their life saving medicine. Sanofi certainly seems to have picked a good time to try to but Genzyme, but I worry that defending themselves if causing Genzyme to completely lose focus.
It seems to me that Genzyme would rather protect its market share by selling in Europe than supply its U.S. patients. It may be a good business decision but not a particularly palatable one.
The US price of Fabrazyme is likely higher than the EU price; if the US Govt. demands it they don't have a choice; patients are better supported and don't die from rationing; Genzyme makes more money on its limited supply of product.
Other than the precedent setting I am not sure I see much of a downside.
I believe in capitolism and the ability to profit from individual or corporate discoveries. However, I believe even more in the Human Race and compass of common morality clearly points in the direction of making this drug available to all. Patents are important and profitable protections but people are special and must be considdered... allow other manufacturers to produce your drug with a portion of profit coming back to you and accessibility to many.
The government does not belong in ANY issues regarding how/when/where a company chooses to export its products. My family has suffered greatly because of the shortage but I remain unwilling to relinquish any liberty to try to fix this situation. Over zealous government policies come in many forms, this is one of them.
US money funded the research that made this treatment possible and now US patients are being denied access? Talk about denying liberty...how is that just? This goes beyond politics and red tape. It simply isn't right. People should not be allowed to suffer, die or have life-threatening setbacks because a company holds a patent and can't meet supply demands because they have decided to export. Either allow another company to come meet the demand of patients or demand that Genzyme changes their distribution of this government funded treatment. Enough is enough. The government DOES have an obiligation to help its citizens.
I think it's ridiculous that this treatment is being provided to the country that funded it either...all for the love of profit. If this were something like grain or wheat that we were talking about I would maybe not be so offended by what some people are saying on this blog but since it's human life at stake I take it much more seriously and so should you. Money isn't everything....
As much as I like rainbows and unicorns, the fact is this: Money IS everything to a company. The laws in place that allow a company to corner the market are flawed. This process must be changed to eventually have a positive effect on the market. As superior as we may think we are, American lives are no more precious and important than any one else's lives.
Megan, I make my comments keeping in mind that you said you are affected by the shprtage as well, but I am not sure how they would be losing money making the drug available to Americans first. Since there is no competion here, unlike Europe, they can set the price without regard to competition. As a Fabry patient living in the US, I would hope that a company would consider its obligation to provide the drug to its home based constituents first as we have no alternative at this point. If the company cannot honor its obligation to provide medcine to a suffering community, the government should step in and provide other sources.
Jeff, I was not really commenting on whether or not Genzyme would lose or make money, just addressing the previous comment that "money isn't everything. No matter how you try to package it, free enterprise MUST be allowed to win out. No government interference should occur. What about the Americans on Replagal right now. What would happen if the world was suddenly forced onto Replagal because of American supremism? Then that could create a shortage of Replagal, forcing U.S. citizens as well as less important citizens of the world (note tongue in cheek)into a position of not having a drug available to them. The market MUST work this out on its own without Government interference. The Government does not hold all of the answers and typically tends to make matters worse. Everyone in the U.S. simply needs to ride it out while the market corrects itself.
Megan: I don't think that this is an issue that the free market can correct, at least not in its current regulated state. Government interference has already occurred. Other companies cannot begin production of Fabrazyme because it is patented. Americans cannot buy Replagal without showing "critical medical need" because it is not approved in the US. Of course if these restrictions were lifted across the board, the pharmacutical industry as we know it would cease to exist. Genzyme could correct the problem if they could fix their production issues, but this seems to be difficult for them despite the obvious market incentive to fully meet demand. A single producer is not "the market" and shouldn't be expected to show the same self-correcting tendencies.
Matt, If you read my previous posts, you will see that I have stated that the government needs to butt out of the process completely. It is government interference that has caused this problem already, more interference is NOT the answer to fix it. Period.
Megan,
Your first post stated that "the government does not belong in ANY issues regarding how/when/where a company chooses..." I imagine given that stance, that government should not in ANY way choose / fund what a company researches in product development. For consistency in argument you have to agree with that. But without government funding there would not be Fabrazyme as the R&D for the product and clinical trials was primarily funded by the federal government for the free market did not have incentive to fund R&D for rare diseases / orphan drugs that are life-saving. I suppose you are right the government interference caused this problem. Sad - the only word that I can use to describe the posted positions.
I find it troubling that you attempt to "one up" someone that does not agree with you by making silly comments such as "sad". I will repeat my position ONE last time, the government does not belong ANYWHERE in the pharmaceutical business. Due to the fact that taxpayers funded research and develpment of enzyme replacement therapy drugs, shouldn't your beef be with the federal government? If we funded the research and development, then why was Genzyme given the exclusive right to profit? Why did the government not grant access to more than one company to market, sell and make profits from the drug? Hold on, I thought that the reason Genzyme was given exclusive rights to the patent was to reimburse THEM for developing the drug. Hmmm.... something about this situation does not sit right with me. That's okay, I am not thirsty for any Kool-aid right now, guess I'll go off and be sad : (
I agree with Jeff: "As a Fabry patient living in the US, I would hope that a company would consider its obligation to provide the drug to its home based constituents first as we have no alternative at this point. If the company cannot honor its obligation to provide medicine to a suffering community, the government should step in and provide other sources." I feel fortunate to have gotten on Replagal via the compassionate use clinical trial. I personally was getting sicker and sicker on partial dose. It has been almost a year that I have been on Replagal and I pray I can stay on it. We (US citizens) have no option unless we got lucky enough to get in the compassionate use trial. Genzyme may have spent money developing Fabrazyme but a great deal of the development of Fabrazyme was subsidized with US tax $ and at the NIH, so yes, I do think the first priority should be to the US. Sorry if some do not agree.
Megan: You really need to get your facts straight. Genzyme did not develop Fabrazyme - Mt. Sinai did, with NIH funding support. And CAPITALIZING your words does not strengthen your agruement. I suppose that under your "government does not belong anywhere in the pharmaceutical business" that if I wanted to market shredded glass chewing gum as an anti-smoking drug that would be just dandy. How about crack? We need more creative minds like yours, those that can throw out overgeneralizations without any logic to back them up - the world would be a much nicer place. Do us a favor - drink up!