The University of California San Diego, Skaggs School of Pharmacy and Pharmaceutical Sciences Drug Commercialization course brings you lectures from both faculty and industry experts. With this course, recorded on campus at UCSD, we seek to share our access to top people in the field who bring an unprecedented range of expertise on drug commercialization. This course will cover pharmacoeconomic, marketing strategy, intellectual property strategy, portfolio management, managed markets and strategic alliances. It will also have a lecture case study from startup to success. In addition, the course will discuss post-marketing clinical trials or Phase 4 trials. These are conducted after a new drug has been approved by the regulatory agencies and launched. In these studies, the new drug is prescribed in an everyday healthcare environment using a much larger group of patients. This enables new treatment uses for the new drug to be developed, comparisons with other treatments for the same indication to be made, and determination of the clinical effectiveness of the new drug in a wider variety of patient types, and more rare side effects, if any, may be detected . Pre-marketing strategy should be instigated as early as Phase 1 clinical trials to ensure that the market's needs are incorporated into the new drug's overall development. Later phases when clinical results are presented at international medical conferences the marketing strategy is then refined in order to develop an awareness amongst the medical community who will be prescribing the new drug. In addition to the marketing strategy, pricing strategy and a tactical plan will be developed. Promotional material, and the sales force will be trained so that when the product is approved they can promote the drug to physician, pharmacist and nurses. This course is intended as part 3 of a series: Drug Discovery (https://www.coursera.org/learn/drug-discovery), Drug Development (https://www.coursera.org/learn/drug-development) and Drug Commercialization. We would highly recommend that you take the courses in order since it will give you a better understanding on how a drug is discovered in the lab before being tested in clinical trials and then launched in the market place.
From Start up to Success in Biotech V
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来自 University of California San Diego 的课程
Drug Commercialization
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从本节课中
From Start up to Success in Biotech, Barry Quart, Pharm.D.
与讲师见面
Williams S. Ettouati, Pharm.D.Director, Industrial Relations & Development; Health Sciences Associate Clinical Professor, N.S.
Skaggs School of Pharmacy and Pharmaceutical Sciences
Joseph D. MaAssociate Professor of Clinical Pharmacy
Skaggs School of Pharmacy and Pharmaceutical Sciences
[BLANK_AUDIO]. So just to kind of conclude, we, based on
the information that we gained in Phase 2, we moved the compound into Phase 3.
It's currently in four, Phase 3 studies that will en-, enroll about 2,000
patients total. and they'll be completed around mid-year
next year. And hopefully the application will be
filed somewhere in that same in, in the same year.
And the product will be on the market, hopefully, a year later.
so it's moving along very nicely in terms of the development process.
And it's, all of it's based on what we've done in Phase 2.
>> So, the patients that are on this medication, is it like statins, where
they could be on it [INAUDIBLE] life long or is it a short, short term?
>> you know there's been studies of kind of intermittent withdrawal.
And what happens is if you get a patient and they're on it for several years,
they're flare free if you stop urea lowering therapy, within six months
they'll start having flares. So, you know, number one, our goal is to
eliminate flares. and two, if the flares are there, that
means the crystals are in the joints. And there's a lot of evidence that
there's joint destruction associated with that.
So, we think both from a commercial point of view and a medical point of view,
patients should stay on it pretty much constantly.
Yes. >> were there any side effects with
that drug? And were there any dose dependent side
effects? >> Actually the interesting thing is
that didn't want to bore you with all the details of the product, but it's very
highly protein bound. So there's very little drug circulating
free in blood. about a third of the drugs excreted
through the kidney, and that's the site of action.
It's actually the urine side in the proximal tubule.
and so the only place you see free drug is at the site of action.
and this, I think that's one of the reasons why we see no dose-related
toxicity. and we actually don't see any side
effects per se. We have, you know, we don't see any GI
toxicity. We don't see headaches.
We really through Phase 2, we did not see any consistent side effects.
Yes? >> Have you started pricing the drug or
do you know like what, what dose and how much it will be?
>> Well, we, you know, we're currently testing in Phase 3.
We're looking at 200 milligrams and 400 milligrams.
It turns out we didn't feel like we even needed to go to 600 milligrams.
even though there was no, no safety issue from going up there or higher.
But we decided that two and 400 was plenty, particularly in combination.
and the, the less you give, the cheaper the cost of goods are.
and obviously in, in the United States, at least as of today, there's a certain
amount of pricing flexibility that companies have.
I mean [NOISE] I won't cut the price for this project.
It'll be something that's set now by AstraZeneca.
and it'll be based on what the other therapies are in the field and what they
believe payers will reimburse for. So payers have a lot of influence
nowadays in terms of what you can charge. In Europe it's even further down that
path. The government tells you what they're
going to reimburse you for and that's your prize.
So you know, I can't tell you what it will, what it will cost.
It will be more expensive than Allopurinol but hopefully we'll be able
to justify through pharmaco-economic studies, quality of life benefit.
In fact, the patients don't have flares, so they don't lose work for a week, they
don't show up in the ER. That whatever we charge for, it is well
supported by the, the economic benefit to the payer and the patient.
Sorry it's not a perfect answer but to, to tell you the truth, I have no idea
what it, what it would cost or will cost. So just briefly on the cancer program
that we've been involved with one of the, one of the great things about cancer
research today is the fact that we understand the molecular underpinnings of
many, many cancers and the signaling pathways that cause them.
And, and we now go after specific signaling targets rather than kind of a
shotgun approach of, of cell poisons. and so we, we're developing a series of
MEK inhibitors. MEK being a last in the Ras-Raf-MEK
pathway. so therefore the theory was you'd have
less resistance developing to a less opportunity for bypassing it.
and we know that drugs that work on the ra-, the early part of the pathway can
work extremely well. so MEK looked like a very interesting
target. We had a compound that was very selective
against MEK versus other kinases and better than the precursor Pfizer
compound. And work remarkably well in combination
with many other agents including Sorafenib.
and this, this data is the reason why that, that our compound and all of our
MEK inhibitors were acquired by Bayer, because Bayer sells Sorafenib and they
were quite intrigued by this combination data showing that even in
Sorafenib-resistant tumors you get 100% cell kill with the combination.
>> And you do that deal before the acquisition [INAUDIBLE].
>> Correct. So that, I, you see, I, you know.
Obviously the acquisition was unbelievable, but when it, when it comes
to kind of pretty exciting deals, this one actually is right up there, because
we acquired this program for zero money upfront.
Okay. We then with very small investment.
a couple years later, sold it to Bayer for 35 million up front, 15 million.
a couple years later when they hit the, the development milestone and another 7.5
a year later when they hit a second development milestone.
So you're looking at 57 and a half million of which I've hit one milestone
to back to the [UNKNOWN] the pharma. I got it from, if I paid them a million.
So when it comes to return on investment, this one has a really high, really high
return. Yes?
>> For I guess when you were looking at your program, were there other companies
working on the same project or like what are, what were their [UNKNOWN]?
>> So actually that was one of the things that I liked about gout was, this
is an area that has been almost completely ignored by large Pharma.
So there is, was zero competition. In fact, nobody even had any early stage
discovery work going on in gout. It's because, again, there was this
perception that, well, gout's already taken care of, Allopurinol works in
everybody and it's cheap. So I'm not going to spend my research
dollars on something going against the cheap generic, I'm going to spend it on
something where there's no treatment at all.
And, and so Pharma completely ignored gout, basically for the last 40 years.
The only people that have spent time, money, and study on gout are actually the
Japanese. Because the Japanese are convinced, based
on a 48,000 person railway workers study that hyperuricemia is closely linked to
increased cardiovascular death. And so, if you have hyperuricemia even if
you don't get gout, you're treated in Japan.
and so that other drug that was approved a few years ago, Uloric, actually it was
invented by Teijin, a Japanese company, because gout's important in Japan.
Not important in the rest of the pharma world.
So, it was a great opportunity to come up with something innovative with zero
competition. Don't get too many of those
opportunities. And finally even though stock performance
is not the metric that we use to see whether or not we've developed a good
product, it's really how well it helps the patient, certainly from the point of
view of return to investors on, you know, appropriate return for their investment.
you know, I don't think anybody who bought into the company early on at you
know, three or $4 is unhappy with $32 that we sold the company for.
[SOUND]. so, you know [LAUGH] I guess to finish
off you know, thinking about lessons learned.
you know, obviously being scientifically curious can pay off and sometimes pay off
big time. this was a situation of a completely
serendipitous observation, which truthfully could have been easily
ignored. first of all, it wasn't even made early
on, because clinicians tend not to look at uric acid.
and so it was ignored completely for awhile.
And even once it was identified, we could have just said okay, well it doesn't look
like it hurts anybody so that's fine, let's move on.
But we decided to spend some time investigating it.
and, you know, we weren't embarrassed to take what was a free new molecular entity
and, and develop it. And because it just happened to have all
the properties that one would, would hope for.
the other thing in this business, you gotta move as quickly as you can because
time is really critically important. And every step of development you build
value in that asset. and so if you want to get a high return,
you've got to get to that value as quickly as you can.
and so with this compound, we entered Phase 3, basically, three years from the
time we entered Phase 1. Not lot, not light speed, but in drug
world, that's pretty quick. Kind of like dog years this is, this is,
this is fast in drug development. and then as I mentioned before when we
were talking, you really have to build a commercial story.
You can't just say, oh, here's a drug, it clearly works, we've got spectacular
efficacy data. Great, nobody cares.
Like, okay, fine, if you got it approved, now show me you can sell it.
and that is something that, you know, very frequently gets missed by, by very
small biotechs cause they find some cool technology.
And they're sure that oh no, no, no problem, everybody will want it.
You gotta go and, you gotta prove it. And particularly to the people who come
in from large pharma, you gotta prove it to them with the data that they would
generate, if they went out and did market research.
so we spent millions of dollars on market research to prove what we thought.
and then you can't just stop on initial success.
We continued to do drug design around the initial finding of, of 594.
And we've now entered into, just about entering into Phase 2, is the next
generation compound that's 200 times more potent, has beautiful attributes and will
probably be dosed at you know something under ten milligrams a day.
So, very exciting compound. and ultimately, as I mentioned, the goal,
you know, that we have and most biotechs have is, is bringing a valuable drug to
patients in need. and then if you focus on that the
financial, you know, rewards follow. You don't have to worry about the
financial rewards. But the real focus is on the patient.
So with that, thank you very much for your attention.
[NOISE] If there's any other questions
