9 ms·

FDA Challenges Stem Cell Companies As Patients Run Out Of Time

29 points by winstonsmith 14y ago

tomjen3 14y ago
It is shit like this that makes me what to shut down the FDA and ban anybody who has every worked there from having a job again. Isn't it enough that sick people have to suffer from their sickness? Does these nazies have to destroy their only chance of a better life? If it turns out that the cure doesn't work, then let the estate sue the company -- don't go around ruining peoples life just to increase your own power.
- geoffschmidt 14y ago
  There's another side though. Do you want to see pseudoscience stem cell "therapies" sold on late night infomercials as a panacea? A balance has to be struck between the number of people that could potentially be helped and the number of people that could potentially be harmed. We can debate how this balance should be struck without assuming bad faith on the part of those that disagree with us. On the internet, when startups overpromise, misrepresent, and take risks, the worst that usually happens is that a few million people lose their data or get their embarrassing photos shared with their coworkers. In health care the consequences of an overhyped technology are far more grave.
  - tomjen3 14y ago
    How would you react if your father couldn't get the kind of treatment that he needed because the FDA wouldn't let him? If the supplier is lying, that is fraud (and can be prosecuted even after the fact); If they find out that it was a good deal after he has died, how does that benefit him?
    - gus_massa 14y ago
      They will say that it's not a fraud. It was only an experimental treatment fundamented in abstract "principles" and they had some signs that it improves some of the symptoms in a few patients. Regrettably all the new studies proved that it doesn't work but the risk were clear in the small letter of the contract. Luckily now they realized that this treatment can be possible useful for another illness and/or they have another experimental treatment for this illness.
  - ataggart 14y ago
    >Do you want to see pseudoscience stem cell "therapies" sold on late night infomercials as a panacea? Mandatory FDA approval is orthogonal to over-the-counter sales. Surely you don't think the government should prohibit a medical decision between a patient and her doctor... > when startups overpromise, misrepresent, and take risks, the worst that usually happens... Yes, the risk people are willing to bear is proportional to the damage it can cause. This is probably why terminal patients are willing to risk experimental drugs.
  - rdouble 14y ago
    Do you want to see pseudoscience stem cell"therapies" sold on late night infomercials as a panacea?" This already happens in Koreatown. http://la.curbed.com/archives/2010/11/beyond_boba_tea_koreatown_galleria_tenant_advertises_pet_cloning.php http://la.curbed.com/archives/2010/11/beyond_boba_tea_koreat...
- GiraffeNecktie 14y ago
  I don't see that shutting down the FDA is a good alternative. There are countless therapies that are dangerous and/or ineffective and plenty of unscrupulous or incompetent people who want to flog them. Yes there's a human cost to regulation, but that's nothing compared to the cost of opening the floodgates to every half-baked idea out there, not to mention the damage to medical science when the medical trials are bypassed.
  - Gormo 14y ago
    > There are countless therapies that are dangerous and/or ineffective and plenty of unscrupulous or incompetent people who want to flog them. There are countless regulatory policies that are dangerous and/or ineffective and plenty of unscrupulous or incompetent politicians who want to flog them. So what process validates the "safety" and "effectiveness" of the FDA's universal-scope regulations? Certainly, given that you and I can't opt out of them as we can with drugs and medical procedures, the danger of bad policy can be far graver than the danger of bad medicine, yet there's no regulation of regulatory policy at all! I understand the desire to protect people from fraud and deceit, especially when the consequences of bad medicine are literally life-or-death, but surely the proper way to accomplish this end is to supply people with the data that will allow them to make informed decisions, rather than usurping their right make decisions in the first place. The FDA should function as a certification body, evaluating drugs and treatments, and informing patients as to the likely results of using each, but it should not have the power to prohibit anyone from undergoing any treatment or taking any drug.
    - GiraffeNecktie 14y ago
      Sounds good in theory but I don't think the average doctor, let alone the average patient, is capable of independently weighing the pros and cons of every novel treatment. And then you have desperately people who might normally be more skeptical but will believe just about anything in the pain and torment of a moment when they or a loved one falls ill. No thanks. I'll take the imperfect regulation over the "free market" any day.
      - 14y ago
gus_massa 14y ago
What's the difference between buying hard disk and getting a medical therapy? * You can ask for a chargeback. * You get a one year guaranty. * You are not desperate. * If it goes wrong, it's almost sure you aren't going to die for this. Let's suppose that someone tries to sell a homeopathic "hard" disk: The information is stored homeopathically in water and read later. Is it a possible business model? It's important that someone control the medical procedures and control that the effectively work. There have been a lot of horror stories of medical procedures that were totally ineffective of even dangerous. A similar case, with less emotional charge, is the need of regulation of airplanes. > Wilkinson tells NPR's Laura Sullivan she got better immediately — 11 of her 25 MS-related symptoms have either improved or completely disappeared. I hope that it's true, but I have a lot of doubts. * How were these improvements measured? Self-reported or an independent test? * Is this case reported in a medical journal? * Can be only the placebo effect? * Is this only a spontaneous temporal remission? * Is she taking another treatment?
- Gormo 14y ago
  > It's important that someone control the medical procedures and control that the effectively work. It's valuable to have a impartial observer who can quantify the characteristics of a new drug or technique in a valid and useful way, and if an unbiased organization were to produce histograms of likely consequences of using each drug/procedure, and supply them to patients as an input into their decision-making, I'm certain that it would be universally lauded. But that's just supplying the data. Determining whether something is "safe" or "effective" isn't just about quantifying data; it's fundamentally a value judgment. Whether a drug is "safe" depends on how much risk the patient is willing to bear. Whether it's "effective" depends on what results the patient finds satisfactory. These are deeply intimate and personal judgments that are rightly the patient's to make. The problem with the FDA is that it doesn't actually supply the data in a way accessible to patients at all; instead it usurps their inherent right to make their own value judgments regarding their health, and simply allows or denies everyone access to the drug/procedure according to its own criteria. > A similar case, with less emotional charge, is the need of regulation of airplanes. Well, no, this isn't similar at all. It's far, far easier to generalize the risk/reward tradeoffs of flying in an airplane: people want to arrive at their intended destination on time, without being killed or injured in the process. A plane trip lasts a few hours, and has essentially no lasting consequences for a passenger once he disembarks; unlike medicine, flying on a plane doesn't have vast consequences for the quality and quantity of one's subsequent life. Plane trips, like hard drives, are both commodity goods for which the intended results and acceptable risks can be assumed in advance. Medicine is not.
ramanujan 14y ago
The issue is not really whether these treatments work or not. Every new treatment is by necessity unproven and more risky than an established one. Some people are early adopters or terminal patients with high risk-tolerances; others would rather suffer for a while than chance a drug that might make things worse. Both of those are fine as preferences. The trouble arises because every US citizen is forced to have the global minimum of risk-tolerances across the population. It would be as if you could not try a development version till it became user-friendly enough for your grandparents. Specifically, in the US, you can't be an early adopter: the FDA does not allow citizens to opt-out unless they leave the borders of the USA. Indeed, in Cowan vs. US (1998) it successfully sued in federal court to prevent a dying AIDS patient from trying an experimental drug: http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=199812405FSupp2d1235_11084.xml&docbase=CSLWAR2-1986-2006&SizeDisp=7 http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=19981... Plaintiff requests that Dr. Davis be authorized to inject Plaintiff with the with the experimental goat neutralizing antibody drug [1] and that the FDA be enjoined from interfering with Dr. Davis' treatment of Plaintiff. ... The Court is sympathetic to Plaintiff's situation. However, the law is very clear, and under the current statutes and regulations, Plaintiff's physician may not administer the goat neutralizing antibody drug absent prior approval of the FDA. In Court, Plaintiff argued that he should have the right to take whatever treatment he wishes due to his terminal condition regardless of whether the FDA approves the treatment as effective or safe, and that to prohibit him from taking the treatment he wishes violates his rights under the US Constitution. ... This Court is in no way criticizing the intentions of Plaintiff and his physician or the potential effectiveness of the proposed treatment. Plaintiff's physician should pursue approval of his Investigational New Drug application as quickly as possible. Plaintiff's doctor must obtain appropriate approval through the proper regulatory authorities. As much as this Court may empathize with Plaintiff, the authority to provide some type of exemptions for individuals such as Plaintiff rests with Congress and not with this Court. Plaintiff was denied. Plaintiff died. Who knows whether the drug would have saved him, but he wasn't given the chance to try. Occasionally, if you have tremendous political connections, you can get a waiver, like Fred Baron: http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-bill-clinton-help-fred-baron-get-tysabri/ http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-b... “We did a safety review, consulted with experts on PML, and worked closely with FDA to come up with a risk management program that allowed us to bring it back on the market in a way that limited its use,” a Biogen spokeswoman told the Law Blog. The plan prohibits giving Tysabri for unapproved uses. Biogen Idec is running an early-stage trial of the drug in multiple myeloma, but Baron doesn’t meet the criteria to participate. Baron’s a prominent donor to the Democratic party, and many of his powerful friends, including Lance Armstrong and Bill Clinton, made appeals on his behalf. And the family agreed not to sue if anything goes wrong. Ultimately, his doctors at the Mayo Clinic worked directly with the FDA to find a “legal basis” for giving Baron Tysabri. The deal was announced on Baron’s son’s blog late yesterday. So, if you are wealthy you can travel outside the US to opt-out (though US-based companies will usually not administer treatment for fear of getting on the FDA's bad side). And if you are politically connected you can sometimes get an experimental treatment, like Baron. But this is not really optimal. If you are an academic, you accept the concept of QC/quality checking, but you aren't stuck with just one journal to submit to. You can revise and resubmit somewhere else. And if you are an end-user, you don't have to take a reviewer's opinion into account when choosing between movies, books, bikes, or virtually any other physical good with star ratings on Amazon.com. Except for drugs. Then you, as the end-user, cannot opt-out and take the FDA's opinions with a grain of salt. In part this is because the FDA will sue you directly. In part it is because companies that even think of trying this route will get slapped by the FDA for trying to game the system, and subsequently find their approvals slowed or (nowadays) outright denied. We need to carve out a jurisdiction where patients and entrepreneurs alike are free to take informed risks, recognizing up front that sick people do die in medicine, and also recognizing that society already allows people to take incredible risks in other contexts (joining the military, bungee jumping, walking tightropes). Whether that new jurisdiction is Singapore, or Estonia, or a seastead, or a medical cruise ship, or something else is to be determined. But that has to be the goal. [1] In case a "goat antibody" sounds weird to you, it's a common thing in molecular biology. Google it, or see for example Thermo's web page: http://www.pierce-antibodies.com/custom-antibodies/goat-antibody-production-protocols.cfm http://www.pierce-antibodies.com/custom-antibodies/goat-anti...
jeffdavis 14y ago
It seems like this is what the FDA is designed to do. Cost of FDA: someone might miss out on a beneficial treatment because it has not been reviewed by the FDA (and may never be, given the cost of FDA approval). Benefit of FDA: prevents charlatans from fleecing medically-ignorant people (which is pretty much everyone) of their life savings just before they die, which might be better spent on proven treatments or left as inheritance. At some point, we decided that the benefits were worth the costs. Unfortunately, the benefits tend to be obvious and the costs tend to be more hidden. That usually results in overdoing it (in this case, perhaps overburdening the development and use of new treatments).

29 points by winstonsmith 14y ago

It is shit like this that makes me what to shut down the FDA and ban anybody who has every worked there from having a job again. Isn't it enough that sick people have to suffer from their sickness? Does these nazies have to destroy their only chance of a better life? If it turns out that the cure doesn't work, then let the estate sue the company -- don't go around ruining peoples life just to increase your own power.

geoffschmidt 14y ago
There's another side though. Do you want to see pseudoscience stem cell "therapies" sold on late night infomercials as a panacea? A balance has to be struck between the number of people that could potentially be helped and the number of people that could potentially be harmed. We can debate how this balance should be struck without assuming bad faith on the part of those that disagree with us. On the internet, when startups overpromise, misrepresent, and take risks, the worst that usually happens is that a few million people lose their data or get their embarrassing photos shared with their coworkers. In health care the consequences of an overhyped technology are far more grave.
- tomjen3 14y ago
  How would you react if your father couldn't get the kind of treatment that he needed because the FDA wouldn't let him? If the supplier is lying, that is fraud (and can be prosecuted even after the fact); If they find out that it was a good deal after he has died, how does that benefit him?
  - gus_massa 14y ago
    They will say that it's not a fraud. It was only an experimental treatment fundamented in abstract "principles" and they had some signs that it improves some of the symptoms in a few patients. Regrettably all the new studies proved that it doesn't work but the risk were clear in the small letter of the contract. Luckily now they realized that this treatment can be possible useful for another illness and/or they have another experimental treatment for this illness.
- ataggart 14y ago
  >Do you want to see pseudoscience stem cell "therapies" sold on late night infomercials as a panacea? Mandatory FDA approval is orthogonal to over-the-counter sales. Surely you don't think the government should prohibit a medical decision between a patient and her doctor... > when startups overpromise, misrepresent, and take risks, the worst that usually happens... Yes, the risk people are willing to bear is proportional to the damage it can cause. This is probably why terminal patients are willing to risk experimental drugs.
- rdouble 14y ago
  Do you want to see pseudoscience stem cell"therapies" sold on late night infomercials as a panacea?" This already happens in Koreatown. http://la.curbed.com/archives/2010/11/beyond_boba_tea_koreatown_galleria_tenant_advertises_pet_cloning.php http://la.curbed.com/archives/2010/11/beyond_boba_tea_koreat...
GiraffeNecktie 14y ago
I don't see that shutting down the FDA is a good alternative. There are countless therapies that are dangerous and/or ineffective and plenty of unscrupulous or incompetent people who want to flog them. Yes there's a human cost to regulation, but that's nothing compared to the cost of opening the floodgates to every half-baked idea out there, not to mention the damage to medical science when the medical trials are bypassed.
- Gormo 14y ago
  > There are countless therapies that are dangerous and/or ineffective and plenty of unscrupulous or incompetent people who want to flog them. There are countless regulatory policies that are dangerous and/or ineffective and plenty of unscrupulous or incompetent politicians who want to flog them. So what process validates the "safety" and "effectiveness" of the FDA's universal-scope regulations? Certainly, given that you and I can't opt out of them as we can with drugs and medical procedures, the danger of bad policy can be far graver than the danger of bad medicine, yet there's no regulation of regulatory policy at all! I understand the desire to protect people from fraud and deceit, especially when the consequences of bad medicine are literally life-or-death, but surely the proper way to accomplish this end is to supply people with the data that will allow them to make informed decisions, rather than usurping their right make decisions in the first place. The FDA should function as a certification body, evaluating drugs and treatments, and informing patients as to the likely results of using each, but it should not have the power to prohibit anyone from undergoing any treatment or taking any drug.
  - GiraffeNecktie 14y ago
    Sounds good in theory but I don't think the average doctor, let alone the average patient, is capable of independently weighing the pros and cons of every novel treatment. And then you have desperately people who might normally be more skeptical but will believe just about anything in the pain and torment of a moment when they or a loved one falls ill. No thanks. I'll take the imperfect regulation over the "free market" any day.
    - 14y ago

gus_massa 14y ago

What's the difference between buying hard disk and getting a medical therapy? * You can ask for a chargeback. * You get a one year guaranty. * You are not desperate. * If it goes wrong, it's almost sure you aren't going to die for this. Let's suppose that someone tries to sell a homeopathic "hard" disk: The information is stored homeopathically in water and read later. Is it a possible business model? It's important that someone control the medical procedures and control that the effectively work. There have been a lot of horror stories of medical procedures that were totally ineffective of even dangerous. A similar case, with less emotional charge, is the need of regulation of airplanes. > Wilkinson tells NPR's Laura Sullivan she got better immediately — 11 of her 25 MS-related symptoms have either improved or completely disappeared. I hope that it's true, but I have a lot of doubts. * How were these improvements measured? Self-reported or an independent test? * Is this case reported in a medical journal? * Can be only the placebo effect? * Is this only a spontaneous temporal remission? * Is she taking another treatment?

Gormo 14y ago
> It's important that someone control the medical procedures and control that the effectively work. It's valuable to have a impartial observer who can quantify the characteristics of a new drug or technique in a valid and useful way, and if an unbiased organization were to produce histograms of likely consequences of using each drug/procedure, and supply them to patients as an input into their decision-making, I'm certain that it would be universally lauded. But that's just supplying the data. Determining whether something is "safe" or "effective" isn't just about quantifying data; it's fundamentally a value judgment. Whether a drug is "safe" depends on how much risk the patient is willing to bear. Whether it's "effective" depends on what results the patient finds satisfactory. These are deeply intimate and personal judgments that are rightly the patient's to make. The problem with the FDA is that it doesn't actually supply the data in a way accessible to patients at all; instead it usurps their inherent right to make their own value judgments regarding their health, and simply allows or denies everyone access to the drug/procedure according to its own criteria. > A similar case, with less emotional charge, is the need of regulation of airplanes. Well, no, this isn't similar at all. It's far, far easier to generalize the risk/reward tradeoffs of flying in an airplane: people want to arrive at their intended destination on time, without being killed or injured in the process. A plane trip lasts a few hours, and has essentially no lasting consequences for a passenger once he disembarks; unlike medicine, flying on a plane doesn't have vast consequences for the quality and quantity of one's subsequent life. Plane trips, like hard drives, are both commodity goods for which the intended results and acceptable risks can be assumed in advance. Medicine is not.

ramanujan 14y ago

The issue is not really whether these treatments work or not. Every new treatment is by necessity unproven and more risky than an established one. Some people are early adopters or terminal patients with high risk-tolerances; others would rather suffer for a while than chance a drug that might make things worse. Both of those are fine as preferences. The trouble arises because every US citizen is forced to have the global minimum of risk-tolerances across the population. It would be as if you could not try a development version till it became user-friendly enough for your grandparents. Specifically, in the US, you can't be an early adopter: the FDA does not allow citizens to opt-out unless they leave the borders of the USA. Indeed, in Cowan vs. US (1998) it successfully sued in federal court to prevent a dying AIDS patient from trying an experimental drug: http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=199812405FSupp2d1235_11084.xml&docbase=CSLWAR2-1986-2006&SizeDisp=7 http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=19981... Plaintiff requests that Dr. Davis be authorized to inject Plaintiff with the with the experimental goat neutralizing antibody drug [1] and that the FDA be enjoined from interfering with Dr. Davis' treatment of Plaintiff. ... The Court is sympathetic to Plaintiff's situation. However, the law is very clear, and under the current statutes and regulations, Plaintiff's physician may not administer the goat neutralizing antibody drug absent prior approval of the FDA. In Court, Plaintiff argued that he should have the right to take whatever treatment he wishes due to his terminal condition regardless of whether the FDA approves the treatment as effective or safe, and that to prohibit him from taking the treatment he wishes violates his rights under the US Constitution. ... This Court is in no way criticizing the intentions of Plaintiff and his physician or the potential effectiveness of the proposed treatment. Plaintiff's physician should pursue approval of his Investigational New Drug application as quickly as possible. Plaintiff's doctor must obtain appropriate approval through the proper regulatory authorities. As much as this Court may empathize with Plaintiff, the authority to provide some type of exemptions for individuals such as Plaintiff rests with Congress and not with this Court. Plaintiff was denied. Plaintiff died. Who knows whether the drug would have saved him, but he wasn't given the chance to try. Occasionally, if you have tremendous political connections, you can get a waiver, like Fred Baron: http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-bill-clinton-help-fred-baron-get-tysabri/ http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-b... “We did a safety review, consulted with experts on PML, and worked closely with FDA to come up with a risk management program that allowed us to bring it back on the market in a way that limited its use,” a Biogen spokeswoman told the Law Blog. The plan prohibits giving Tysabri for unapproved uses. Biogen Idec is running an early-stage trial of the drug in multiple myeloma, but Baron doesn’t meet the criteria to participate. Baron’s a prominent donor to the Democratic party, and many of his powerful friends, including Lance Armstrong and Bill Clinton, made appeals on his behalf. And the family agreed not to sue if anything goes wrong. Ultimately, his doctors at the Mayo Clinic worked directly with the FDA to find a “legal basis” for giving Baron Tysabri. The deal was announced on Baron’s son’s blog late yesterday. So, if you are wealthy you can travel outside the US to opt-out (though US-based companies will usually not administer treatment for fear of getting on the FDA's bad side). And if you are politically connected you can sometimes get an experimental treatment, like Baron. But this is not really optimal. If you are an academic, you accept the concept of QC/quality checking, but you aren't stuck with just one journal to submit to. You can revise and resubmit somewhere else. And if you are an end-user, you don't have to take a reviewer's opinion into account when choosing between movies, books, bikes, or virtually any other physical good with star ratings on Amazon.com. Except for drugs. Then you, as the end-user, cannot opt-out and take the FDA's opinions with a grain of salt. In part this is because the FDA will sue you directly. In part it is because companies that even think of trying this route will get slapped by the FDA for trying to game the system, and subsequently find their approvals slowed or (nowadays) outright denied. We need to carve out a jurisdiction where patients and entrepreneurs alike are free to take informed risks, recognizing up front that sick people do die in medicine, and also recognizing that society already allows people to take incredible risks in other contexts (joining the military, bungee jumping, walking tightropes). Whether that new jurisdiction is Singapore, or Estonia, or a seastead, or a medical cruise ship, or something else is to be determined. But that has to be the goal. [1] In case a "goat antibody" sounds weird to you, it's a common thing in molecular biology. Google it, or see for example Thermo's web page: http://www.pierce-antibodies.com/custom-antibodies/goat-antibody-production-protocols.cfm http://www.pierce-antibodies.com/custom-antibodies/goat-anti...

jeffdavis 14y ago

It seems like this is what the FDA is designed to do. Cost of FDA: someone might miss out on a beneficial treatment because it has not been reviewed by the FDA (and may never be, given the cost of FDA approval). Benefit of FDA: prevents charlatans from fleecing medically-ignorant people (which is pretty much everyone) of their life savings just before they die, which might be better spent on proven treatments or left as inheritance. At some point, we decided that the benefits were worth the costs. Unfortunately, the benefits tend to be obvious and the costs tend to be more hidden. That usually results in overdoing it (in this case, perhaps overburdening the development and use of new treatments).