FDA Challenges Stem Cell Companies As Patients Run Out Of Time
npr.org
npr.org
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).
* 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?
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.
In most proposed treatments the discussion is not if they are more or less "effective", the problem is that they don't work, and some of them even have side effects. Terminal ill people are often desperate and would try whatever is trendy: http://theincidentaleconomist.com/wordpress/the-rise-and-fal... This was a very expensive, dangerous and ineffective treatment.
Well, in this context, whether treatments work or "don't work" is the definition of effectiveness, and whether or not they have deleterious side effects is the definition of safety. But apart from that, I'll again point out that whether or not a treatment works is determined by what results the patient considers satisfactory, and whether or not something is safe depends on what risks the patient is willing to bear; generalizations simply don't suffice here.
If the unlikeliness of a procedure's success and the nature of the possible side-effects are known, then they can be communicated to the patient so that he can decide whether the higher probability of side effects is justified by the lower probability of relief according to his own hierarchy of values. If how well the treatment works and the nature of its side effects aren't known, then no one has the relevant knowledge to make an informed decision, and it still remains up to the patient to weigh the unquantifiable possibility of relief against the equally unquantifiable risk of a bad outcome.
There's simply no way around the fact that the question always boils down to a deeply personal value judgement that no third party has the right to usurp.
> Terminal ill people are often desperate and would try whatever is trendy
Right. They're desperate because they're terminally ill. They're going to die if they don't get cured, so their risk calculation is probably entirely skewed to the "reward" side of the equation, no matter how remote it is. They've literally got nothing to lose, so it's unconscionable to forcibly deny them the right to "lose" it, third parties' objections to "trendiness" being entirely immaterial.
> http://theincidentaleconomist.com/wordpress/the-rise-and-fal...
Perhaps this was an ineffective treatment, but the question of whether it should be paid for by insurance is entirely unrelated to the question of whether people should be forbidden from choosing to undergo the procedure at all. The FDA has nothing to do with the former question, and everything to do with the latter; in the article itself, there's no indication that anyone actually tried to forcibly prohibit the ABMT procedure. Doctors simply stopped recommending it after it was demonstrated not to be of much value. If, in some specific case in the future, patient and doctor agree that the ABMT procedure might be worth trying, no third party is going to actively overrule their judgment. But, unfortunately, this isn't the case for certain other procedures.
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=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.
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.
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-anti...
If FDA approval was optional (perhaps requiring a big "NOT FDA APPROVED" label), would the number of people who die from taking unapproved drugs (and who otherwise would have lived) be more than the number who die from being denied a drug (and who otherwise would have lived)?
As I write this, it reminds me a bit of the IP issue, and whether the patent system really is a net benefit or not.
When did utilitarianism become an acceptable justification for usurping the right of individuals to make their own risk-reward tradeoffs?
Treating individuals' health as something to be optimized with respect to a putative macro-level abstraction of society as opposed to treating individuals' health as something to be optimized with respect to each individual's particular happiness is the problem here.
The post I replied to was also an implicit argument from principle, in treating the net-lives-saved measurement, with respect to the aggregate population, as the overriding decision criterion. It's just that this is a bad principle in that it gives "saving" the life of someone living in misery due to an uncurable but not terminal disease the same moral weight as curing someone of a terminal disease, and gives the potential death of someone consciously willing to risk death in an attempt to alleviate suffering the same moral weight as the potential death of someone denied access to a treatment they were willing to try and which might have saved their lives.
http://www.cato.org/pubs/regulation/regv27n2/v27n2-8.pdf
http://marginalrevolution.com/marginalrevolution/2011/10/and...
http://fdareview.org/harm.shtml
The delay and large reduction in the total number of new
drugs has had terrible consequences. It is difficult to
estimate how many lives the post-1962 FDA controls have
cost, but the number is likely to be substantial;
Gieringer (1985) estimates the loss of life from delay
alone to be in the hundreds of thousands (not to mention
millions of patients who endured unnecessary morbidity). ...
If the U.S. system resulted in appreciably safer drugs, we
would expect to see far fewer postmarket safety
withdrawals in the United States than in other countries.
Bakke et al. (1995) compared safety withdrawals in the
United States with those in Great Britain and Spain, each
of which approved more drugs than the United States during
the same time period. Yet, approximately 3 percent of all
drug approvals were withdrawn for safety reasons in the
United States, approximately 3 percent in Spain, and
approximately 4 percent in Great Britain. There is no
evidence that the U.S. drug lag brings greater safety.
The only way to prove this definitively is a side-by-side experiment with a new jurisdiction in which patients and entrepreneurs alike are free to choose and the FDA has no power.
The real question is the unpopular one: by what right does the FDA insert itself between doctor and patient, between drug manufacturer and doctor?
Ah, dogma. It's killing us.
Some drugs investigations are discontinued because they cause complications or deaths in the small test groups. Another important number is how many additional dead could have been if everyone could take unapproved treatments.
And most people would sign whatever the doctor tells them to sign, for example a "NOT FDA APPROVED"-waiver. People thrust whoever has a white coat and promises a cure. There has been problems with peopled enrolled in official experimental drug test, that didn't understand the details, but had signed just another "usual" form handled by the doctors.
Why does the Thalidomide tragedy justify the FDA testing for _effectiveness_?
How do you distinguish between "this patient is making an informed decision to opt out and use this unapproved drug" and "this patient is an idiot who thinks that throwing money at a "doctor" to mainline bleach will cure his disease"?
First, many (most?) medical advances seem crazy and kooky because they haven't been tried before. So it's not usually as obvious as "this is a proven scam" vs. "this really works". It's much more frequently "this is unproven". And like Barry Marshall's famous self-experiment with H. pylori, someone has to be first for it to ever get proven.
Second, when the government gets it wrong, it gets it catastrophically wrong. The USDA Food Pyramid recommending "6-11 servings of grain" is still being slavishly followed, and in the fullness of time we might well find it partially responsible for the epidemic of obesity and type II diabetes. The FDA is still doing Phase I/II/III clinical trials despite all the evidence in favor of adaptive trials. And tens of millions of people were irradiated by TSA x-ray scanners fast-tracked through the FDA approval process, scanners criticized by UCSF scientists, scanners which have now (finally) been withdrawn. These errors are magnified in impact because no one can opt-out, because a .gov has a bully pulpit, and because strong political incentives exist to silence criticisms.
Third, if people have the right to euthanasia, or the right to walk near bridges, I do believe they have the right to try what treatments they want. Frankly I don't consider someone else's medical affairs my business, anymore than I'd ask why they had an abortion. You can argue that vaccinations present a public health issue, and I might agree with you there. But otherwise this strikes me as a right to privacy and right to bodily integrity issue.
btw: "mainlining bleach" is not an exaggeration: http://www.youtube.com/watch?v=H7tkyK8r3yw
People can inject just about anything they want into
themselves
http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=19981...
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 United States Constitution.4 The United States
Supreme Court previously addressed and rejected this
argument in Rutherford. In Rutherford, cancer patients
requested the right to use Laetrile, arguing, as does
Plaintiff, that for terminally ill patients the
effectiveness or safety of the proposed treatment is
irrelevant since such treatment is a last chance effort.
However, as identified by the Supreme Court in Rutherford,
to permit terminally ill patients to seek any type of
treatment regardless of the effectiveness of such treatment
would create a cottage industry existing solely to provide
potential panaceas to highly vulnerable patients. The
language of the Supreme Court in rejecting the Laetrile
argument is equally applicable here.
"If history is any guide, this new market would not be long
overlooked. Since the turn of the century, resourceful
entrepreneurs have advertised a wide variety of purportedly
simple and painless cures for cancer, including liniments
of turpentine, mustard, oil, eggs, and ammonia; peat moss;
arrangements of colored floodlamps; pastes made from
glycerin and limburger cheese; mineral tablets; and
`Fountain of Youth' mixtures of spices, oil, and suet. In
citing these examples, we do not, of course, intend to
deprecate the sincerity of Laetrile's current proponents,
or to imply any opinion on whether that drug may ultimately
prove safe and effective for cancer treatment. But this
historical experience does suggest why Congress could
reasonably have determined to protect the terminally ill,
no less than other patients, from the vast range of self-
styled panaceas that inventive minds can devise."
The absolute worst case scenario is that they lose some money and die a little sooner. The best case scenario is that they live!
To say that someone else can or should have the power to constrain another human in this way, to keep them from a chance at living, in the name of "protecting" them from doctors or companies...well, we are likely at a fundamental philosophical impasse. Which is why I return to my original point. Feel free to stay in the United States with the FDA. Those with a different cast of mind need a jurisdiction where we can take conscious risks, where we aren't "protected" from medical innovation.
> The absolute worst case scenario is that they lose some money and die a little sooner. The best case scenario is that they live!
No, the worse case scenario is that charlatans, given a license to operate with perceived legitimacy by an "opt-out" system, convince sick people to neglect real treatment for woo. In the vast majority of cases, your best case scenario never happens.
We have a system designed to separate the woo from the real. Despite what one might think from the narrative you paint, human trials are involved in this system and sick people receive experimental treatments every day in a controlled fashion. Perhaps this process could be streamlined, but it must stay in place.
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.
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.
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?
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.
This already happens in Koreatown.
http://la.curbed.com/archives/2010/11/beyond_boba_tea_koreat...
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.
No thanks. I'll take the imperfect regulation over the "free market" any day.
Why not? Who else can be more qualified to weigh the pros and cons of a novel treatment than the patient himself? What we're talking about here is the act of applying one's own value system to the likely distribution of results associated with that treatment. If the patient is properly informed of the latter data, then the remaining variable is the set of personal risk-reward value judgments that only the patient himself has access to. If the patient is not properly informed of the relevant data, well, then, why isn't he?
If the patient himself isn't capable of weighing the options, and neither is the doctor, that means that even more remote people who aren't familiar with the particulars of the case at hand, are going to be less likely, not more likely, to make a good decision. You're going in the wrong direction here.
> And then you have desperately people who might normally be more skeptical but will believe just about anything if they or a loved one fall ill.
Right. This is part of the point that I'm making; people who are "desparate" because they're literally in life-or-death situations will tend to have a much more idiosyncratic and organic hierarchy of risk-reward tradeoffs that isn't likely to be captured by generalized presumptions. You're trying to cast "desperation" as a impediment to informed decision-making, when in reality, "desparation" displays the underlying priorities of the patient in much clearer relief than comparative indifference ever could. People are desperate precisely because of the drastic implications of the situation, which means that it's much more important for the patient to make a choice compatible with his underlying value system than it would be in other, less impactful situations.
It's not a question of people being willing to "believe just about anything" to alleviate a horrific illness; it's their willingness to try even remote options, because even a tiny chance is better than none that you're not giving due consideration to.
> I'll take the imperfect regulation over the "free market" any day.
It's not about a "free market" as much as it's about your individual freedom to determine the disposition of your own life and health
Of course, if you'd rather trust one group of third parties over both another group of third parties and your own judgment, that's your prerogative. If the FDA were merely a certification group, you'd still have the option of relying only on that FDA certification as your sole decision criterion, and not considering the matter any more deeply than that. But you've got absolutely no right to demand that others outsource their own value judgments to your preferred third parties.
The FDA forces their own therapy of "do nothing for yourself" on the patient when the patient has run out of options. If something costs me my life, I don't care if something else would have cost me my fortune. At least my right to defend my own life would not have been infringed on by the FDA.
The problem is, people want to believe. That's a bug in our mental firmware. The FDA is an ugly, flawed, but arguably vital workaround for that bug. If you want to get rid of it, you need to offer another fix for the bug.
Here is an idea of a work around.
First, FDA "recommendations" shouldn't be mandatory. Even things the FDA claims to be safe can be more harmful than some of the things that it warns are unsafe.
Second, There should be competing private agencies other than just the FDA. Their reputations would rely on how safe their "approved" medication and procedures turn out to be.
Third, A patient looking for a treatment should be able to look up the ratings of doctors, medications, procedures. If they find a potential scam artists, they will see the negative reviews and reports of patients who died under their care. They will have valuable information to make their medical decisions that they don't have today. The ratings and information would be maintained by private agencies and the FDA. Kind of like a credit score for doctors, medications, and medical procedures.
This obviously doesn't solve every single problem, but I think its a pretty good start.