
The FDA is powerless to stop right-to-try profiteers - aaavl2821
https://www.statnews.com/2018/06/20/right-to-try-opportunism/
======
Thriptic
I totally believe right to try should be a thing. Who is the FDA or anyone
else to tell dying patients that they can't put something in their body to try
to save their life? The reason this is necessary is because under the previous
compassionate use policy, pharma conpanies are required to report any adverse
events from these patients using these products to FDA which could result in a
halt of their independent clinical trials running in parallel. Because the
patients utilizing compassionate use many be in any stage of disease with
multiple comorbidities, it makes root cause analysis of adverse events much
more difficult than in a clinical trial, and presents a much higher risk to
developers.

This strongly disincentivizes companies from leveraging the compassionate use
pipeline, and patients are left to die and suffer as a result.

Also as far as profiteering goes, there is no way that an insurance company
would reimburse for any untested therapy which by default would keep costs
down, and patients would still be required to get a prescription from their
physician who would hopefully review the trial data. The idea that this would
be used as an end run around FDA regulation for huge profits is moronic.

~~~
kazinator
People shouldn't be charged to serve as guinea pigs. On the contrary, they
should be paid.

Here is a problem: if a pharma company can simply make money from someone's
desperation, they have a reduced incentive to actually make the drug work.

The situation is reduced to a simple scam: the business model of selling some
desperate people the promise of a cure which is just snake oil.

We always have to consider the effect of how the flow of money in a situation
creates incentives that have unintended consequences.

~~~
Thriptic
What are you talking about? Pharma conpanies want therapies to work because
billing insurance companies at scale is where the money is, not individual
patients who can't pay. That requires FDA approval. Historically iirc
compassionate use distributions were free, and it is likely that will continue
to be the case because the amount of money companies can make off of
individual patients is rounding error relative to bringing a product to market
and getting widespread approval for reimbursement.

Also, the whole point is that these people are not guinea pigs. They are
allowed access to promising therapies in late stage clinical trials in the
hopes that they will help them, not just random investigational compounds.
Let's remember that these requests would require physician approval.

The result of blocking this is to say that dying patients should just die,
tough shit, but hey at least they will die with more money right?

~~~
jonhendry18
"Pharma conpanies want therapies to work because billing insurance companies
at scale is where the money is"

Small-scale quackery scams can be highly profitable even if there's never any
move to large scale production. It doesn't cost much to run a clinic giving
coffee enemas, but you can charge an arm and a leg. At that rate a few
patients a year can keep you riding in Ferraris.

"hey at least they will die with more money right?"

Their kids might appreciate not being left homeless by an unethical fraud.

~~~
beavisthegenius
Their kids should get jobs if they want money.

~~~
jonhendry18
Their kids might be too young.

~~~
beavisthegenius
Too bad. That's the parent's decision.

------
DennisP
Last year I read a book by one of the top oncologists in the U.S., who's been
in the field for five decades and held senior positions at leading
institutions. He did not have good things to say about the FDA.

In decades past, researchers could come up with ideas and use them on patients
who had no other options, and often saved lives that way. The field advanced
quickly. Now progress has slowed and clinical practice lags well behind the
state of the art in research, largely due to FDA restrictions.

So I suspect the book's author is happy about this law. Here's the book:
[https://www.amazon.com/Death-Cancer-Pioneering-Oncologist-
Wi...](https://www.amazon.com/Death-Cancer-Pioneering-Oncologist-
Winnable/dp/0374536481)

~~~
xevb3k
> BrainStorm CEO Chaim Lebovits told Bloomberg he’s considering charging
> $300,000 for each NurOwn treatment

The problem isn’t that they get to try an unproven treatment to see if it
works. The problem is that they get to charge desperate people huge amounts of
money for it.

Do it for free, as a way to validate a drug and advance research and this
wouldn’t be a problem (or at least it’d be a completely different problem).

~~~
DennisP
I don't disagree; a better middle ground might be cost plus some reasonable
percentage for unapproved treatments.

~~~
filoeleven
Isn’t that akin to making people pay to participate in a study? Shouldn’t
their payment consist of all the information a company receives from
essentially testing their new treatment on a human?

~~~
danielnesbitt
They are not participating in the study though, at least not the regulated
portion of the trial.

The rule prevents negative outcomes for risky non-qualified patients from
harming the trial, but the converse is also true. Positive outcomes for
patients outside the regulated trial will not help gain approval at Phase II
or Phase III.

On a board like hn, there's a tendency to look at all data as useful data, but
this isn't true in the case of a clinical trial.

On further thinking, I'm not even sure what happens in the case of a patient
receiving a pre-approval drug outside of the trial. Handling their data would
likely need to meet the same standards required in trial (consent tracking,
electronic signatures, etc) without any of the machinery of the trial to help.
It could well be the case that nothing is collected if the cost to keep it and
use it are too high.

~~~
mbreese
_On further thinking, I 'm not even sure what happens in the case of a patient
receiving a pre-approval drug outside of the trial. Handling their data would
likely need to meet the same standards required in trial (consent tracking,
electronic signatures, etc) without any of the machinery of the trial to help.
It could well be the case that nothing is collected if the cost to keep it and
use it are too high._

IIRC, some of this was handled by FDA (or could have been), if they were part
of the process by granting an unapproved treatment waiver. This way some
amount of 'informal' data could be collected and used to guide others, even if
the treatment was outside of a formal trial. Now, without FDA having a role, I
fear that none of this data will be captured (or at least not in a useable
form).

------
jedberg
A lot of folks commenting in here should look up Thalidomide.

Approved by most of the agencies in Europe, but denied by the FDA, it turned
out it was really really bad.

So yes, the FDA does slow things down, and yes, people have a right to try
what they want, but just keep in mind that the FDA does serve a purpose.

[0]
[https://en.wikipedia.org/wiki/Thalidomide#Birth_defects_cris...](https://en.wikipedia.org/wiki/Thalidomide#Birth_defects_crisis)

~~~
tsomctl
According to your link, Thalidomide was introduced in 1957, and banned in
Canada and most European countries by the early 60's. I really, really hope
that the FDA and EMA have improved their methodology in the past 50 years.

------
dahart
> “We have to have an incentive,” he said.

There already is an enormous incentive for therapies that work. Whether you
agree or not, you kinda have to admire the leaps of logic that allow people to
rationalize needing to make huge profits on unproven trials from day 1, for an
industry that has some of the highest profit margins of any.

~~~
emiliobumachar
"for an industry that has some of the highest profit margins of any"

Big Pharma profitability generally is pretty good but not stellar, unless you
only look at profitable drugs and ignore unprofitable drugs.

~~~
XorNot
The 50% on marketing number isn't BS:
[https://www.vox.com/2015/2/11/8018691/big-pharma-research-
ad...](https://www.vox.com/2015/2/11/8018691/big-pharma-research-advertising)

Everytime I've gone looking for this, it's just been confirmed over and over.

~~~
hackerman12345
It's not just marketing in that graph, it's sales & marketing. Insurance
companies don't make getting drugs covered and to patients easy. Newly created
drugs are pretty useless if neither doctor nor patient has heard of them.

~~~
rhizome
_Insurance companies don 't make getting drugs covered and to patients easy.
Newly created drugs are pretty useless if neither doctor nor patient has heard
of them._

Ergo hookers and blow? I don't buy it.

------
maym86
Read bad pharma by Been Goldacre. It's a detailed outline of many issues with
the industry. The extent that pharmaceutical companies go to make profit,
mislead doctors and harm patients is crazy. This law will definitely be abused
for profit.

[https://en.m.wikipedia.org/wiki/Bad_Pharma](https://en.m.wikipedia.org/wiki/Bad_Pharma)

------
raverbashing
Let me rewrite the headline: "Redtape fetishists outraged that people now have
a choice"

 _Every therapy was unproven at a certain point in time_. It can only become
proven with time and tests.

Now, I agree that an experimental drug should be sold at a discounted price,
or even free. We can totally discuss that.

But blocking the last possibility of hope of a dying person is just being
petty.

~~~
hexane360
The article is about "right-to-try profiteers", not just "right-to-try"
period.

>We can totally discuss that.

That's what this article is trying to discuss.

------
peacetreefrog
It's "right-to-try" not "have-to-try". Articles like this make it sound like
patients are powerless and have to take and pay for whatever treatments these
companies offer.

~~~
mrguyorama
A dying and constantly miserable human is not a rational actor

~~~
openasocket
Especially when the drug seller and the patient have asymmetrical information.
As far as I can tell, there are no requirements for the drug maker to report
the effectiveness of the drug on those that they sell it to.

------
WC3w6pXxgGd
And this is bad? The healthcare industry desperately needs experimentation and
cheaper options. If a product works, people will continue to buy it. Welcome
to how markets work.

~~~
freedomben
I agree, but there's an even more compelling argument in my opinion.
Government (which is basically an abstraction for "another person that isn't
me") has no right to tell me what I can and can't put into my own body.

~~~
hexane360
Unless those other people are directly entangled with your finances and
responsible for ensuring the wellbeing of you and others.

------
ada1981
I’m curious how this law might be leveraged for PTSD and other mental health
folks access to MDMA and other psychedelics.

Would this allow for access to scheduled substances?

Could this law be useful in that regard?

~~~
narag
Only if they can charge $300,000 for the privilege. Anyway it seems that the
drug must have passed a first phase of testing. Are illegal drugs allowed to
be tested to begin with? I don't think so.

Most disgusting news I've read this year.

~~~
ada1981
MDMA is in Phase III clinical trials for PTSD and showing very promising
results. It was given a special designation by the FDA as a breakthrough
treatment to speed up the process.

Previous trials with the VA found 86% of soldiers with PTSD no longer had it
after a couple sessions.

So, in this case illegal drugs are. It’s the only way they would ever get
unscheduled, as schedule 1 means no known medical use.

~~~
narag
That's interesting, but wasn't MDMA a prescription drug _before_ it became
popular as a recreational drug? I mean, not the only drug that's legal if
prescribed by a doctor and illegal otherwise, like morphine etc.

~~~
ada1981
MDMA was not a prescription, it just wasn’t a scheduled compound. It was
originally used by therapists however before it spread into the mainstream and
then it was scheduled despite the recommendation that it be scheduled to allow
for medical use.

There was a study cited where they accidentally used Meth vs MDMA as evidence
to schedule it and then after it came out the study made a mistake, the
legislation still stood.

But keep in mind that most other drugs were used medicinally before they were
scheduled.

Cocaine, Pot, LSD, Mushrooms, etc were all legal before they were illegal.

The war on drugs is a relatively new obsession.

------
anm89
Those gosh darn profiteers providing optional life saving therapies for
patients. Drat them!

I'm not saying the FDA has no purpose but it would be really hard to convince
me that giving additional options to people with terminal illnesses is a bad
thing.

------
ekianjo
> The idea that BrainStorm could make a profit from NurOwn before the
> treatment is proven effective and approved by the FDA is a bad look for the
> company and the entire pharmaceutical industry. It reeks of opportunism,
> even when couched in compassionate rhetoric.

If anything we need more experiments like that. If you have no treatments left
to try and you are dying, being able to try something that is not 100% proven
but that has some potential is better than nothing. It's not like selling
snake oil or something, most therapies have clear identified pathways and
targets based on actual fundamental research.

Companies are hardly going to make tons of money on unapproved therapies
anyway, because the word will get out if x patients tried it and had no
benefits.

~~~
Kuiper
The author of the article also seems to imply that FDA approval is the only
process by which a treatment could be "proven effective." There are many
treatments that are already available in Europe but spend years waiting for
FDA approval.

There's also the fact that even when a drug _does_ successfully pass through
the FDA testing process, there can be a period of up to a year between the
drug being successful in Phase 3 trials and actually getting approved for
prescription. If you're a terminally ill patient, a 12 month waiting period
between "proven in phase 3 trial" and "actually finally getting the FDA's
rubber stamp" can be a long time.

~~~
XorNot
The FDA allows "compassionate use" exceptions for this type of thing and
generally grants them. The point of the approval process (and the entire FDA)
is to prevent charlatans peddling dangerous and ineffective therapies and
drugs to desperate people for profit.

Which is what this article is about: you can have no choices, but here, pay us
a ton of money for UnprovenDrug(TM)! It might work according to nobody but us.

You're making the classic misstep of assuming a Pascal's wager type situation
- as though there's only ever 1 unproven option out there so you might as
well, when in reality there's many and getting scammed out of all your money
on one means you can't try others which actually might work.

------
RickJWagner
An expensive chance is probably better than no chance at all.

------
frgtpsswrdlame
>BrainStorm CEO Chaim Lebovits told Bloomberg he’s considering charging
$300,000 for each NurOwn treatment offered through a new pathway set up as a
result of the right-to-try law.

Drug manufacturers have finally raced to the bottom. The explicit purpose of
charging high costs for experimental drugs is to entirely empty the bank
accounts of the most desperate people without even having to tell them a
potential rate of success. This is sick and preys on people in the most
desperate situation one is likely to face in life.

------
briandear
Could the lede be any more biased or willfully ignorant as to how long FDA
processes take? A terminally ill patient doesn’t have much need for money if
they are dead, so what business is it of anyone’s if they choose to spend all
of their money of a last ditch hope.

People waste billions on quack medicine such as Traditional Chinese Medicine
but now we are concerned about the finances of dying people? And yes, TCM is
quackery; any form of “medicine” that prescribes black rhino horn as a cure
for impotence is pure nonsense. Is statnews writing strongly worded opinions
about that? Or are they just interested in picking on big pharma— that,
despite its warts, actually does make drugs that have benefited society in
countless ways.

