Rocketboom Founder Fighting for Father's Life
October 14, 2008 8:36 AM Subscribe
RocketBoom's co-founder Andrew Baron found out last week that his father had Multiple Myeloma, and likely less than 48 hours to live. Then a miracle occured. A drug that could save his father's life existed. However the drug was not approved by the FDA to be used this way. They sought and quickly got approval from the FDA. But now, the drug's manufacturer Biogen won't approve usage despite pleas to Biogen's president from Lance Burton, President Clinton, and others. Read this open letter and request for help from Andrew to learn what you can do to save his father's life.
Yes. Another drug is extending his life somewhat, but 'Tysabri' is the only the researchers found that was a 100% match against the anti-bodies.
posted by IndigoSkye at 8:45 AM on October 14, 2008
posted by IndigoSkye at 8:45 AM on October 14, 2008
Why don't you read the link and find out:
"After you refused on Friday, the doctors decided that they must do something, and resorted to another antibody called Campath which is working to prolong his life."
posted by Manhasset at 8:45 AM on October 14, 2008
"After you refused on Friday, the doctors decided that they must do something, and resorted to another antibody called Campath which is working to prolong his life."
posted by Manhasset at 8:45 AM on October 14, 2008
I'm guessing this is his father?
I'm also guessing the CEO of Biogen is a Republican.
posted by vacapinta at 8:54 AM on October 14, 2008
I'm also guessing the CEO of Biogen is a Republican.
posted by vacapinta at 8:54 AM on October 14, 2008
from Lance Burton...
It's actually Lance Armstrong and not the Las Vegas magician.
posted by ericb at 8:55 AM on October 14, 2008
It's actually Lance Armstrong and not the Las Vegas magician.
posted by ericb at 8:55 AM on October 14, 2008
How did Lance Burton get involved in this?
Back on topic -- I suspect there's way more to this story than what we can learn from these links. FDA, bioethics, politics, business, insurance - one huge jumbled mess. I hope the man gets the medicine he needs.
posted by davidmsc at 8:58 AM on October 14, 2008
Back on topic -- I suspect there's way more to this story than what we can learn from these links. FDA, bioethics, politics, business, insurance - one huge jumbled mess. I hope the man gets the medicine he needs.
posted by davidmsc at 8:58 AM on October 14, 2008
I'm guessing this is his father?
I'm also guessing the CEO of Biogen is a Republican.
If that is true - and the denial stems from something to do with Democrats/trial lawyers - then I was wrong when I thought Republican slime couldn't get any more disgusting.
I hope all their fears come true, and Obama puts them up against the wall.
posted by bashos_frog at 8:58 AM on October 14, 2008
I'm also guessing the CEO of Biogen is a Republican.
If that is true - and the denial stems from something to do with Democrats/trial lawyers - then I was wrong when I thought Republican slime couldn't get any more disgusting.
I hope all their fears come true, and Obama puts them up against the wall.
posted by bashos_frog at 8:58 AM on October 14, 2008
As much as I think Rocketboom sucks, the guy doesn't deserve this.
Does suck that no doctors are willing to violate the law and just do it. Where's House when you need him?
Link to the drug in question.
Wonder how hard it would be to accidently get the guy a diagnosis for MS.
posted by cjorgensen at 9:05 AM on October 14, 2008 [1 favorite]
Does suck that no doctors are willing to violate the law and just do it. Where's House when you need him?
Link to the drug in question.
Wonder how hard it would be to accidently get the guy a diagnosis for MS.
posted by cjorgensen at 9:05 AM on October 14, 2008 [1 favorite]
It's not clear to me how the company can control distribution so tightly, if there's already a batch of it downstairs at that same hospital. Docs go off-label all the dang time; why is this case so very different?
Heck, I personally have been prescribed things this way: "Um, you also hurt right here, don't you? Yeah, quite a terrible pain, isn't it? This drug will fix that. It may also fix this other thing you've got, but that's totally coincidental and very much not the reason I'm giving you this prescription. No, no, it's because of the pain you have right here that you're getting this prescription. Not any other reason."
Or hell, just lose a bunch. Terrible coincidence, that. Can't imagine how it happened.
...so there's got to be something else going on here.
posted by aramaic at 9:05 AM on October 14, 2008 [3 favorites]
Heck, I personally have been prescribed things this way: "Um, you also hurt right here, don't you? Yeah, quite a terrible pain, isn't it? This drug will fix that. It may also fix this other thing you've got, but that's totally coincidental and very much not the reason I'm giving you this prescription. No, no, it's because of the pain you have right here that you're getting this prescription. Not any other reason."
Or hell, just lose a bunch. Terrible coincidence, that. Can't imagine how it happened.
...so there's got to be something else going on here.
posted by aramaic at 9:05 AM on October 14, 2008 [3 favorites]
Intersting comment posted at TechCrunch:
"My sympathies go out to the Baron family.posted by ericb at 9:07 AM on October 14, 2008 [12 favorites]
This unfortunately is a common problem in the fight for cancer. I lost my brother to cancer and saw many of the same issues. On cause of this problem is that drug companies & researchers attempt to find cures that can be isolated and proved in formal FDA trials if they hope to get a drug approved as a treatment option (which can be worth billions ala Avastatin etc. and cost hundreds of millions to go through the process).
This applies for new drugs as well as for off-label or re-approval of an existing medication for cancer.
When CEO’s make the decision not to share drugs that show some promise with terminal cancer patients they are protecting their future options to submit these drugs as part of formal trials with controlled patient groups. Giving out the drug to terminal patients who request it, have no other options and are happy to experiment with anything that may offer hope can in the future prevent or disrupt their data resulting in problems conducting formal FDA trials. So some would say the CEO’s are doing their fiscal or moral responsibility to run the drug through the proper process that would enable it’s approval as a treatment for millions of patients rather then just the one patient showing the urgent need at that time.
(I would disagree with this, but the case could be made)
At the same time, the approach that doctors took to fighting the AIDS virus which was a series of experimental cocktails created outside of the FDA trial system created a number of treatment protocols that have made huge improvements in the life of afflicted patients. This type of process is not possible in Cancer research since even if you are participating in the FDA trial of an experimental treatment you are forbidden from combining it with other experimental treatments which would pollute their studies.
This approach to approving drugs has led to a slow down in innovation of one the front lines treatment protocols as drug companies are hesitant to led their drugs be combined & experimented on by doctors & patients who have no other hope and are more then willing to combine treatments to attempt a cocktail based treatment protocol.
Many leading cancer researchers have begun to push for a change in this process which would allow them more flexibility in combining different experimental treatments with terminal patients - but the costs & risks to the drug companies under the current FDA process is too great.
This is part of the reason why some of the best treatment protocols are being developed and advanced in Europe & around the world. Anti-angiogensis, Cox-2 inhibitors and a number of other treatment options (in the case of colorectal cancer which I was involved in researching for my brother) were all medications that had better options in Europe then in the United States and Canada.
The incremental, prove of efficacy approach of the FDA may seem like a good idea to create good data for proof of efficacy - but I would argue in the war against cancer, which continues to be a elusive and complicated disease we need to have the option to pull out all the stops and throw everything we have at it to be able to create more data about off label use of drugs, cocktails and experimental therapies in combination with the standard chemo treatments.
This is an FDA problem more so then the CEO of the drug company. They’ve created this environment and there are millions of terminal patients who are every year left in the same hopeless situation of having to beg for access to something that has shown some results in labs - but not yet made it through the arduous FDA approval process for treatment in Cancer."
I'm confused. In the USA, physicians are allowed to prescribe any approved drug for any purpose (off-label use). I don't think they require permission from the manufacturer.
posted by sevenyearlurk at 9:08 AM on October 14, 2008
posted by sevenyearlurk at 9:08 AM on October 14, 2008
I suspect that Biogen's issue is because Tysabri has had some problems getting approval in the past. What in the industry are called "adverse events" (i.e. dead patients). They voluntarily pulled it before the FDA had to get involved, but it's still a mess.
On one hand there are people who are literally dying for want of the drug, and for whom it's almost certainly the best option available; but giving it to them risks screwing up the approval process. The companies are petrified of having the next Vioxx debacle on their hands, and would rather kill people (in effect) by withholding a drug than run the risk of killing them by letting them have it. At least that way, nobody can blame their drug for it.
It's a prime example, in my view, of both market and regulatory failure. We have regulation in the form of the FDA that's supposed to protect patients, but here it's just made the drug companies so risk-averse that they're not delivering products to customers who would probably quite literally beat down their door if they thought it would do any good.
posted by Kadin2048 at 9:08 AM on October 14, 2008 [2 favorites]
On one hand there are people who are literally dying for want of the drug, and for whom it's almost certainly the best option available; but giving it to them risks screwing up the approval process. The companies are petrified of having the next Vioxx debacle on their hands, and would rather kill people (in effect) by withholding a drug than run the risk of killing them by letting them have it. At least that way, nobody can blame their drug for it.
It's a prime example, in my view, of both market and regulatory failure. We have regulation in the form of the FDA that's supposed to protect patients, but here it's just made the drug companies so risk-averse that they're not delivering products to customers who would probably quite literally beat down their door if they thought it would do any good.
posted by Kadin2048 at 9:08 AM on October 14, 2008 [2 favorites]
Yeah, something's really fishy. I mean, how on earth does the Biogen even know about this? Why didn't the doctor's just prescribe it and be done with it before any of these "important" people were contacted? This whole thing gets a "Seriously, wtf?" from the Manhasset.
posted by Manhasset at 9:08 AM on October 14, 2008
posted by Manhasset at 9:08 AM on October 14, 2008
I suspect that Biogen's issue is because Tysabri has had some problems getting approval in the past. What in the industry are called "adverse events" (i.e. dead patients). They voluntarily pulled it before the FDA had to get involved, but it's still a mess.
Yep.
Yep.
“The safety and legality of the drug is highly contested. Tysabri is also know as Natalizumab, and its FDA file details a controversial history dating back four years.posted by ericb at 9:13 AM on October 14, 2008
According to the FDA, Tysabri was approved in 2004, and voluntarily withdrawn by manufacturer Biogen in 2005 when patients developed deadly brain infections during trials. Tysabri was approved again in January 2008, but only for Crohn's disease, and only as part of a strict program limiting risk of developing progressive multifocal leukoencephalopathy (PML), the deadly brain infection.
On August 26, 2008, two more cases of PML-- the deadly brain infection-- were uncovered in European patients using Tysabri to treat multiple sclerosis. The patients are European because use of the drug to treat multiple sclerosis is not allowed in the US.”
sevenyearlurk: I think the problem is probably acquiring the drug in the first place. You can't exactly go down to CVS and fill a prescription for Tysabri. Presumably you have to get it directly from Biogen, or you'd have to get someone to synthesize it for you.
I don't think this is a case of an existing, commonly-available drug or substance that's being used in a novel way -- if that were the case it would be pretty trivial to acquire it. It's a true 'designer drug,' to the best of my knowledge.
posted by Kadin2048 at 9:13 AM on October 14, 2008
I don't think this is a case of an existing, commonly-available drug or substance that's being used in a novel way -- if that were the case it would be pretty trivial to acquire it. It's a true 'designer drug,' to the best of my knowledge.
posted by Kadin2048 at 9:13 AM on October 14, 2008
Here's to the awesome future where rich people live forever!
posted by Artw at 9:13 AM on October 14, 2008 [3 favorites]
posted by Artw at 9:13 AM on October 14, 2008 [3 favorites]
I'm also guessing the CEO of Biogen is a Republican.
A quick search of Open Secrets indicates that James Muller of Biogen Idec donated $1000 to The Committee for a Democratic Majority.
Let's not drag politics into this, eh?
posted by barkingpumpkin at 9:14 AM on October 14, 2008 [18 favorites]
A quick search of Open Secrets indicates that James Muller of Biogen Idec donated $1000 to The Committee for a Democratic Majority.
Let's not drag politics into this, eh?
posted by barkingpumpkin at 9:14 AM on October 14, 2008 [18 favorites]
If distribution is controlled, and Mayo goes off-label, they could risk losing access to the drug (just a theory). But I doubt if a lot of these people would have gotten involved in something dodgy.
If the solution was as easy as going off-label I'm pretty sure the doctors at Mayo have heard about doing this.
I wouldn't say it smells fishy at all, just complicated.
posted by cjorgensen at 9:15 AM on October 14, 2008
If the solution was as easy as going off-label I'm pretty sure the doctors at Mayo have heard about doing this.
I wouldn't say it smells fishy at all, just complicated.
posted by cjorgensen at 9:15 AM on October 14, 2008
I don't think this is a case of an existing, commonly-available drug or substance that's being used in a novel way
It says in the link that the drug is available, cheap, and sitting on the shelf in the local pharmacy.
posted by Manhasset at 9:19 AM on October 14, 2008
It says in the link that the drug is available, cheap, and sitting on the shelf in the local pharmacy.
posted by Manhasset at 9:19 AM on October 14, 2008
Embarrassing. My first post to Metafilter in forever, and I mix Lance Burton with Lance Armstrong. Oy.
That said, I hope there is someway we can get this issue brought front and center in national media.
posted by IndigoSkye at 9:20 AM on October 14, 2008
That said, I hope there is someway we can get this issue brought front and center in national media.
posted by IndigoSkye at 9:20 AM on October 14, 2008
That said, I hope there is someway we can get this issue brought front and center in national media.
What is the issue?
posted by Combustible Edison Lighthouse at 9:24 AM on October 14, 2008
What is the issue?
posted by Combustible Edison Lighthouse at 9:24 AM on October 14, 2008
This is all pretty bizarre, but the bigger question is, what can we do to help?
posted by dejah420 at 9:25 AM on October 14, 2008
posted by dejah420 at 9:25 AM on October 14, 2008
It says in the link that the drug is available, cheap, and sitting on the shelf in the local pharmacy.
Tysabri is not cheap. A single dose is about $2180, which makes a year's supply (13 injections) about $28k. The year's supply is for MS, not cancer, and I don't know how many injections would be used to treat multiple myeloma. Nonetheless, even if he only needed a single shot, it would still be pretty expensive.
It is also not available just anywhere. "TYSABRI is available only under a special restricted distribution program called the TOUCH Prescribing Program. Under the TOUCH Prescribing Program, only prescribers, infusion centers, and pharmacies associated with infusion centers registered with the program are able to prescribe, distribute, or infuse the product. In addition, TYSABRI must be administered only to patients who are enrolled in and meet all the conditions of the TOUCH Prescribing Program."
So, I'm sure he is willing (and likely able) to pay for the medicine, even out of pocket, and I'm sure he is willing and able to get to a TOUCH Prescribing Program-registered hospital, but I don't think it's fair to call it 'available, cheap, and sitting on the shelf in the local pharmacy.'
posted by jedicus at 9:29 AM on October 14, 2008 [4 favorites]
Tysabri is not cheap. A single dose is about $2180, which makes a year's supply (13 injections) about $28k. The year's supply is for MS, not cancer, and I don't know how many injections would be used to treat multiple myeloma. Nonetheless, even if he only needed a single shot, it would still be pretty expensive.
It is also not available just anywhere. "TYSABRI is available only under a special restricted distribution program called the TOUCH Prescribing Program. Under the TOUCH Prescribing Program, only prescribers, infusion centers, and pharmacies associated with infusion centers registered with the program are able to prescribe, distribute, or infuse the product. In addition, TYSABRI must be administered only to patients who are enrolled in and meet all the conditions of the TOUCH Prescribing Program."
So, I'm sure he is willing (and likely able) to pay for the medicine, even out of pocket, and I'm sure he is willing and able to get to a TOUCH Prescribing Program-registered hospital, but I don't think it's fair to call it 'available, cheap, and sitting on the shelf in the local pharmacy.'
posted by jedicus at 9:29 AM on October 14, 2008 [4 favorites]
Ok.. I'm not understanding one thing... I mean, in a world/society/city where one can get Oxycontins, Percosets, Zanaxes, to name a tiny few of illegally bought prescriptions of that sort, and antibiotics, allergy meds, and more "harmless" prescriptions, albeit also obtained "illegally." If this med is still being produced for a tightly controlled few, why can't someone just offer a bucket load of cash, or a Romp in the hay with Lance Armstrong (or something equally unique) for a supply of these meds?
Of course, I'm probably completely missing something here, but for the life of me, I can't figure this one out.
posted by Debaser626 at 9:33 AM on October 14, 2008
Of course, I'm probably completely missing something here, but for the life of me, I can't figure this one out.
posted by Debaser626 at 9:33 AM on October 14, 2008
Debaser626, Tysabri is an antibody -- I'm pretty sure it has to be delivered intravenously, under controlled conditions. It's not like a bottle of tablets that someone can deliver in a brown paper bag.
posted by sevenyearlurk at 9:39 AM on October 14, 2008
posted by sevenyearlurk at 9:39 AM on October 14, 2008
Let's not drag politics into this, eh?
Ah, Sorry, it was a badly-timed joke. But I'm pretty sure it is the Fred Baron who was famous for his involvement in the John Edwards "thing." Again, not unnecessarily bringing in politics, but thats clearly how get managed to get Clinton and Kerry and others involved. He was Kerry's campaign finance manager!
I wish him the best. And, yes, this is an odd story.
posted by vacapinta at 9:39 AM on October 14, 2008
Ah, Sorry, it was a badly-timed joke. But I'm pretty sure it is the Fred Baron who was famous for his involvement in the John Edwards "thing." Again, not unnecessarily bringing in politics, but thats clearly how get managed to get Clinton and Kerry and others involved. He was Kerry's campaign finance manager!
I wish him the best. And, yes, this is an odd story.
posted by vacapinta at 9:39 AM on October 14, 2008
I'm with others in thinking that there's more to this story. Why can't/won't his doctors prescribe the drug off-label? Why in this instance is the manufacturer involved? Is it really due to him not qualifying for TOUCH Prescribing Program?
"Once the drug gets on market, the FDA cannot tell doctors what to do with that medication. Doctors may choose to use a drug that is not listed by the FDA for a certain condition. This is off-label use."posted by ericb at 9:40 AM on October 14, 2008
It says in the link that the drug is available, cheap, and sitting on the shelf in the local pharmacy.
I think what they mean by that is that it's sitting in the Mayo Clinic's pharmacy. Presumably (and I don't know this for sure, but it's the only explanation I can think of), the Mayo Clinic gets its supply from Biogen, as part of a controlled trial, or with some sort of agreement that it only gets used for Crohn's. Maybe they're afraid to give it out because if they do, they won't get any more from Biogen. (I don't know enough about this sort of thing to know if it's typical, but it wouldn't totally surprise me if drugs got sent to hospitals with "license agreements" just like software...)
It's hard to believe that the doctors involved haven't considered just prescribing off-label, since they're apparently the ones who have settled on this being the ideal treatment.
What I wonder, though, is if the doctors had just written the off-label Rx, and they had just filled it at their pharmacy at the Clinic, and there hadn't been a fuss made, whether Biogen would have ever noticed or done anything punitive about it. I suppose they didn't want to take the chance and risk their supply being cut off, but it seems a bit like a "better to beg forgiveness than ask permission" situation.
The problem seems that, since they went and asked permission from Biogen, that Biogen felt that they can't condone or approve (even tacitly) the use, since it's unapproved and they don't want to risk their FDA approval. What's odd is that Biogen hasn't just come out and said that, flatly; it seems like it would be in their best interest as well to put public-policy pressure on the FDA for approval reform. Tysabri is sort of the poster child (even before this) of a drug being withheld from desperate people with nothing to lose for the benefit of the regulators.
I wonder if they have considered holding up the hospital pharmacy? Dead anyway; but the ensuing trial would at least get some media attention for the problem. I'd never advise anyone to do that, but when I imagine being in that situation myself, it's something I'd imagine I'd spend a lot of time mulling over, if I was slowly dying and knew that a cure was sitting six floors down behind a counter.
posted by Kadin2048 at 9:41 AM on October 14, 2008
I think what they mean by that is that it's sitting in the Mayo Clinic's pharmacy. Presumably (and I don't know this for sure, but it's the only explanation I can think of), the Mayo Clinic gets its supply from Biogen, as part of a controlled trial, or with some sort of agreement that it only gets used for Crohn's. Maybe they're afraid to give it out because if they do, they won't get any more from Biogen. (I don't know enough about this sort of thing to know if it's typical, but it wouldn't totally surprise me if drugs got sent to hospitals with "license agreements" just like software...)
It's hard to believe that the doctors involved haven't considered just prescribing off-label, since they're apparently the ones who have settled on this being the ideal treatment.
What I wonder, though, is if the doctors had just written the off-label Rx, and they had just filled it at their pharmacy at the Clinic, and there hadn't been a fuss made, whether Biogen would have ever noticed or done anything punitive about it. I suppose they didn't want to take the chance and risk their supply being cut off, but it seems a bit like a "better to beg forgiveness than ask permission" situation.
The problem seems that, since they went and asked permission from Biogen, that Biogen felt that they can't condone or approve (even tacitly) the use, since it's unapproved and they don't want to risk their FDA approval. What's odd is that Biogen hasn't just come out and said that, flatly; it seems like it would be in their best interest as well to put public-policy pressure on the FDA for approval reform. Tysabri is sort of the poster child (even before this) of a drug being withheld from desperate people with nothing to lose for the benefit of the regulators.
I wonder if they have considered holding up the hospital pharmacy? Dead anyway; but the ensuing trial would at least get some media attention for the problem. I'd never advise anyone to do that, but when I imagine being in that situation myself, it's something I'd imagine I'd spend a lot of time mulling over, if I was slowly dying and knew that a cure was sitting six floors down behind a counter.
posted by Kadin2048 at 9:41 AM on October 14, 2008
I wonder if they have considered holding up the hospital pharmacy?
Brings to mind the film John Q in which Denzel Washington plays a father who takes a hospital full of patients hostage until the hospital puts his son's name on an organ donor's list, so that he can receive a transplanted heart.
posted by ericb at 9:45 AM on October 14, 2008
Brings to mind the film John Q in which Denzel Washington plays a father who takes a hospital full of patients hostage until the hospital puts his son's name on an organ donor's list, so that he can receive a transplanted heart.
posted by ericb at 9:45 AM on October 14, 2008
with all the special consideration being given to those with wealth and connections lately I have to ask: Would you all take the same amount of time if the sick individual was homeless?
Nope. You wouldn't even know of it, and I'm gonna treat this poor (but well-connected) soul with the same level of compassion. Sad, but true.
posted by HyperBlue at 9:48 AM on October 14, 2008
Nope. You wouldn't even know of it, and I'm gonna treat this poor (but well-connected) soul with the same level of compassion. Sad, but true.
posted by HyperBlue at 9:48 AM on October 14, 2008
Put him in a car and get him up here to Canada.
According to the MS Society of Canada Tysabri was approved two years ago by Health Canada for the treatment of MS.
Surely that's an option?
posted by chococat at 9:49 AM on October 14, 2008
According to the MS Society of Canada Tysabri was approved two years ago by Health Canada for the treatment of MS.
Surely that's an option?
posted by chococat at 9:49 AM on October 14, 2008
48 hours to live? Multiple Myeloma is nasty, but 48 hours from diagnosis to death? This guy must have been horrendously ill for quite some time to be within 2 days of death from myeloma.
posted by Mister_A at 10:02 AM on October 14, 2008
posted by Mister_A at 10:02 AM on October 14, 2008
I'm also guessing the CEO of Biogen is a Republican.
I was going to guess replicant.
Because he's not helping. Why is that?
posted by namespan at 10:12 AM on October 14, 2008 [2 favorites]
I was going to guess replicant.
Because he's not helping. Why is that?
posted by namespan at 10:12 AM on October 14, 2008 [2 favorites]
I get the feeling that Mr. Baron has overinflated expectations about the likely efficacy of natalizumab in treating his father's condition. Looking on pubmed, I can't even find a single reference to even a case report of Tysabri used in this way, though there is an ongoing study run by Biogen whose results have not yet been reported. I have some conceptual objections to the use of Tysabri in this case anyway- natalizumab is an IgG4 antibody, which has a low capability to activate the complement system or to stimulate phagocytes, making it unlikely to kill myeloma cells, though it may slow their migration through the body. Alemtuzumab, while binding either to a subset of the myeloma cells or more poorly, is an IgG1 antibody, which has substantial capability to activate the complement system and kill cells. Given the apparently substantially progressed state of the elder Mr. Baron's disease, Tysabri, even if effective at slowing migration, is likely too little too late.
There is the additional question of possible harms. Tysabri has a troubled history, with some evidence that it may induce progressive multifocal leukoencephalopathy in a subset of patients. The whole TOUCH system as discussed above was initiated as a consequence of this. You could argue that PML is not too much of an issue for someone with 48 hours to live, but there is still a situation of relatively proven harms being weighed against relatively unproven benefits.
OK, you say, but Biogen has been running a study examining Tysabri in just this condition. Was the elder Mr. Baron considered for this study? Well, if he was considered, he would be rejected, according to the inclusion criteria, criteria which have the twofold function of protecting the scientific integrity of the study and protecting the patients included within it. Beyond legal liability, Biogen has a moral responsibility to act in a way that protects the patients that use its products. This calculus is unfortunately more fuzzy than the law, and it's possible to conclude both that Biogen should and shouldn't supply the drug based on one's measure of benefit and harm. Ultimately, this is a way more complicated story than just "craven Republican drug company refuses to supply drug to needy patient for liability reasons", and we should be directing tough questions to both sides before assigning condemnation.
posted by monocyte at 10:20 AM on October 14, 2008 [16 favorites]
There is the additional question of possible harms. Tysabri has a troubled history, with some evidence that it may induce progressive multifocal leukoencephalopathy in a subset of patients. The whole TOUCH system as discussed above was initiated as a consequence of this. You could argue that PML is not too much of an issue for someone with 48 hours to live, but there is still a situation of relatively proven harms being weighed against relatively unproven benefits.
OK, you say, but Biogen has been running a study examining Tysabri in just this condition. Was the elder Mr. Baron considered for this study? Well, if he was considered, he would be rejected, according to the inclusion criteria, criteria which have the twofold function of protecting the scientific integrity of the study and protecting the patients included within it. Beyond legal liability, Biogen has a moral responsibility to act in a way that protects the patients that use its products. This calculus is unfortunately more fuzzy than the law, and it's possible to conclude both that Biogen should and shouldn't supply the drug based on one's measure of benefit and harm. Ultimately, this is a way more complicated story than just "craven Republican drug company refuses to supply drug to needy patient for liability reasons", and we should be directing tough questions to both sides before assigning condemnation.
posted by monocyte at 10:20 AM on October 14, 2008 [16 favorites]
If Mr. Baron's already on Campath-- which also has a chance of inducing PML in patients-- they might as well just go ahead with the Tysabri. Who's to say which one caused an adverse outcome, if any adverse outcome occurs.
posted by fairytale of los angeles at 10:27 AM on October 14, 2008
posted by fairytale of los angeles at 10:27 AM on October 14, 2008
THE US SENATE HAS BEFORE IT BILL S. 1956 (Access, Compassion, Care and Ethics for Seriously Ill Patients Act)
The bill would permit (Tier 1) marketing approval on the basis of data from a Phase I trial and other supporting evidence.
Argument Pro: It would allow patients with otherwise untreatable conditions to have access to experimental treatment (slim/last hope) where otherwise they would only have palliative treatment
Argument Contra: This would tend to undermine the controlled trials to determine safety and effectiveness.
posted by ageispolis at 10:42 AM on October 14, 2008
The bill would permit (Tier 1) marketing approval on the basis of data from a Phase I trial and other supporting evidence.
Argument Pro: It would allow patients with otherwise untreatable conditions to have access to experimental treatment (slim/last hope) where otherwise they would only have palliative treatment
Argument Contra: This would tend to undermine the controlled trials to determine safety and effectiveness.
posted by ageispolis at 10:42 AM on October 14, 2008
The drug has a good chance of saving the life of a terminally ill patient, it is on the shelf in the building with the patient. The owner will not allow the patient access to the drug of which there is for purposes of this question there is an excess supply. (The story said there is no shortage of this drug, so I am assuming they are not lying)
If the above is true I totally remember this question from college when ever discussions of morality came up. I'm pretty sure the answer is almost always you give him the drug and say fuck you to the "owner".
posted by MrBobaFett at 10:48 AM on October 14, 2008
If the above is true I totally remember this question from college when ever discussions of morality came up. I'm pretty sure the answer is almost always you give him the drug and say fuck you to the "owner".
posted by MrBobaFett at 10:48 AM on October 14, 2008
The problem with saying fuck you to the owner is this:
If Biogen let's you experiment willy nilly with their drug, the FDA will examine the results of the experiment in excruciating detail. They have to- it's the law. The upshot is that the uncontrolled experiment will cause the drug's release to the general public to be hugely delayed.
Biogen's hands are tied- they want to sell the drug, they want to offer compassionate use, they don't want people to die, and they certainly don't want this PR disaster. They also don't want to be forced into holding the drug off the market for even a week longer than necessary- that will in all likelihood kill more people.
Channel your anger at your senators and congressional representatives who have allowed this perverse situation to go on for years- while the quack medicine "food supplement" industry is nearly entirely unregulated and killing people.
posted by jenkinsEar at 10:57 AM on October 14, 2008 [1 favorite]
If Biogen let's you experiment willy nilly with their drug, the FDA will examine the results of the experiment in excruciating detail. They have to- it's the law. The upshot is that the uncontrolled experiment will cause the drug's release to the general public to be hugely delayed.
Biogen's hands are tied- they want to sell the drug, they want to offer compassionate use, they don't want people to die, and they certainly don't want this PR disaster. They also don't want to be forced into holding the drug off the market for even a week longer than necessary- that will in all likelihood kill more people.
Channel your anger at your senators and congressional representatives who have allowed this perverse situation to go on for years- while the quack medicine "food supplement" industry is nearly entirely unregulated and killing people.
posted by jenkinsEar at 10:57 AM on October 14, 2008 [1 favorite]
An interesting comment at Andrew Baron's website:
"Andrew - having been in the pharma industry for many years I have seen countless doctors prescribe drugs off-label for patients in need. It is common practice in oncology, psychiatry and on and on and on. Biogen had many problems with Tysabri as you probably know by now and the FDA pulled it off the market for a time for a particular indication - MS I want to say. There are NO circumstances where a representative of a drug manufacturer can 'approve' off label usage of one of their drugs but they routinely employ Medical Science Liaisons to discuss off label usage with physicians. While not 'recommending' off label usage they are able to legally speak with doctors about unusual applications and even recommend that a physican speak to another doctor that has used the drug off label. There is NO reason in the world why your dad's doctor has not (ESPECIALLY with the FDA's blessing) been speaking to other docs about using Tysabri in this case. Your dad's doc should have already administered the drug which is absolutely NO problem to acquire at ANY drug store. Please push your dad's doctor!! He/She is the one that is blocking progress for your dad at this juncture! I sincerely hope that this doctor is not fearful of liability, getting sued, getting in 'trouble' and that is why he has not proceeded.....that would be reprehensible. Good luck Andrew, my heart goes out to you and your family."posted by ericb at 11:10 AM on October 14, 2008 [1 favorite]
Everybody, this has zero to do with off-label prescribing or the FDA or Congress or greedy drug companies. It has to do with several different groups of highly skilled medical professionals, and whether they think this crazy drug will actually help in this situation. The patient's son has latched onto this cure/not-cure, but he's really in the absolute worst position to judge here.
Now, go re-read what monocyte said.
posted by Nahum Tate at 11:25 AM on October 14, 2008 [2 favorites]
Now, go re-read what monocyte said.
posted by Nahum Tate at 11:25 AM on October 14, 2008 [2 favorites]
...the drug which is absolutely NO problem to acquire at ANY drug store.
Vis-a-vis the questions related to availability of Tysabri, I just went to the CVS website where it indicates one can order the medication (TYSABRI 20 MG/ML SOL -- Natalizumab) for online or store pickup with a valid doctor's prescription. Granted it's a 20 mg vial injection and not a larger quantity (300 mg) used by intravenous applications.
It looks like the TOUCH Prescribing Program applies for higher dosages and only to its use treating MS:
posted by ericb at 11:29 AM on October 14, 2008
Vis-a-vis the questions related to availability of Tysabri, I just went to the CVS website where it indicates one can order the medication (TYSABRI 20 MG/ML SOL -- Natalizumab) for online or store pickup with a valid doctor's prescription. Granted it's a 20 mg vial injection and not a larger quantity (300 mg) used by intravenous applications.
It looks like the TOUCH Prescribing Program applies for higher dosages and only to its use treating MS:
"The recommended dose of natalizumab for the treatment of relapsing-remitting multiple sclerosis is 300 mg administered intravenously every four weeks. Tysabri is administered intravenously by trained professionals at infusion centers."So, on one hand the drug is available at lower dosages (20 mg) quite readily, but larger dosages (300 mg) require participation in the TOUCH Prescribing Program with intravenous application at an approved facility.
posted by ericb at 11:29 AM on October 14, 2008
It seems like something is missing here... This just doesn't make any sense. Is he looking for a donation of the drug?
posted by mr_roboto at 11:33 AM on October 14, 2008
posted by mr_roboto at 11:33 AM on October 14, 2008
The story said there is no shortage of this drug, so I am assuming they are not lying
Let's be careful here. The primary hyperlink in the FPP is not to a "story," nor to an article, but a letter written to Biogen by a rightfully distraught man. More information and objective balance might help us (especially medical novices like myself) better understand the situation and issues at hand. At this stage we are trying to "suss-out" the story with very little information from the other parties involved (i.e. the physicians, Mayo Clinic, Biogen, FDA, etc.).
posted by ericb at 11:33 AM on October 14, 2008
Let's be careful here. The primary hyperlink in the FPP is not to a "story," nor to an article, but a letter written to Biogen by a rightfully distraught man. More information and objective balance might help us (especially medical novices like myself) better understand the situation and issues at hand. At this stage we are trying to "suss-out" the story with very little information from the other parties involved (i.e. the physicians, Mayo Clinic, Biogen, FDA, etc.).
posted by ericb at 11:33 AM on October 14, 2008
This would tend to undermine the controlled trials to determine safety and effectiveness.
How does giving it to someone who's not part of the controlled trial "undermine" the trial? To me, it sounds like saying gay marriage undermines heterosexual marriage. What's the link?
posted by languagehat at 11:36 AM on October 14, 2008 [2 favorites]
How does giving it to someone who's not part of the controlled trial "undermine" the trial? To me, it sounds like saying gay marriage undermines heterosexual marriage. What's the link?
posted by languagehat at 11:36 AM on October 14, 2008 [2 favorites]
This would tend to undermine the controlled trials to determine safety and effectiveness.
From a comment at TechCrunch:
From a comment at TechCrunch:
"If the patient has been immunocompromised, giving him Tysabri would put him at extreme risk of PML, a brain virus that has a lethal history. While Biogen and Elan have developed a way to remove Tysabri from the system quickly, in an immune compromised patient, a patient that doesn’t have the natural ability to produce the antibodies necessary to check the virus, the PML would continue to develop and eventually kill the patient. Biogen and Elan are both struggling against a pr nightmare with Tysabri and PML.posted by ericb at 11:43 AM on October 14, 2008 [1 favorite]
On top of that, they have initiated trials specifically for multiple myeloma. Their hope is to carefully select patients who can benefit from Tysabri that also have a relatively intact immune system. Outside of the trial, if the patient dies, it will have negatively impact the trial results even though the person wasn’t in the trial under controlled conditions.
In the big picture, they may not want to risk drug development complications by providing it to a patient for whom it may have an unacceptable risk profile. If the drug works, think of the thousands of multiple myeloma patients who would suffer if development of the drug were slowed or stopped. Beyond those patients, another incidence of PML would affect the perception of Tysabri risk overall. The loss of Tysabri to patients could go well beyond the multiple myeloma indication which would be a catastrophe for Biogen, Elan and the many patients who suffer from diseases for which Tysabri helps."
FYI -- the Phase I/II Tysabri clinical trial for Multiple Myeloma launched last month (September 4, 2008).
"Elan and Biogen Idec have started a Phase I/II clinical trial of their multiple sclerosis drug Tysabri to treat multiple myeloma, a plasma cancer.posted by ericb at 11:47 AM on October 14, 2008 [1 favorite]
The first multiple-myeloma patient took Tysabri (natalizumab) Sept. 4. The open-label, two-arm study will evaluate the safety and potential anti-tumor activity of the drug in adult patients with relapsed or refractory multiple myeloma.
In the Phase I portion of the trial, a standard dose-escalation design will be used to assess the safety and tolerability of Tysabri in as many as 12 patients. In the Phase II portion, as many as 30 patients will be randomized to the tolerated doses identified in Phase I.
Treatment cycles will be intravenous infusions of Tysabri once every 28 days for six months. After that, patients who have had a partial or complete response may continue to receive Tysabri once every 28 days until the disease begins to progress, according to a statement from the companies."
TechCrunch comment:
"Something about this whole story is out of whack. The FDA approved clinical trials for treating multiple myeloma with Tysarbi over a month ago...posted by ericb at 11:49 AM on October 14, 2008 [1 favorite]
I am assuming that what Biogen determined was that his father was not suitable to be included in the clinical trials (either because he was so far progressed in his cancer or for other reasons). Given that clinical trials have to be conducted under very defined conditions, nothing unreasonable about that.
However, I don’t see why that would prevent off-label usage. It is, as others have pointed out, a widely used proceedure that doesn’t require any permission from the drug company (or for them to even know)."
Heartbreaking, but as noted, if he has 48 hours to live, there is probably very little that can be done anyway. If I am ever struck by rapidly progressive cancer, I would not want to put my family through the agony of last-minute treatments that would probably only extend my life a small amount.
That said, I'm old enough to remember patients and families begging for laetrile treatments. While this drug isn't quackery -- laetrile was an apricot-pit extract with no scientifically proven efficacy -- but the same hunger for a "miracle cure" continues.
posted by dhartung at 1:30 PM on October 14, 2008
That said, I'm old enough to remember patients and families begging for laetrile treatments. While this drug isn't quackery -- laetrile was an apricot-pit extract with no scientifically proven efficacy -- but the same hunger for a "miracle cure" continues.
posted by dhartung at 1:30 PM on October 14, 2008
The idea of further data somehow "undermining" scientific research is so ludicrously stupid that you could not make it up. At worst, it generates a report as follows: "we gave X amount of the drug to the patient, intravenously. He suffered an adverse reaction to it, described as follows, of which he died. Autopsy and comparison of his posthumously and prehumously drawn blood and brain fluid reveals ..." and so on.
It may be that the regulatory environment has been so absurdly mishandled under malcompetent administration that the situation has been created where someone has the impression that such a state of affairs, where further data is undesirable, could occur. That needs to be addressed, ASAP, regardless of the outcome of this man's case.
posted by aeschenkarnos at 4:24 PM on October 14, 2008
It may be that the regulatory environment has been so absurdly mishandled under malcompetent administration that the situation has been created where someone has the impression that such a state of affairs, where further data is undesirable, could occur. That needs to be addressed, ASAP, regardless of the outcome of this man's case.
posted by aeschenkarnos at 4:24 PM on October 14, 2008
Gee, I'm all cut up. Except when my mother was diagnosed with a fatal disease, she didn''t know anyone powerful and connected.
So she died.
posted by nax at 5:32 PM on October 14, 2008 [1 favorite]
So she died.
posted by nax at 5:32 PM on October 14, 2008 [1 favorite]
Not just to be contrarian here, but in the early days of the AIDS/HIV epidemic, there were many physicians who were absolutely positive that certain therapies would cure AIDS or prevent major opportunistic infections, if only the big bad drug companies would let them have the drugs. I was a senior statistician for the Community Program for Clinical Research on AIDS and we designed and ran randomized trials for some of those therapies. In my experience, it turned out that nearly half of the proposed therapies were killing people prematurely; placebo did better than the active drugs.
I work with a hematologist/oncologist who is a well known multiple myeloma guy. He has worked with the heme/oncs at Mayo. We have done research on multiple myeloma and he has educated me to just how difficult a disease it is. And I feel really bad for Andrew Baron's father, because he will probably die soon.
It is often true that physicians feel very strongly that they know something works without the randomized trial evidence it takes to really know that. And it is often true that they are wrong. This guy has a drug he can access that is working to keep him alive. That is very good for him. Switching to another drug may help him live even longer...or it may kill him. It is unfair for his physicians to tell him they know which.
posted by Mental Wimp at 5:36 PM on October 14, 2008 [1 favorite]
I work with a hematologist/oncologist who is a well known multiple myeloma guy. He has worked with the heme/oncs at Mayo. We have done research on multiple myeloma and he has educated me to just how difficult a disease it is. And I feel really bad for Andrew Baron's father, because he will probably die soon.
It is often true that physicians feel very strongly that they know something works without the randomized trial evidence it takes to really know that. And it is often true that they are wrong. This guy has a drug he can access that is working to keep him alive. That is very good for him. Switching to another drug may help him live even longer...or it may kill him. It is unfair for his physicians to tell him they know which.
posted by Mental Wimp at 5:36 PM on October 14, 2008 [1 favorite]
Presumably you have to get it directly from Biogen, or you'd have to get someone to synthesize it for you.
I'm afraid it's Biogen or no one.
Tysabri is a monoclonal antibody. To put it in perspective aspirin weighs about 200 Daltons, Penicillin weighs about 400 Daltons and Tysabri weighs about 150,000 Daltons.
To make it you'd have to know the amino acid sequence of the antibody, make your DNA, put that it a cell line, pick a high performing clone, run a culture, purify the protein, do any analytical work you were going to do.... If you weren't cutting any corners in terms of normal drug production, knew everything that the folks at Biogen knew and didn't have to file anything with the FDA, you could probably pull this off in under a year if you were dedicated.
If you did this five times and had one patient pull through while the remainder died, you would have a pretty good batting average as drug discovery goes.
posted by Kid Charlemagne at 10:13 PM on October 14, 2008
I'm afraid it's Biogen or no one.
Tysabri is a monoclonal antibody. To put it in perspective aspirin weighs about 200 Daltons, Penicillin weighs about 400 Daltons and Tysabri weighs about 150,000 Daltons.
To make it you'd have to know the amino acid sequence of the antibody, make your DNA, put that it a cell line, pick a high performing clone, run a culture, purify the protein, do any analytical work you were going to do.... If you weren't cutting any corners in terms of normal drug production, knew everything that the folks at Biogen knew and didn't have to file anything with the FDA, you could probably pull this off in under a year if you were dedicated.
If you did this five times and had one patient pull through while the remainder died, you would have a pretty good batting average as drug discovery goes.
posted by Kid Charlemagne at 10:13 PM on October 14, 2008
In my experience, it turned out that nearly half of the proposed therapies were killing people prematurely; placebo did better than the active drugs.
That is useful information, but you couldn't have found it out without the proposed therapies having been tried.
No matter how critical or trivial your condition, how hard or easy it is to cure, anything a doctor gives or does to you is always under the presumption of "we think this has a high chance of working". It might not. If it doesn't, that's not your, the doctor's, or the drug company's fault, provided all of you have at all times been honest with each other. (Caveat for placebos, but a placebo is as harmless a substance as possible, and anyone given a placebo is given it with the expectation that the patient themselves, having been given it, may cure their condition themselves: quite often true, especially in the case of pain.)
Also, informing the decisions made by patient, doctor, and drug company, is the risk of the treatment: whether or not it works, it might have other adverse effects. This is a fairly easy ethical problem, in my opinion - the doctor and drug company ought to state the risks as known, and if the patient is capable of making decisions, or decisions made on his/her behalf, the patient can decide whether to try the drug. If the risks are extremely remote (1:100,000 chance of allergic reaction, say) or comparatively trivial (1:100 chance of not shrinking the tumor, but 1:3 chance of causing itching sores), a general caution like "there is an extremely small chance of an adverse reaction" or "it's likely you'll get itching sores, here's a prescription for cream if you need it" would be enough. But a person who is dying is a special case. If you die, you lose everything. There is, on the face of it, no greater risk. There may be actual greater risks than dying, ie living in agonizing pain, horrible disfigurement, permanent locked-in syndrome, bankrupting or putting at risk of jail the rest of your family, etc, but once again, anything known about these should be stated and it should be the patient's decision. I don't know where I'd draw the line, personally: a drug with a 10% chance of curing you, 90% chance of killing you, may be worth taking if your condition has a 90% chance of killing you, 10% chance of leaving you permanently incapacitated. It's a personal decision. Taking it out of the patient's hands, for reasons unrelated to the success or failure of the treatment itself, is bad policy.
I think it's fair to have a caveat for unreasonable cost, but that needs to be determined consistently according to some fair formula--not whether the drug company CEO knows your golf buddy--and in any case, should be weighed against the immense research benefit of having a person with a known condition who is willing to subject themselves to the treatment, whose reactions to it can be fully recorded, offering an opportunity that can never be provided by millions of rats and PCR reactions and computer simulations.
posted by aeschenkarnos at 10:35 PM on October 14, 2008
That is useful information, but you couldn't have found it out without the proposed therapies having been tried.
No matter how critical or trivial your condition, how hard or easy it is to cure, anything a doctor gives or does to you is always under the presumption of "we think this has a high chance of working". It might not. If it doesn't, that's not your, the doctor's, or the drug company's fault, provided all of you have at all times been honest with each other. (Caveat for placebos, but a placebo is as harmless a substance as possible, and anyone given a placebo is given it with the expectation that the patient themselves, having been given it, may cure their condition themselves: quite often true, especially in the case of pain.)
Also, informing the decisions made by patient, doctor, and drug company, is the risk of the treatment: whether or not it works, it might have other adverse effects. This is a fairly easy ethical problem, in my opinion - the doctor and drug company ought to state the risks as known, and if the patient is capable of making decisions, or decisions made on his/her behalf, the patient can decide whether to try the drug. If the risks are extremely remote (1:100,000 chance of allergic reaction, say) or comparatively trivial (1:100 chance of not shrinking the tumor, but 1:3 chance of causing itching sores), a general caution like "there is an extremely small chance of an adverse reaction" or "it's likely you'll get itching sores, here's a prescription for cream if you need it" would be enough. But a person who is dying is a special case. If you die, you lose everything. There is, on the face of it, no greater risk. There may be actual greater risks than dying, ie living in agonizing pain, horrible disfigurement, permanent locked-in syndrome, bankrupting or putting at risk of jail the rest of your family, etc, but once again, anything known about these should be stated and it should be the patient's decision. I don't know where I'd draw the line, personally: a drug with a 10% chance of curing you, 90% chance of killing you, may be worth taking if your condition has a 90% chance of killing you, 10% chance of leaving you permanently incapacitated. It's a personal decision. Taking it out of the patient's hands, for reasons unrelated to the success or failure of the treatment itself, is bad policy.
I think it's fair to have a caveat for unreasonable cost, but that needs to be determined consistently according to some fair formula--not whether the drug company CEO knows your golf buddy--and in any case, should be weighed against the immense research benefit of having a person with a known condition who is willing to subject themselves to the treatment, whose reactions to it can be fully recorded, offering an opportunity that can never be provided by millions of rats and PCR reactions and computer simulations.
posted by aeschenkarnos at 10:35 PM on October 14, 2008
That is useful information, but you couldn't have found it out without the proposed therapies having been tried.
We couldn't have found it out without testing them in randomized trials.
posted by Mental Wimp at 7:59 AM on October 15, 2008
We couldn't have found it out without testing them in randomized trials.
posted by Mental Wimp at 7:59 AM on October 15, 2008
Biogen Turns Down Dying Patient’s Request For Tysabri—and Explains Why
“Frederick Baron’s physician hit upon the idea of treating him with Tysabri because samples of his cancer cells turned out to carry a molecular marker that Tysabri, an antibody drug, is designed to seek out and block, according to Andrew Baron’s letter. Biogen has done some animal tests that suggest Tysabri might be an effective treatment for multiple myeloma, and in fact, it began treating the first patient in a clinical trial of Tysabri against the condition last month, says company spokeswoman Naomi Aoki. Frederick Baron’s physician is an investigator on this study and well-familiar with its rules, but Baron isn’t eligible to participate in the trial because he is too sick, Aoki says.posted by ericb at 9:32 AM on October 15, 2008
In his letter, Andrew Baron wrote that FDA commissioner Andrew von Eschenbach ‘has granted special approval’ for his father to receive Tysabri, which essentially puts the ball in Biogen’s court. Under FDA rules, the company has discretion to give out the drug for such unapproved uses—what is known as ‘emergency use’ or ‘compassionate use’—but it chose not to, Aoki says. If something went wrong in an uncontrolled setting, it might lead to further restrictions on the drug’s use among existing patients with MS and Crohn’s disease, she says.
‘We concluded when we re-introduced Tysabri that we cannot make it available for uses other than the FDA-approved use, or for an FDA-approved clinical trial,’ Aoki says.
Still, I asked if this was a difficult decision, given the life and death nature of the matter. Andrew Baron’s letter says his father may have only a day or two left to live. ‘Of course, it’s a difficult decision for us. Our thoughts go out to the family. We don’t take this lightly, but we feel we have a responsibility to protect patients already on this drug who are benefiting from this drug,’ Aoki says.
When asked if legal liability is part of the company’s concern, Aoki referred back to her statement about protecting the interests of patients already on the drug.
This sort of ethical quandary has come up for other companies, most recently Plainfield, NJ-based PTC Therapeutics, which has refused to give a boy with muscular dystrophy an experimental medicine outside of the protocol of a clinical trial.”
It seems pretty clear what happened here.
Tysabri isn't a garden variety drug. It's used in people who are devastatingly ill, and it's been killing some of them with complications that never would have occurred if the drug hadn't been used. It's approved for MS and some GI disease - Crohn's? - yet those approvals are not set in stone, if more people die of PML it could well be yanked from the market and that would pretty well put Biogen out of business.
Because of this, Biogen contracted with the docs who are allowed to prescribe the medicine that they wouldn't use it off-label.
Now this guy is at the Mayo. They have Tysabri in their pharmacy. Some bright spark in the lab, probably an oncologic genius, decided that Tysabri would probably be effective against this guy's tumor because of its particular properties. The next logical step would have been to infuse the drug.
Instead, they called Biogen.
Biogen *can't* approve an off-label use, no matter what the FDA says. The FDA can approve a one-time compassionate use, but they can't change the regulations that say a drug manufacturer is not to promote or approve of off-label uses. Furthermore, they don't want to be affiliated with an off-label use of their drug because any bad outcome will surely reflect on them (and they might take liability for it - liability for drug-use decisions is something drug companies don't want, they prefer to keep that on the doctor.) Finally, if the patient were to suffer a drug-related bad outcome like PML, it might quash the ongoing myeloma trial or result in Tysabri's approval for any indication being yanked.
And that would be bad, not just for Biogen, but for the thousands, maybe millions of people worldwide who stand to benefit from a drug that has been properly investigated to determine when it can be used safely and when it can't.
There's more at stake here than just one guy's life or one corporation's profits. The right thing to do would be for the docs at the Mayo to infuse the drug - but the doctors are not in control of drug decisions, haven't been for many years; the lawyers and businessmen are. Those docs know if they infuse the drug they will get their permission to use it in future yanked, possibly for their entire hospital, and they daren't risk that.
This was a decision our society made years ago. It occurs on a smaller scale every day, when I prescribe the "right" drug for a patient and a high school graduate at a pharmacy benefit manager corporation (MedCo, Express Scripts, CareMark) faxes me an illiterate, illegible letter instructing me that the "right" drug will not be permitted, so use a "wrong" drug that is cheaper instead. I still bear the full liability for the effects of prescribing the "wrong" drug, too - PBMs are shielded by legislation.
posted by ikkyu2 at 10:42 AM on October 15, 2008 [6 favorites]
Tysabri isn't a garden variety drug. It's used in people who are devastatingly ill, and it's been killing some of them with complications that never would have occurred if the drug hadn't been used. It's approved for MS and some GI disease - Crohn's? - yet those approvals are not set in stone, if more people die of PML it could well be yanked from the market and that would pretty well put Biogen out of business.
Because of this, Biogen contracted with the docs who are allowed to prescribe the medicine that they wouldn't use it off-label.
Now this guy is at the Mayo. They have Tysabri in their pharmacy. Some bright spark in the lab, probably an oncologic genius, decided that Tysabri would probably be effective against this guy's tumor because of its particular properties. The next logical step would have been to infuse the drug.
Instead, they called Biogen.
Biogen *can't* approve an off-label use, no matter what the FDA says. The FDA can approve a one-time compassionate use, but they can't change the regulations that say a drug manufacturer is not to promote or approve of off-label uses. Furthermore, they don't want to be affiliated with an off-label use of their drug because any bad outcome will surely reflect on them (and they might take liability for it - liability for drug-use decisions is something drug companies don't want, they prefer to keep that on the doctor.) Finally, if the patient were to suffer a drug-related bad outcome like PML, it might quash the ongoing myeloma trial or result in Tysabri's approval for any indication being yanked.
And that would be bad, not just for Biogen, but for the thousands, maybe millions of people worldwide who stand to benefit from a drug that has been properly investigated to determine when it can be used safely and when it can't.
There's more at stake here than just one guy's life or one corporation's profits. The right thing to do would be for the docs at the Mayo to infuse the drug - but the doctors are not in control of drug decisions, haven't been for many years; the lawyers and businessmen are. Those docs know if they infuse the drug they will get their permission to use it in future yanked, possibly for their entire hospital, and they daren't risk that.
This was a decision our society made years ago. It occurs on a smaller scale every day, when I prescribe the "right" drug for a patient and a high school graduate at a pharmacy benefit manager corporation (MedCo, Express Scripts, CareMark) faxes me an illiterate, illegible letter instructing me that the "right" drug will not be permitted, so use a "wrong" drug that is cheaper instead. I still bear the full liability for the effects of prescribing the "wrong" drug, too - PBMs are shielded by legislation.
posted by ikkyu2 at 10:42 AM on October 15, 2008 [6 favorites]
Full disclosure, by the way: I own stock in Medco, Express Scripts, CVS/Caremark, and Altria Group. Considering the profits I am making from owning these stocks helps alleviate the feeling of frustration I have that their "products" do nothing but kill my patients on a regular, continuous basis.
posted by ikkyu2 at 10:53 AM on October 15, 2008
posted by ikkyu2 at 10:53 AM on October 15, 2008
Update. Fred Baron has received Tysabri.
I hope this has been a useful teaching vignette: it shows just how badly doctors' ability to advocate for their patients' best interest has been eroded. Eventually perhaps the system can change for the better.
posted by ikkyu2 at 5:22 PM on October 16, 2008 [2 favorites]
I hope this has been a useful teaching vignette: it shows just how badly doctors' ability to advocate for their patients' best interest has been eroded. Eventually perhaps the system can change for the better.
posted by ikkyu2 at 5:22 PM on October 16, 2008 [2 favorites]
Another Tysabri patient has PML
"Biogen Idec Inc. reported late yesterday that another patient contracted a potentially fatal brain disease after taking the multiple sclerosis drug Tysabri, the third case since the biotechnology giant reintroduced the treatment two years ago. The news sent shares tumbling 12.1 percent in after-hours trading."posted by ericb at 1:53 PM on October 30, 2008
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posted by fungible at 8:41 AM on October 14, 2008