Phase I Clinical Trials: What's at Stake
Clinical trials are an integral aspect for the advancement of scientific and medical knowledge. Without the conduct of clinical trial studies, there is no way information about the safety and effectiveness of a drug or new therapeutic can be acquired. In a previous post, I give you sort of like a general oview of clinical trials. In the conduct of pharmaceutical clinical research studies, a drug undergoes four phases before making it to the market shelves. I am going to introduce you to phase 1 clinical trials. After this introduction, you are welcome to make your judgement of the pros and cons of clinical trials.
What do Phase I Clinical Trials Entail?
Let me reiterate what I said in a previous post: before phase 1 clinical trials can begin, sufficient evidence obtained in laboratory or animals must show the drug is safe enough to be tested in humans. Phase 1 clinical trials is what clinical researchers call "first in man" trials (FIH). At this stage, the drug is being introduced for the first time in people. But before phase 1 research can begin, an application called Investigational New Drug (IND) application must be submitted to the FDA or appropriate regulary authorities for authorization (More on the IND application on another post). Let's assume the IND is "in effect" (INDs are not approved), trial phase 1 can then begin. In phase 1 trials, the drug is tested in healthy subject volunteers for a limited time, while they are closely monitored for the effects of the drug in all bodily systems. There are, of couse, special circumstances when the drug cannot be tested in healthy volunteers (cancer, HIV drugs). In this case, diseased patients with the condition for which the drug is will be used. Phase 1 clinical trials, usually last for about a couple months (9-12 mos). Now you know what phase 1 clinical trials are, but what exactly do investigators want to know about the drug?
Research Outcomes of Phase I Clinical Trials
Okay, remember that during preclinical setting, researchers monitored whether or not the drug was safe to be introduced in people using animal models? Well, in phase 1 clinical research trials, the goal is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug in those healthy individuals. In addition, they also want to know more about the drug's biological activity or mechanism of action (MOA). Another important thing they are trying to do here is to establish dose ranges for subsequent study phases. A small number of volunteers are used in this phase (10 to 50 is a good number). Notice that healthy subjects are used in this phase. The question that we can ask here is, why would healthy people--not affected by the drug's indication--want to participate? Or is it even ethical to introduce a drug, for which the safety and efficacy profile have not yet been established, in healthy people? There are a gazillion of answers that can be given. I think one must know how the mechanism of action of the drug in both healthy and diseased inviduals so that signs of "OD" can be known. Research participants can get compensated as well, especially clinical trial phase 1 people. I am not going to rant about all the possible reasons why people should participate in clinical trials, especially healthy people. But suffice it to say, phase 1 clinical trials involve a small number of individuals, and that the purpose is to determine the safety, tolerability, and the establishment of dose ranges for subsequent studies.
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