[LAUGH] Hello, everyone. My name is Susan Trieu and I will be giving a lecture today on regulatory considerations when filing an investigational do, new drug application. A little bit of background about myself, I've been in the pharmaceutical industry for over 12 years. One of those capacities was in regulatory affairs. And so that's why I've been asked to speak to you about it today. I'm currently in a different role though. I'm working at Medimpact which is a pharmacy benefits management company. So I've transitioned from being a Pharm.D.,. to industry, now to managed care and population-based work. So I'm hoping that I give you some real-life and real-world perspective. Please feel free to ask questions today, and I hope you enjoy the lecture. So my objectives today are, hopefully you've had a very interactive, couple of lectures already. But really what I want to discuss today is the interaction between health authorities and sponsors in drug development. Describe the roles of two major, heath authorities, the FDA, which you're all familiar with. And then as we live in this very global world, the EMEA, which is also known as the EMA or the European Medicines Agency. And then I will describe a little bit about filing of the IND. And then I've added another section this year which talks about what the goals of the FDA were in 2012, and how. The agency itself is kind of moving into the future if you will. So I'll be interested to hear what your comments are. And please let me know if you have any questions or if I need to clarify anything. One thing that you'll notice in this industry is there are a plethora of acronyms. So if you do not know anything on this slide, please let me know and we'll. We’ll address it promptly. So as many of you know and, as I’m sure it’s been addressed already in these lectures, drug development takes longer than it did in the past. Now I say that with the caveat of small molecular entities. So small molecular entities. Take a very long time, i.e., drugs to treat hypertension, drugs to treat migraines, drugs to treat depression. What I'm not referring to is, and call it speciality drugs. Like oncolytics or biologics in natures that would treat orphan diseases. Because as you know, those agents go through an accelerated program. And they often are approved in a short duration of time, because the need is so great. So what I'm really referring to here is, your run of the mill beta blocker, or your, or some agent along those lines, how long is it really taking? Well, we know that really, at the very beginning we're talking about pre-discovery. And does everyone understand the delineation between pre-discovery or pre-clinical versus clinical? Okay, so pre-clinical, when you talk about pre-clinical work, is that done in humans or non-humans? >> Non-humans. >> Right. So pre-clinical work is animal. Or as I tell my P.H.D. husband, it's rat data, and then anything outside of that, clinical data is done in humans. So it could be in a disease patient population or non disease patient population, but that's kind of the delineating factor. So, you know, we have all these drug candidates that are coming up, you know, 5,000 to 10,000 compounds. And it takes a while for those things to be processed. I mean, there's a lot of modeling that takes place. So you have your pre-clinical work. And then you say, we have enough evidence to say, I'm going to actually put this is a human being, a live person. And can you imagine what the regulatory complications or the, liability goes with that. So what we’re going to talk about is that big step. But I feel like before we do a who, whole lot of that, I have to give you a little bit of background. So, we’re going to spend the very beginning of the portion of the lecture today talking about the FDA. And who in a company actually interacts with these people. So, what do the authorities actually regulate, and when do you actually begin interacting with health authorities? Who do you think in a pharmaceutical company or a biotech company actually interacts with the FDA? Regulatory Affairs, so who makes up Regulatory Affairs, you think? So you'd be very surprised that regulatory affairs actually is a gamut of people. P.H.Ds, MDs, Pharm.D.s, people who've just had regulatory experience, if you will, they don't actually have to be lawyers. They're interacting with health authorities and they're reading federal guidelines and regulations. And making sure that, the whole drug process is being followed, if you will. And the best way that I can describe it is, this is probably a terrible analogy but the FDA is probably the worst boyfriend that you'll ever have in your life. And I'm glad I got you to chuckle a little bit. But, there's very little communication, it's usually one directional and you know it's one of these things where. They kind of give you an idea of what they want, but you don't really know. And so you're kind of following what you think is the right path, but it can change at any moment. So that's the kind of relationship that you should kind of consider. I mean, you do, and, and that could be the, you know, the worst girlfriend ever. I mean, I'm just kind of taking it from my perspective. But that's kind of the way you kind of have to view this relationship. I mean it has to be symbiotic in a sense where the FDA obviously is there for a reason, right? They're there to protect, serve, help the public if you will look, find effective medications that are safe as well. They are not at the whim of the sponsor or the manufacturer by any stretch of the imagination. But the manufacturer needs the FDA’s blessing in order to enter the market. So it’s a very interesting relationship, to say the least. So the Regulatory Affairs department, offers regulatory strategy for the development of products, domestic and global. That's why we're taking a little bit, a little bit about the EMEA today, or the EMA. We're not talking Japanese Health Authorities or the Canadian Health Authorities, because that's completely different as well. The two larger markets, although Asia is really expanding and growing, we'll talk, I guess we can dab, dabble in that a little bit. But those are the two big sections when you talk about a big pharma company. It's really when are we filing in Europe and when are we filing in the U.S.? And usually, I say usually, usually things kind of follow that course, okay? So Regulatory Affairs. If you're in the, if you're in the throes of trying to file an IND, trying to file an NDA, that's very exciting times for the company. That's product development strategy, things like that. But what about the maintenance, of a product that's already out on the market? Say you join a company that's had a product out on the market for three years. You'd be doing a review of product labeling, advertising, promotional material. Any time, a new trial comes out you'd have to review that. If there's a new hand-out that's given or new slide-deck, you from a regulatory perspective has to make, you have to make sure that that. Follows in suite with what the FDA allows the company or the sponsor to talk about. And then you have to also maintain drug applications and filings through periodic and routine submissions. We'll talk about what those routine submissions are fore an IND. I know that someone else, is it Doctor Lions again that's giving the. NDA talks, so she'll touch about, touch upon the NDA a little bit, but the IND requires maintenance as well. So, the evolution of the FDA, this is just a little historical, look if you will. But the FDA is an agency within the Department of Human and Health Services. This is pop culture. Does anybody know currently who the secretary of, of the Department of hu, Health and Human Services cur, is currently in this administration? >> Sebelius. >> Yes, very good. Kathleen Sebelius. Very good. So these are some things that we should probably know [LAUGH] at some point in time. But this, the FDA actually falls under this department. The Commissioner of the FDA is appointed by the President and affirmed by Congress. Confirmed by Congress. So their annual budget here, 3.1 billion in 2010, 74, 748 million of that came in user fees. Do you all know what user fees are? >> Fees are paid by the sponsor. >> Right. So when you file an application to the FDA, you pay a user fee for them to review it. Does it mean that they will give you a yes, thumbs up or a thumbs du, I mean, they're either going to give you this, this or this. I mean, really goes one way. Which is for those who can't see me, it's a thumbs up, a maybe we'll think about it if you give us more information or a thumbs down. Which is no way, and take your stuff and you know, work on it again or there's no way we're approving this product. But that's with the user fee. And you can see that from 2010 to 2012, there annual budget increased by over a billion dollars and so did their user fees. So it went up to 1.6 billion. And so what you can see is this, although the FDA doesn't really have any major obligations to the sponsor, a significant portion of their budget comes from sponsors innovations. So manufacturers, they can't really, there's, I mean, there's not a whole lot for the FDA to do if there are not, there's not constant innovation going on.
