Clinical Trials: An Overview of the Approval Process for Medications

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Dr. Morris: Welcome to an MUSC Health Podcast. Hi, I'm Dr. Pam Morris, and I'm here with Dr. Terry O'Brien, who is Professor of Medicine and Director of Clinical Cardiovascular Research at the Medical University of South Carolina. We're talking now about the approval process for new drugs through the Food and Drug Administration. And Terry, this has really become quite a problem now as we rush to find new treatments for many of the diseases that are devastating our nation. And yet after these medications are approved we're oftentimes finding side effects that we had not anticipated or uncovered during the approval process. So let's talk about how does a drug go from development to clinical trial and release for the public.

Dr. O'Brien: Well thank you, Pam. That's an excellent question. Although I don't know if I would use the term problem so much as inspirational. If you look at our society since World War II, the average adult lifespan has gone up twenty-five years. If you say what's done that in medicine, it's the scientific approach to clinical trials. And so I would offer that this is the primary thing that our field, the field of medicine, has offered to the United States and the developed world. As with any such process, and there's really nothing else in our society that has this kind of contribution or track record, there are going to be problems. And there are going to be issues that have to be dealt with. For the approval process of a new drug or medication there generally involves five to eight years of lab work, work with cells, and work with animals, taking that good idea and moving it forward. When a medication or therapy is finally to the point where we think it would benefit people, it has to be a very ethical, humanistic approach on the one hand to balance the needs of an individual who's volunteering or who has, commonly, a devastating disease, versus the overall benefit to society. The FDA has deemed the first phase a Phase I. And that's where you have a medication or a device and essentially, after extensive laboratory and animal testing that is highly ethical and highly regulated, it is used in normal, healthy volunteers. The thinking being is if there is an unanticipated side effect, that a healthy person would be able to tolerate it more. And these healthy volunteers receive the medication, it's looked at for side effects, and appropriate doses are maintained.

Dr. Morris: Now Terry at that stage in a Phase I, you would assume then that going into the Phase I study there would be the assumption of relative safety for a healthy volunteer.

Dr. O'Brien: That's absolutely an excellent point. Any medication in that the average is 5,000 medications to one just to make it to Phase I. It would have been extensively tested before it gets near a human being.

Dr. Morris: I would assume Terry then that the dose that is used in the Phase I studies would be a dose that has been found to be safe in animals, and the assumption would be it's safe in humans.

Dr. O'Brien: That's absolutely correct. One starts with a small dose, and one is looking for a therapeutic effect, and you try to balance the two.

Dr. Morris: Now, in the Phase II study, after you've found safety in the healthy volunteers, what would be the next research point?

Dr. O'Brien: The next step is to take people who actually have disease, whether it be a heart attack patient, a heart failure patient, or a cancer victim, and one has to be very careful here. These patients have a devastating disease. They're often very intelligent, and they know the limitations of therapy, and they're very eager to try new things. So this is a highly regulated process, where ethics and individuals' safety is of paramount importance. Basically, one takes the fi

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