
A million-dollar drug - rpledge
https://newsinteractives.cbc.ca/longform/glybera
======
tptacek
People are fixated on the price for this drug, but the article itself is
pretty clear on the rationale for the price tag: current alternative therapies
have a price tag of up to $300k/yr, indefinitely. This drug is a one-time
therapy. Presumably, any health system would jump at the chance to replace a
$300k/yr recurring charge with a 1-time $1MM fee.

But they don't. Health systems in Europe apparently refuse to pay for this
therapy. The reason for that, from reading other articles, appears to be that
it is of questionable efficacy. Patients report fewer pancreatitis attacks,
but clinical indicators like blood fat levels are apparently unchanged. The
tiny market and efficacy concerns might explain why the therapy is no longer
available at any price.

~~~
refurb
Actually, single payers in the EU are much better equipped to pay $1M now to
save $300k/yr in perpetuity. All medical spending comes from one big pile.

In the US, not people don’t stay with the same health plan for more than 2-3
years. So why would an insurance company pay $1M when they get 2-3 years of
pay back, then the next insurer gets a free ride?

I know there is an MIT prof who is pushing the idea of something like a bond.
Some external party pays the $1M and the insurer makes X payments until it’s
paid off.

A interesting idea that even the single payers like as they have a more
predictable budget.

~~~
tptacek
Yeah, I'm not saying it doesn't make sense or that the EU wouldn't do it. I'm
saying they _aren 't_ doing it, because the treatment is of unclear efficacy.

~~~
fyolnish
But that doesn’t have much to do with the price. Your comment sounded like you
were saying the eu was being cheap.

~~~
jstanley
I thought it sounded like quite the opposite: he's not saying they aren't
buying it because they're cheap (he points out that if it worked, and they
were trying to be cheap, they _would_ buy it). He's saying they're not buying
it because it might not work very well.

------
iamleppert
I was recently one of the first recipients of gene therapy for my condition. I
have Hemophilia A, which is a bleeding disorder that results in bleeding into
joints, muscle and soft tissue due to lack of a naturally occurring clotting
factor that my body can't produce due to a defective gene. I received my dose
of 30 billion viral particles about 13 weeks ago as part of a phase II
clinical trial at UCSF.

It is amazing to me that we can manufacture and program viral particles to
target specific cells. In my case, my treatment is a non-nucleonic technique
that didn't repair the actual chromosomal hereditary defect but inserted a
working gene, additional and freely available genetic material into my liver
cells. It's amazing that the body can just recognize this extra bit of code
and just start producing the factor. The engineer in me is also impressed they
can make so many copies of the engineered virus.

It appears to be working. My factor levels have steadily increased and I’m now
no longer a severe or even moderate hemophiliac. I’m their first patient to
have achieved these results for hemophilia A severe and it puts me well into
the therapeutic range. It really feels like I have been given a new body, it’s
indescribable. For the first time in my life, I’m pain free.

It’s a weird feeling. Pain, mostly from bleeds into my joints, has been my
constant companion. I was never able to do sports as a kid and had to avoid
many other activities that would put me at risk. I have mixed feelings about
all of this. On one hand I’m extraordinarily grateful, and the other side I
see what a huge disadvantage I’ve been at compared to others for all of my
life. I thought this day would never come, I had resigned myself to my
limitations but now they are gone in a matter of weeks through the miracle of
a medical experiment.

My old medication, a replacement clotting factor that was synthetically
produced in a lab, worked but not nearly as well as this. Not to mention the
fact it cost over $100,000 per month and I had to give myself an IV every
other day. It's truly been a life changing experience for me, and I hope that
gene therapy becomes widely available to others.

Also, the thing that was a milestone in this case was that the Hemophilia A
factor VIII gene has been notoriously difficult to create a genetic therapy
for because of the complexity of the actual gene itself. On the scale of what
the body produces, its one of the more complicated proteins. So it basically
means many other genetic conditions will be able to be treated with and
addressed with similar technology.

Such an exciting time to be alive, and yes there are amazing things happening
right now despite all the bad news you may hear!

~~~
MRD85
Thank you for sharing. My first undergrad studies were a double major in
Science (Chemistry/Biology) 12 years ago and yet I had zero clue that we were
at the stage currently to do what you described. To hear what you've just
described is absolute music to my ears and it makes me very happy. While you
may have lived a number of years dealing with your issues it means that we are
likely approaching a time when children will be able to be cured early and
live pain-free lives.

~~~
iamleppert
You’re welcome and yes I agree about this being most impactful for children.
Hemophilia had a big effect on my life, both physically and psychologically,
as well as my family. It’s hard to grow up knowing you’re so different from
others. Now, someone born with hemophilia may never even have to know. It
changes things dramatically.

In a single generation we’ve gone from being able to treat the condition at
all (replacement factor was just becoming available when I was born) to being
able to completely cure it.

~~~
DoctorOetker
a next step would be for the virus to fix the cells from which the gametes
derive, then your future offspring would be cured as well

------
apo
_Glybera was never sold in North America and was available in Europe for just
two years, beginning in 2015_

The patent will expire within a few years, setting the stage for generic
manufacturers to step in, but the article doesn't mention it.

In the US, patent protection extends for 20 years after the earliest filing
date for the application on which the grant is based. There can be adjustments
to the term if the grant was delayed.

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

The article is fuzzy about the timeline of events, bu has this quote:

 _The stunning results of the mouse experiments were featured on the cover of
the journal Human Gene Therapy in September 2004. The cover image showed how,
week by week, the mouse blood changed from milky white to a clear, transparent
red, illustrating just how effectively the gene therapy was working._

So it seems like a good first guess to put the patent filing at or before
2004.

If so, then the Glybera patent would expire sometime after 2024. At that
point, generic manufacturers can step in to sell their version of the product,
assuming regulatory approval.

~~~
refurb
As you can see from the lack of biosimilars in the US, manufacturing a
biological is a hell of a lot more complex and expensive than doing it for a
small molecule.

And in fact, since this is version 1.0, I’ll bet a better version comes out
before any generic company launches.

~~~
akvadrako
If they come out with a new version, they must be expecting it to sell in a
quantity that makes the investment worth it. So maybe the pricing worry is
just a temporary concern.

------
apsec112
This seems bizarre to me:

 _Van Deventer says the company never considered lowering the price. "Why
would we? Pricing shouldn't be a political decision. It should be a rational
decision based on merits and value."_

If no one will pay $1 million, your revenue is $0. Assuming the drug costs
much less than $1 million to make, surely any revenue number is better than
$0?

~~~
wpietri
I don't think he understands pricing at all. Market price isn't arrived at via
a rational process. It's an inherently social process arrived at by
negotiation between producers and consumers.

And it's especially weird that he thinks pricing shouldn't be political when
his product has a government-granted monopoly and the bulk of its customers
will be government-run health-care systems and government-licensed insurers.

Sure, there's a real question of how to pay for treatments for rare diseases.
But the millions in development are a sunk cost. To take his ball and bat and
stomp off would be disappointing in any circumstances. But when people will
die because of it?

~~~
quotemstr
> I don't think he understands pricing at all. Market price isn't arrived at
> via a rational process. It's an inherently social process arrived at by
> negotiation between producers and consumers.

Are you suggesting that prices don't have anything to do with scarcity and are
just set arbitrarily by social forces? That doesn't seem quite right.

~~~
maxander
Well, yes, that’s how supply and demand (supposedly) works. The “correct”
price is where the demand, at that price, equals the available supply.

If the product is scarce, that can drive up the price, but only as far as
demand is willing to follow- there’s no _inherent_ value in being scarce.
“Price” is a social thing, so of course it’s determibed by social forces.

~~~
yholio
To illustrate your point: if I paint an unique painting of myself sitting on
the toilet, that's quite a scarce item, there is only one in the world, yet,
it's utterly worthless.

Purely social forces somehow came to the (in my opinion, debatable) conclusion
that ladies with square faces painted by Picasso are art, while a
representation of defecation by me is not.

------
Mvandenbergh
NICE, which makes value for money decisions for the NHS, uses a threshold of
£25k per Quality Adjusted Life Year for assessing cost effectiveness of
treatments.

At that threshold, to pay for an £800k drug treatment you'd need to give
someone 32 extra years of life (or 64 years at double the quality of life and
so on). This is enough to pay that kind of amount for life saving gene
therapies, especially if given to young children but not enough to treat
something that can be largely managed through diet control.

Since the majority of the world's patients are in Quebec, I don't understand
why the provincial government doesn't cut a deal for this drug. I'm sure
they'd rather sell it $250k a dose to a nice big patient population than at
$1m to nobody. Quebec has a lot of pricing power here as the only large
potential buyer.

~~~
xxpor
The fact that women can't have children without the drug, but apparently can
with it adds a new complication to the QALY calculation I think.

------
mabbo
The point of the patent system is that we are giving inventors a time-limited
monopoly on their invention in exchange for them sharing it with the world. We
all benefit because we have access to this new invention and soon can make it
ourselves, and the inventor has an incentive to promote and sell as much as
they can while they own the monopoly.

But it's not working here. The patent holder is effectively saying society
isn't allowed to have the invention. They're using a legal means meant to
share the knowledge with the world to instead horde it away from the rest of
us.

When the system doesn't work, the system must be improved. How can we
incentivize inventors to not do this?

~~~
rayiner
It's bad to analyze this issue through the medium of a long-form article,
which wraps the story up in emotional narrative. Here is a better write-up in
the MIT Technology review: [https://www.technologyreview.com/s/601165/the-
worlds-most-ex...](https://www.technologyreview.com/s/601165/the-worlds-most-
expensive-medicine-is-a-bust).

The gist of the problem is this:

> The Amsterdam company spent more than $100 million testing the drug and
> carving a path through Europe’s medical rules and regulations, which weren’t
> geared to consider a new technology like gene therapy. Initially, for
> instance, regulators said they expected a clinical trial of 342 patients.
> Executives wryly noted that there were _only 250 people_ with the disease in
> all of Europe.

> Nor has Glybera convinced the national regulators in Europe who decide what
> drugs get reimbursed. Last year, French authorities said they would not pay
> for the drug. Germany judged Glybera’s benefits “non-quantifiable.” It
> leaves doctors and insurers to make decisions on a case-by-case basis.

Here's a drug that cost $100 million to make, and will require even more money
to move through clinical trials in the U.S. And the pool of potential patients
is a less than 1,000 people, and oh many of then can't pay either because they
lack insurance (U.S.) or the cost/benefit panel math doesn't work out
(Europe).

Viewing this after-the-fact leads one astray, into thinking there is some
problem with the system that leaves this potential cure out of the hands of
patients. Patents, money, it's all just a proxy for effort in, benefit out.
Even if you had the government make this drug, you're talking huge amounts of
money per patient, which few governments would pony up. (And rightfully so,
because you could save a lot more lives with that amount of tax dollars.) The
answer is probably that this drug doesn't make economic sense, and the
abandonment of it by the maker is just an indication that it was a misguided
effort to begin with.

~~~
derefr
The drug didn't cost $100 million to make. It cost $100 million to _prove that
it works in order to get it approved for sale in Europe_.

In a world where you could just put a drug on the market (where "the market"
is, say, hospitals), the drug would have cost far less, and so the inventor
would need to recoup far less.

Note that I'm not arguing for complete deregulation of drug manufacturing.
Testing for _product integrity_ through factory audits and the like is the
original—and still core—purpose of bodies like the FDA and its EU
equivalent(s). You want to know that your "ibuprofen, 20mg" indeed contains
20mg of ibuprofen in each pill, and nothing else.

But that's cheap! That doesn't cost a hundred million dollars to do. Proving
product integrity is something that any _food processing plant_ manages on the
regular, not to mention manufacturers of generic/non-patented drugs.

If we stop with the need to prove—with overwhelming statistical power—the
safety-and-efficacy of a molecule that we're planning to dispense to fewer
than 1000 people (and even then only through doctors who can carefully monitor
them for adverse reactions), the pharma industry would go back to being just
another quiet industry that makes stuff, instead of the crazy behemoth it is.

Yes, certainly, there should be a stringent process before you stick something
on a store shelf for OTC purchase. But most drugs never seek to be OTC anyway.
Prescription drugs require an interaction with a prescribing doctor, a
dispensing pharmacist, and possibly (in in-patient settings) a bunch of
watchful nurses. Why, also, must they require an interaction with a standards
body regulating the molecule they're composed of? The patients who were in the
clinical trial itself got effectively the same treatment, minus the standards
body, and they were all fine.

Maybe that's what I'm saying: why not just allow any random chemical to be
given to people (without intention of standards-body registration) as long as
the prescribing doctor is willing to do clinical-trial-like monitoring for
adverse side-effects?

~~~
panzagl
So maybe it should be more like the UL model? Let the insurance companies fund
the proof of safety/efficacy, but it's not mandatory.

~~~
howard941
If by UL you mean Underwriters Laboratories in the US or the other US NRTLs,
compliance testing costs are borne by manufacturers, not insurance companies.

------
GeekyBear
Getting a drug approved for use is an expensive proposition, but that doesn't
mean that it is impossible for a nonprofit organization to step in and get the
job done when the target treatment group is too small or too poor to make it
worthwhile for those with a profit motive.

The Drugs for Neglected Diseases Initiative, a nonprofit which grew out of
Doctors Without Borders, just received approval for a pill that successfully
treats African Sleeping Sickness.

>Ultimately, the drive for approval cost $63 million and involved clinical
trials including 750 patients in Congo and the Central African Republic. Two
million villagers were screened.

The costs were paid by seven European countries, the Bill and Melinda Gates
Foundation, Doctors Without Borders and other donors.

[https://www.nytimes.com/2018/11/16/health/sleeping-
sickness-...](https://www.nytimes.com/2018/11/16/health/sleeping-sickness-
africa-cure.html)

------
gfodor
in society, we have another large, one-time purchase people often make in
which there are special financial arrangements and rules: buying a home. it
seems to me that gene therapies (high priced, one-time use drugs) are going to
warrant alternative financial instruments. there's absolutely no reason this
drug should have failed when the amortized cost over 10 years is less than an
inferior product. regardless of the other questions this is a pure market
failure by the financial industry.

edit: the downvotes probably assume I mean the patient is going to pay out of
pocket. of course not. the insurance companies would be paying. the only
inferiority between gene therapies and non-cures is their pricing structure.
we invented a solution to this problem thousands of years ago: debt.

~~~
wrong_variable
The problem is with a house / car / TV / phone you can always reposes it.

~~~
gfodor
It just backs out to interest rates commensurate with the risk. Lack of
collateral doesn't mean you can't get a loan.

Perhaps the economics wouldn't work, but it sure sounds like nobody tried in
this case. What it would boil down to is if the insurance company expected a
larger net cost on a traditional drug regimen for the life of the patient, vs
servicing a loan at the market rate over N years. Interest rates are still
very low so in general debt is seeking out risk.

~~~
ThrustVectoring
Insurance companies don't cover total health costs over the life of the
patient. They cover the next month of health costs, and a certain percentage
of their customers churn afterwards. For insurance companies, providing
treatment that lasts a longer amount of time can often be a terrible business
decision - if the customer switches insurance providers afterwards, then
they're essentially subsidizing the insurance company they switched to.

This more commonly happens with prescription quantities. Medication compliance
is far better when people can pick up a three-month supply of medication.
Insurance companies want patients to pick up one month supply instead, so that
they aren't paying for two months of medication for every patient who switches
insurance companies.

~~~
gfodor
Right, so what is necessary is for Gene therapy to have similar financial
characteristics to an insurance company as a monthly perscription for a
chronic illness. There is _no_ reason this cant be securitized properly,
unless a) the lifetime healthcare costs for a person are higher with the
therapy than without (unlikely for most chronic illnesses) or b) the interest
rate would need to be so high as to make it so. If gene therapy provides a
more optimal use of resources and capital than continual therapy for chronic
illness than the up-front high price tag nature of it being a problem is an
opportunity for someone to come along and make the market more efficient.

------
umanwizard
> "Why would we? Pricing shouldn't be a political decision. It should be a
> rational decision based on merits and values," he said. "Hundreds of
> millions of investor money has gone into the company, and if there is no
> return for those investments, there will be no new drugs because nobody's
> going to do that in the future, right?"

As terrible as it sounds, this logic seems sound to me. If we think having
this type of drug is worth the > $100MM it took to develop, then health
systems should either fund the $100MM research, or they should let private
companies do it and pay them fairly for their work. $1MM for something so
expensive to develop with so few possible users doesn't seem crazy to me.

If the $100MM is in fact _not_ worth it, then, well, you get the situation we
have now.

I'm sure a lot of people will read this article and think the owners/funders
of the drugs are the bad guys. But nobody can be expected to work for free.

~~~
eloff
Yeah, people just look at the price and think that's terrible, but it's a
business not a charity. It's up to the government to step up if people really
deem this to be a problem worth solving. With their tax dollars. I bet a lot
of critics would go quiet at the point they actually have to pony up.

------
charlesism
"Well, one pharma company has the patent, and we need to let them charge what
they think the market will bear. Sorry LPLD carriers, you have to die now."
Apparently, this is the best the world can manage in 2018.

~~~
lisper
The last sentence of the article sums it up pretty well:

"If it's not commercially viable to produce a certain therapy, unfortunately,
in our Western society, it does not happen."

And it is far from clear that this is not as it should be, because it's not
just LPLD carriers about whom tear-terking stories can be told. $1M can be
deployed in other ways that could potentially save more lives. Instead of
"Sorry LPLD carriers, you have to die now", it might be, "Sorry, 100 poor
African children without access to clean water, you have to continue to die of
dysentery so we can save this one LPLD carrier."

~~~
mirkules
You can play this moral dilemma forever. If the one LPLD patient happens to be
an expert in 3rd world diseases, then his death would lead to an even bigger
tragedy.

In reality, there is no dilemma. The people who _can_ help are morally
obligated to help in non-theoretical situations.

If a child is drowning, and by jumping in I have to ruin my $5,000 watch which
I would have otherwise later sold to help 100 children, my moral obligation is
still with the immediate need of this drowning child.

There is a very good podcast about this very issue:
[https://itunes.apple.com/us/podcast/philosophy-
bites/id25704...](https://itunes.apple.com/us/podcast/philosophy-
bites/id257042117?mt=2&i=1000407998935)

~~~
lisper
> You can play this moral dilemma forever.

Yes, of course. That's the whole point. It's a very hard problem.

> In reality, there is no dilemma. The people who can help are morally
> obligated to help in non-theoretical situations.

Really? How much money have you donated to help provide clean drinking water
to poor children in third world countries?

> If a child is drowning, and by jumping in I have to ruin my $5,000 watch
> which I would have otherwise later sold to help 100 children, my moral
> obligation is still with the immediate need of this drowning child.

Really? You would sacrifice 100 to save 1? That's a very peculiar moral
calculus you have there.

~~~
ddingus
Seriously?

Tell me how a person watches a child drown.

Sorry fam, next week I plan on donating to save 100 kids living in worse
places than your dead kid lived in...

Actually facing a scenario like that, and the people involved plays out very
differently.

~~~
umanwizard
There is no argument here other than an appeal to emotion.

Yes, it's true that no normal person would actually avoid jumping into a pool
to save a drowning child, even at the cost of $5000 that could save 100 other
people. That is the entire point -- human moral faculties are not based on
logic, and overweight the interests of people you can see, or whom you can
help in obvious, concrete ways, relative to diffuse total global happiness.
But the fact that humans are wired to think in a particular way doesn't prove
that it's rational or correct.

~~~
ddingus
News flash!

Emotion, character and reason are all valid in this discussion.

Let me put it to you just a bit more directly:

An aggrieved mother and father are there, facing you and their dead child.

Now, having made this call to let their kid die, what do you say to them?

I will wait...

Actually, I won't. It is one thing to speak of optimal choices when one is
seperated from, or above, disassociated.

Real life, where the actual humans are plays out much differently.

Above, when I asked, "Seriously?" it was this I was getting at.

The claim of strange morals, based on some 100 to one, as if!

I found the whole thing deeply disturbing. Not a negative to the participants.
Not my intent.

No, what I found disturbing was the detached nature of the whole thing.

Maybe Liberal Arts education remains more relevant than I realize.

Again, not a negative toward anyone. Context matters, that's all.

I very seriously question the general wisdom in having some percentage of us
so far isolated from people overall, and any sort of meaningful policy, moral
debate being beneficial.

You completely failed to make a meaningful rebuttal. Correct... ?

Like I said, seriously?

~~~
umanwizard
> Now, having made this call to let their kid die, what do you say to them?

This wouldn't happen, since I'm a (non-sociopath) human.

You seem to have missed the point of the argument completely.

> Real life, where the actual humans are plays out much differently.

Nobody disagrees here.

~~~
ddingus
The person questioning morality did.

And that is OK. Let me be clear. It was thought provoking!

------
hummingurban
To me, this is akin to a game company raising the price of a game. The costs
are already sunk. The only thing that counts is setting a price on copies of
that game. Those who pirate copies of the game incur to loss of revenue
because those people would've never been able to pay for it. It becomes a
question of projecting the maker's morals onto those who did not pitch to the
coffer instead enjoying the game like a dirty thief. This is crazy, the only
loss to the manufacturer is the uncomfortable idea people are enjoying or
benefiting from your creation for free. Yet there are developers who happily
contribute to open source projects and games with little to no recognition or
reward.

Likewise with drug manufacturing, it's the same idea. It's illegal to copy a
pharmaceutical drug and take it yourself and others without the approval of
the patent holder. Yet, it is the patent holding party that determines how
many licensed copies it is willing to sell. Thus, they are able to take the
moral high ground in pricing this drug, but it's the same principle as above-
pony up for my idea, shame on you for benefitting from it and not contributing
to my wealth.

I really do think this company is out of touch with reality. All in all, I
feel like this only earns them bad press, especially after Martin Shrekli
debacle. There's also a potential for a lawsuit, based on human rights I'm
sure.

------
DoctorOetker
>"You need to maintain the factory, you need to do the paperwork, you need to
test the product, you need to make new product batches all the time because
product expires," he said.

I'm pretty confident LPLD sufferers around the world would be prepared to move
to the LPLD hot spot in Quebec, and work part time in the factory under proper
supervision?

~~~
gus_massa
You can't put a bunch of untrained people in a drug factory and expect them to
make a good job. You need some people with a Chemistry/Biology/Pharmaceutical
degree, or some kind of specialization, or many years working in the industry.

Specially if it is a cutting edge factory with a technology that is being
invented now and changes every month. Whatever that can be automated so anyone
can make it without knowledge is probably automated.

It's like thinking that the Supreme court can fire all the technical staff,
and the judges themselves can administer the web and mail server (perhaps with
a little of help from some experts that come only once in a while).

------
cletus
So I was expecting another story about some IP troll buying the rights to a
drug with a monopoly and then jacking up the price (like the whole EpiPen
fiasco) but this wasn't that.

There's an old Chris Rock bit about Big Pharma where he says there'll never be
another cure for HIV like there was for polio because there's no money in
that. The money is in getting you to the next stop.

It's poignant because this isn't a theoretical scenario. Gilead recently was
downgraded on declining revenues because they're wiping out the disease
(Hepatitis C IIRC?).

So, back to Glybera. There are plenty of low-incidence diseases that are
treated by expensive drugs to manage them. This is a lifelong commitment.
Covering such drugs in company health plans can significantly increase the
per-member costs.

If you have a drug that essentially cures the drug in one dose shouldn't that
be weighed against the lifelong cost of covering a regime to manage the
disease that is inferior? Multiply that by the disease being quite rare and
sure, you end up with a $1 million price tag.

As further evidence for how screwed up the US health insurance system is:
companies enroll in plans for their members typically for a period of a year
or maybe a few years. Let's say your drug's price of $1m compares favourably
to $100k/year for 40 years to manage the disease. How can a company who might
only be covering the employees for 1-3 years be expected to cover that higher
cost?

To be clear, this is further evidence of how stupid the US model is. In a
single payer model this particular concern goes away.

How many rare genetic disorders are out there where $1 million per patient for
an essentially complete cure isn't a bargain compared to the cost of managing
the disease? Probably a lot. Is it fair to decry such expensive drugs just on
their price tag without looking at the facts? Probably not but I bet you it
will happen.

~~~
ken
> Let's say your drug's price of $1m compares favourably to $100k/year for 40
> years to manage the disease. How can a company who might only be covering
> the employees for 1-3 years be expected to cover that higher cost?

Yes, it's essentially a Prisoner's Dilemma. If every insurance company agreed
to pay for this drug, the total cost of treatment overall would be lower (and
the patients would be healthier and happier). If my company decides to pay it
and no other insurance company does, then when the patient leaves our network
(in less than 10 years) and joins another insurance network, my company will
have paid the cost but can't reap the financial benefits.

There's two major differences between this scenario and classic PD, though.

First, companies are allowed to communicate. We could conceivably get
everybody in a room together and come up with some agreement by which
everybody agrees to pay for this drug, even if patients switch insurers.
Everybody wins. Patients are healthier, and insurers save money.

Second, it's not a one-time decision. An insurer can change their mind from
"no" to "yes" at any time. So it's more like "iterated PD", with an indefinite
number of rounds. In that game, interestingly, there is no strictly dominant
strategy, and _altruism_ tends to do better in practice! So maybe you don't
even need an agreement.

> To be clear, this is further evidence of how stupid the US model is. In a
> single payer model this particular concern goes away.

How do you figure? The article points out that they also had trouble
convincing European governments to pay for it. You can't get Glybera anywhere
in Europe or Canada today, either.

The US model is arguably a poor one, but this isn't a good example of that,
because every other country in the world failed at Glybera, too.

------
cubano
This article brings up a thought experiment I've been pondering for a good
while now...

So what would you charge for a drug that guaranteed you would live 30 extra
years after you spent 20 million to develop it?

For an added twist, let's say it's NOT FDA approved but you have solid
clinical evidence that it's efficacy was solid and can your "street cred"
(whatever that means in biotech) allows you access to the world's most
successful (ie richest) people.

I have my answer...what's yours?

~~~
glass_of_water
That's a really interesting thought experiment, and I don't really know what
my answer is.

The following choices assume that more people living an extra 30 years is a
good thing ethically, and that there are no devastating effects
environmentally or otherwise. The following choices also assume that you'll
always have a monopoly on the production of the drug.

1\. Assuming that the supply is practically unlimited, an altruistic actor
should probably give it away for as close to free as possible. A purely
selfish actor should probably try to sell to each individual for as much as
they can afford (though this can be hard pricing strategy to enact). I suppose
you might actually want to charge less than that even as a purely selfish
actor, since you wouldn't want to somehow destroy the world economy by making
everyone really poor except for yourself (would this actually destroy the
economy though? I'm not sure... probably not relevant for the purposes of this
thought experiment).

2\. Assuming supply is limited, a purely altruistic actor will have to choose
the most fitting recipients. This is a really tough choice. How do you define
the most fitting recipients? Those who are most likely to contribute to the
betterment of humanity with their extra 30 years? It seems like an almost
impossible task to pick out who these people would be. A purely selfish actor
should charge the richest as much as they're willing to pay.

If it were me and supply were unlimited, I'd like to think that I'd sell it
for as little as possible but enough to still never have to worry about money
again. Making $10 million in profit would probably be more than enough (though
if you can make more and still help everyone, that'd be even better). If
supply were limited, I don't know what I would do.

One thing I just realized though, is that even if supply were unlimited, if
you had this godly power, you might choose to withhold it those you deem
unworthy, which is a whole other ethical can of worms. I'm guessing this kind
of deviates from the intent of your thought experiment though.

What's your answer?

------
jl2718
Does anybody know which virus vector was used?

~~~
rfinney
_The adeno-associated virus serotype 1 (AAV1) viral vector delivers an intact
copy of the human lipoprotein lipase (LPL) gene to muscle cells._

source:
[https://en.wikipedia.org/wiki/Alipogene_tiparvovec](https://en.wikipedia.org/wiki/Alipogene_tiparvovec)

------
mirimir
> But it's the only way scientific discoveries ever get to patients, because
> universities don't make drugs.

Why not?

If there are so very few patients, a lab could easily produce enough for them.

~~~
ineedasername
Yeah, they had to make some quantity to run their trials. presumably it cost
much less than $1mil/dose or they'd not have research funding at their level

~~~
mirimir
Indeed. A lot less than $1million per dose, I bet.

I understand that there are regulatory issues.

But it seems pretty clear that the standard pharma industry model doesn't work
for drugs like this.

------
dssu
Video for article
[https://www.youtube.com/watch?v=rBSv0deIVzM](https://www.youtube.com/watch?v=rBSv0deIVzM)

------
whydoineedthis
TBH, it sounds like the govt regulation for the drug to even get approved are
what drove the cost up. The product was essentially made before pharma even
had to lift a finger, but they made them jump through a lot of hoops to get it
to market. That's the $100mm they are trying to recoup. Also, I think a lot of
folks are missing that the disease is not necessarily lethal when diet is
controlled. Just saying, that's all.

------
anon2775
If it works but it's not economically-viable: the moral and ethical choice is
to open-source it or give it to a non-profit medical foundation. It sucks to
have a rare disease because there isn't enough profit to recoup r&d; this is
where non-profits, NGOs and GSEs should advance medicine where big pharma
trades lives for profits.

------
ken
Will other scientific or technological advances (like gene therapy drugs for
more common disorders) cause the price of this drug to come down in the
future?

Say, if gene therapy became common, there wouldn't be as much red tape for
approving this particular one, and there might be machines or research that
could be shared with other drugs to amortize the cost?

------
platz
> Pricing shouldn't be a political decision. It should be a rational decision
> based on merits and values," he said.

The money quote. A rational decision? Maybe. Exactly what merits? Exactly
which values?

"Hundreds of millions of investor money has gone into the company"

But none of that led to the development of the drug.

------
ttul
This is what we call “market failure”.

------
timka
That's probably the greatest example of the issue that Open Longevity was
going to solve by combining patient organization with ICO funding to conduct
clinical trials without profit driven big pharma. Unfortunately, that didn't
happen…

------
dev_dull
> _The problem was the price._

I thought that drug patents were basically illegal in India. It seems there’s
a huge market there for this kind of thing. An abandoned drug with a huge
price tag.

Also I’m not terribly worried. The patent will wear off eventually and then
anyone can make it.

------
rbultje
As sad as this story is, the silver lining is that patents expire, and 20
years from the patent filing point (probably 5-10 years from now?), patients
will be able to be affordably cured. It sucks but is better than nothing.

~~~
pierrebai
The story is clear: the problem is not the patent itself but the cost of
passing regulation hurdles and maintaining a plant producing the drug when
there is suck low demand.

No one will invest in this even after the patent expires. The best the
patients could hope is to group togethr to get some done produced?

~~~
whydoineedthis
Regulation hurdles were met, and the cost of a plant is overblown. Most
specialty pharmacy are made in small labs with a few pharmacy techs, and they
can make other things in between this drug. I think the issue is that of
pharma alows the price to drop on this drug, they lose negotiations on other
drugs. They don't want a precedence to be set where they drop thier price if
insurance companies refuse to pay.

------
drannex
The article is fascinating, but the format with the

one sentence

at a time

system is incredibly hard to read and digest

~~~
Raymonf
On the contrary, I find that easier to read! Except for the font, that's just
way too light.

------
ada1981
What’s up with this drug now?

Sounds like it’s patents and IP protection should be up.

I’d imagine you could get a single dose manufactured 1 off for $100k by a lab.

------
DoreenMichele
This is kind of a common issue with genetic disorders. When I was active on
cystic fibrosis lists, sometimes articles were posted that said stuff like "It
costs an average of $100k annually to treat CF" and parents would discuss it
and go "That sounds low to me" and some older patient would chime in and say
"Last year, my drug prescriptions alone were $100k. That's not counting the
cost of doctor's visits and hospitalizations."

(Estimates in articles vary wildly. I searched briefly and just trying to find
something that makes sense for like two minutes just makes me tired. So if you
want to jump up and dispute that with some googled up article, I don't feel
like arguing with you about it. I've already told you actual people living
with the condition read those articles and go "Where on earth are they getting
these ridiculous low ball figures???")

So then they come out with some very expensive drug that costs like a quarter
million or more annually and it only treats around five percent or so of
patients with CF. The price on Wikipedia [1] is currently listed at over $300k
annually.

[1]
[https://en.wikipedia.org/wiki/Ivacaftor](https://en.wikipedia.org/wiki/Ivacaftor)

And then who can afford that? If you have been sick your whole life, you
probably don't have a lot of savings or a lot of ability to come up with big
bucks, etc. You are probably deeply in debt and not making much money.

I don't know the answer. Conventional medicine doesn't really work for people
with genetic disorders. It tends to be crazy expensive and also merely
mitigates things somewhat. The standard expectation is that you will suffer a
bit less and maybe live a bit longer, but you won't ever really be well.

So such people understandably want _a cure._ That's the holy grail for folks
coping with genetic disorders.

I think there are potentially other avenues for some portion of people with
genetic disorders. But I don't currently have the words for that and I get
tired of being ganged up on by boatloads of internet strangers who want to
inform me that I'm imagining things and my entire life experience is a
hallucination and I don't actually know nothing about genetic anything. So
let's just leave it at "I have this opinion and maybe it provides a way out of
this trap and maybe it doesn't."

Because the current approach of inventing incredibly expensive drugs that
might provide a cure is essentially failing as a method. And all other
approaches are so awful it's why such things get labeled "dread diseases" \--
because what it does to your life is so terrible that if a doctor were not
prescribing it, it would be in violation of the Geneva Convention. It's just
not humane.

