Clinical Trials: What is a Randomized Control Trial?
Guest: Dr. Terry O’Brien - Cardiology
Host: Dr. Pam Morris – Cardiology
Dr. Pam Morris: Hi, I am Dr. Pam Morris and I am 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 were talking earlier today about randomized control trials. What is a randomized control trial, and how do we use that to study the benefits of a new drug?
Dr. Terry O’Brien: That is an excellent question, Pam. If one has a new medication or device that you think, based on your preliminary work, will benefit people, then one has to eventually apply it to human beings. This causes a certain very high standard of ethics that have to be applied. Basically, what you do is you offer the patient the very best standard of care therapy and then you do it with, or without, the new therapy. You explain to the patients the potential benefits, based on the preliminary work that has been done, or the potential risks, some of which may not be known for a new therapy. That is called a randomized trial because to try to make it as scientific as possible, which is part of the ethics, you have to get the answer you are looking for.
Neither the patient, nor the nurse, nor the doctor, knows whether they [patients] are on the therapy or not. This allows one at the end of the trial, when you break the code, to see if something really makes a difference or not. We sort of know this in cardiology. When we do a trial to get people to walk longer, pretty much everyone, whether they get a blank pill or not, walks longer, just because of the psychology of the human experience. What is different about a randomized clinical trial is that we scientifically and statistically get the answer to whether there is a real effect of not.
Dr. Pam Morris: When you are designing a randomized control trial, what is the approval process? Who gives you permission to conduct this type of research in humans?
Dr. Terry O’Brien: Well, the first layer is the investigators themselves. I mean, this is human experimentation and it has to be done with the highest ethical and scientific standards. Everyone understands that. So, the people writing the protocol have to be aware of this. The people who are funding the protocol, whether it be the government or whether it be a company, it has to be done independently and it has to have several layers of revision. Then, a particular protocol has to be approved by the institutional review board which is a panel of ethicists, lay people, scientists, who have nothing to do with the trial, who review it for appropriateness. Fundamentally, the Food and Drug Administration, the FDA, also has input on this because they will be the one finally approving this.
These trials also have what is called data safety and monitoring, individual ethicists and clinicians who see all of the information unblinded, unrandomized, so that if there is a surprise, either an unanticipated benefit or an unanticipated risk, it could be quickly recognized and the appropriate action can be taken.
Fundamentally, patients who participate are volunteers. They are volunteering to potentially help society, potentially help themselves, though not necessarily. It is a courageous thing they do and they are to be appreciated for this.
Dr. Pam Morris: You mentioned earlier about some studies being funded by the government and some studies being funded by industry. Are there pros or cons to participating in a research study that is funded by one or the other?
Dr. Terry O’Brien: Government studies, whether they be by the Veteran Affairs Division or by the National Institutes of Health, tend to be scientifically very sound but there are not as many as we would like to see. Industry is very interested in providing new medications because of the profit motive. All of the medications that are in the cardiovascular arena that we have been using over the past 25 years, have all come through, basically, drug companies developing them. So, the medications that we use to prolong life and increase quality of life have been developed by industry. So, if one wants to have advantages, one has to ethically, basically, form a partnership that is in the benefit of individual patients and society.
To say the same thing another way, 90 percent of clinical trials are funded by industry. So, one has to independently and rigorously do the right thing. If that is done, patients benefit, society benefits, the company benefits, for a period of time, until it becomes generic. It is a win-win situation.
Dr. Pam Morris: What are the potential benefits and risks to the patient participating in a clinical trial?
Dr. Terry O’Brien: One has to be very clear that when a patient volunteers for a clinical trial, they are volunteering to help doctors, our society, get the answer to the question at hand, whether this medication is beneficial or not. There may or may not be benefit to the individual patient. They may get a blank pill. That does not hurt them. They still get the standard of care, but there is not necessarily any benefit. The medication, or therapy, may end up not being beneficial. That is why one does the clinical trial. Of one knew it was beneficial, it would not be ethical to do a clinical trial. So, that is something that the volunteer has to keep in mind.
Dr. Pam Morris: I think your point is well taken. At the very minimum, a participant in a clinical trial will always receive the highest standard of care that is currently recommended. That is really an important point when considering your willingness to volunteer in a clinical trial.
Dr. Terry O’Brien: Absolutely. These patients get more attention. They get more blood tests. The get more imaging. They become more educated about their disease and the therapies that are available. Many times, these patients are pretty much on the ball which is why they are trying to seek out new therapies for what is often a devastating disease.
Dr. Pam Morris: Well, Dr. O’Brien, thank you so much for being here. I would like to come back in our next podcast and talk about randomized control trials that are currently available here at MUSC. So, thank you.
Dr. Terry O’Brien: You are welcome.
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