

FDA Challenges Stem Cell Companies As Patients Run Out Of Time - winstonsmith
http://www.npr.org/2013/02/02/170942324/fda-challenges-stem-cell-companies-as-patients-run-out-of-time

======
ramanujan
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=19981...](http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=199812405FSupp2d1235_11084.xml&docbase=CSLWAR2-1986-2006&SizeDisp=7)

    
    
      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-b...](http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-bill-
clinton-help-fred-baron-get-tysabri/)

    
    
      “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-
anti...](http://www.pierce-antibodies.com/custom-antibodies/goat-antibody-
production-protocols.cfm)

~~~
ataggart
Counterfactuals are hard, but I wonder if anyone has studied whether or not
the FDA is actually a net saver of lives. It seems to me that every time the
FDA approves a new drug that will save X lives a year, it has necessarily cost
X lives every year that the drug was delayed in the mandatory approval
pipeline.

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.

~~~
Gormo
> Counterfactuals are hard, but I wonder if anyone has studied whether or not
> the FDA is actually a net saver of lives.

When did utilitarianism become an acceptable justification for usurping the
right of individuals to make their own risk-reward tradeoffs?

~~~
ataggart
Arguing from principle likely won't get you very far with people arguing from
assumed utility.

~~~
Gormo
I'm not arguing from principle as much as I'm pointing out that the utility
value is itself subjective with respect to each individual; I'm criticizing
the socio-political doctrine of utilitarianism which pursues the "greatest
good for the greatest number" in accordance with a single, generalized
definition of "good" applied to all, irrespective of the divergent preferences
of each.

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.

------
jeffdavis
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).

------
gus_massa
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
> 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.

~~~
gus_massa
>> It's important that someone control the medical procedures and control that
the effectively work.

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...](http://theincidentaleconomist.com/wordpress/the-rise-and-fall-of-bone-
marrow-transplantation-for-breast-cancer-a-tragic-success-story/) This was a
very expensive, dangerous and ineffective treatment.

~~~
Gormo
> 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.

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...](http://theincidentaleconomist.com/wordpress/the-rise-and-fall-of-
> bone-marrow-transplantation-for-breast-cancer-a-tragic-success-story/)

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.

------
tomjen3
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
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
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
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.

