The University of California San Diego, Skaggs School of Pharmacy and Pharmaceutical Sciences Drug Development 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 development. In this course you will learn the different stages of clinical development as well as the regulatory including but not limited to, an Investigational New Drug Application (IND), New Drug Application (NDA), and product labeling. Additionally you will learn how to Incorporate study design methods for consideration in the design of clinical protocols to assess safety, tolerability, and efficacy in multiple therapeutic areas. In this course you will learn the different phases of clinical development: * Phase 1 or early stage clinical trial are conducted primarily to determine how the new drug works in humans, its safety profile and to predict its dosage range. It typically involves between 30 and 100 healthy volunteers. * Phase 2 or Proof of Concept POC studies test for efficacy as well as safety and side effects in a group of between 30 to 200 hundred patients with the disease for which the new drug is being developed. * Phase 3 or late stage clinical development involve much larger group of patients, between a few hundred to thousands, depending on the indication, which will help determine if the new drug can be considered both safe and effective. It will involve control groups using placebo and/or current treatment as a comparison. * Product registration and approval process after a drug is considered safe and effective from Phase 3 trials, it must be authorized in each individual country before it can be marketed. All data generated about the small molecule or biologic is collected and submitted to the regulatory authorities in the US at the FDA, Food and Drug Administration FDA, in Europe the EMA or European Medicines Agency, Japan Ministry of Health and other countries which may require their own national approvals. This course is intended as part 2 of a series: Drug Discovery (https://www.coursera.org/learn/drug-discovery), Drug Development and Drug Commercialization (https://www.coursera.org/learn/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.
Clinical Study & Start-up Activities V
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Из курса от партнера University of California San Diego
Drug Development
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Clinical Study & Start-up Activities, Joseph Ma, Pharm.D.
This module is presented by Dr. Joseph Ma, Associate Professor in the UCSD Skaggs School of Pharmacy and Pharmaceutical Sciences.
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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
Okay, so I've got four or five more slides regarding choosing, how to, you
know choosing a site to perform the study.
We're going to highlight SEV, contracts and budgets, and SIV.
So this can be pretty not of the most interesting.
So I tried to make it interesting so I'm going to give you analogy.
I look at this as when you choose a side to perform a study, its, its an analogy
of what you want to get married. So maybe some of us are married, some of
us aren't, probably had the the not much worrying here.
The fun or the stress of planning for a wedding.
So, the analogy I'll choose is kind of when you're choosing a site, it's the
same type of questions and in your approach as you were planning for your
wedding. So, for your site evaluation visit I, I
look at that as the questions you want to ask about the site SEV is kind of
analogist to trying to determine a suitable wedding location.
Contracts and budgets, are just like a wedding, they're just like a wedding, and
it's negotiation, it's getting a cost per procedure for a study.
For a wedding, it's like cost for a DJ, cost for flowers, and et cetera, we'll
get into that. A site initiation visit, the analogy I
have is it's just like a wedding rehearsal where you kind of do a dry run,
so. So for your site evaluation visit, there
are probably three or four key questions you can ask yourself.
One of the most important is, what's the amount of experience at this site where
you want to potentially do your study? How many studies are they currently
doing? the reason we get into that is we want to
know if they have a lot of studies, then how is the study that I'm approaching,
that we want to do, going to be the priority?
there are certain institutions where if it's not being done in a hospital you can
do them at a CRO. So they're called a contract research
organization, where they have a database of healthy subjects where they primarily
do phase one studies. And so if they're doing, let's say, 20
phase one studies from other companies, you, you're competing for those.
You're competing for those healthy subjects who are enrolled in the
university. They can't be enrolled in two clinical
studies at the same time, they can only be enrolled in one.
How many phase one studies has the site done?
so what I mean by that is, I would say, in a traditionally, if studies are done,
let's say at a hospital, or at an institution they may have more experience
in doing phase two, phase three studies in patients.
Becuase they have let's say maybe the expertise and, or the patient load in
terms of having patients. But for healthy studies, and and in our
more standing example we'd probably want to know have they done, not only
have they done a lot of phase one studies.
But do, do they have, or how many muscle biopsies have they done with other
studies, because that's a key element for the study you want to get across is if
they've never done a muscle biopsy, then you're like hmm.
You may be a little bit worried, versus other sites that have done it that may
help in terms of what you need to get done.
So the analogue I have to your wedding is will you be the only wedding that day?
I just remember one wedding I went to was at this big site and they have like four
weddings there at the same time. And I remember walking in and, this is
before wedding crashers came out, but as I'm walking in I said, I'm at the wrong
wedding. And he had literally walked across to
another room, and that was the wedding I had to go to.
And so it's just like at a clinical site, they can be running multiple studies, you
want to make sure that they can do your study and meet your timelines.
Alright, so who will be the PI or principle investigator?
And I think it really depends on the phsyician's experience in clinical trials
in area of expertise. So has he or she done many phase one
clinical studies. If they haven't, then you really want to
be careful in terms of, okay, they may not have the experience that we want in
an effort to do the phase one study that you want to do.
Have they ever been placed on clinical hold by the FDA?
That's just a straight up question where you don't, don't want to cherry-coat it.
You just say, have you been, because some, they will they have to disclose it.
And if they have been, you want to know, well why have you been on clinical hold.
was it something where they had a major protocol violation, was it something
where they weren't meeting good manufacturing or G and P processes, so
you want to know. for our myostatin inhibitor example, I
think it would be, it would make sense that we want an experienced physician who
knows how to do muscle biopsies. So, if you had a site where they've never
done it, you gotta be really careful, at least concerned about whether or not you
want your study there. So, the analogy I had for your wedding,
it's here's going to be your priest minister or judge for the wedding.
you know, weddings can be different in terms of respecting specific cultural or
religious procedures that need to be done.
So does that person, minister, or priest are they aware and able, are they able to
conduct a specific request for that for the ceremony?
This site have the necessary equipment to conduct the study.
So, I mean we were talking everything from 12-lead EKG machines, centrifuges
for sample processing. Freezers like you said freezing muscle,
biopsies we need to snap freeze them in and freeze them right away to their
freezers that are what minus 15 or minus 80.
I mean down to that nitty gritty you know, detail.
So, for our myostatin inhibitor I alluded to this earlier, do they have access to
liquid nitrogen for snap freezing. And and in, in terms of our wedding
analogy you know are there wedding tables, chairs, lighting, outdoor or
indoor setting, changing room, et cetera. So, all those little pieces of equipment
are important. All right, I have one slide on contracts
and budgets. I think it goes without saying, get
everything in writing. And I think, specifically what I mean by
that is you need to have language or content.
What happens if this study is discontinued early?
How will payments be scheduled? And what's also just important is that
there's always going to be legal representation from both sides.
Because,um, there's always going to be a discussion in terms of liability.
So, if there is subject injury over the course of the study, what is the language
in terms of who is liable, what gets paid for, if you will, how is care going to be
provided. And I also think there is also an issue
in terms of publication. So, a lot of institutions want to have
the results, they want to be listed as an author.
They want it to be published but from a pharmaceutical company perspective.
You might not necessarily want to release that info or do so right away and you
have that right to do so. Budgets, there's a cost for everything,
so budgets in a clinical study it is almost a line on per procedure, per study
basis. Cost for everything whether be for blood
draws, the muscle biopsies physician time, nursing time, pharmacy time, data
entry, lab supplies you name it. the sites that you study there are very
creative in terms of trying to get as much and justify as much as they can get
based on the procedures that have been done.
So, the analogy I have for wedding how much to spend for cake, DJ, flowers,
party favors, open bar, et cetera. Okay, two slides on slides on site
initiation visit, and I think one of it is, what's the purpose of an SIV?
It's to clarify the requirements of the protocol.
So you're going to be reviewing the IE criteria with the site.
You're going to be reviewing if they have obtained proper subject consent.
Have they got IAB approval? And you are actually going to review the
actual process of implementing the protocol.
So, what you'll usually do is you will go to the site?
And this is in a context where you've done you SCV.
You've gone through the contract and budgets and you've said, okay, we are
going to use you as a site. Now, what happens next is the SIV, site
initiation visit. So when you go there and you talk about
the process of implementing you basically say alright.
Tell me what you're going to do on that very first day when they come in for your
Day one visit. You know, what gets done first?
Is it vital signs or is it a blood pressure, or is it a lab draw?
what's the timing of when the drug is being given and what time do you take
blood after the drug is being dosed? So all of that detail gets talked about
and the analogy I have for a wedding, it's like a dry run of the wedding
ceremony. And it's to the level of, you know where
are you going to, where' the bride and groom going to be standing, where's the
wedding party going to be placed, almost to that that detail.
So that, again, that first day, there's a lot of anxiety, a lot of stress.
There're hopefully, no hiccups in terms of that, what we call first pay first
patient first dose. Some references for this talk, and if
there are any questions, I'll take them now.
Thank you.
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