Clinical trials are in essence research studies done in a controlled setting that explore whether or not a new or unique medical strategy, device or treatment, is safe and effective when used on patients or groups of patients in the treatment of a specific disease or condition. The primary purpose of any study is to produce quantifiable research, but to accomplish this goal the study follows strict scientific guidelines. These guidelines are necessary to not only protect the patients and study subjects but to also produce the most accurate and reliable data for healthcare decision making. A new study is generally one of the last stages of a generally drawn out and thorough research process on any new medical strategy, or treatment.
These studies are a key tool in advancing medical knowledge and the treatment of disease, infection and common medical conditions. Studies can yield both positive and negative results and on occasion can show completely unexpected results. One of the common types of devices used in studies to test the effectiveness of a new medication, as an example, is the use of a placebo. A placebo or “sugar pill” is introduced to half the test subjects as a control and as a tool to measure what is known as the “placebo effect” on the participants. This placebo effect is any positive result that is seen by those participants not taking the new medicine. Another reason for using placebos is to note the percentage of study patients taking the placebo exhibiting side effects of the disease being treated.
Defining the phases of a clinical trial is important in any kind of study. Pre-trial or phase one looks for changes caused by the drug under study in laboratory investigation or in small controlled testing involving animals. The object in phase two is to hopefully demonstrate that the drug does provide some positive effect and that it is not toxic to the affected population. Phase three is to establish the effectiveness of the drug as required by the FDA. It also establishes toxicity levels and toleration of the compound in the patient population. Phase four clinical trials are in essence post-marketing studies which help identify uses that were not specified in the original studies.
While many clinical trials are done using separate and distinct sites using the same protocols, multi-center trials are often used to provide more and better statistical data. These larger data points help the groups running the clinical trials determine the effectiveness of the treatment or process under study and its probable effect on the population at large. The other factor in these studies is their enormous cost. The total cost can run into the hundreds of millions of dollars and can literally take decades to complete. This is absolutely necessary since the requirements on FDA approval and advertising of any new treatment or drug require that it be thoroughly tested and deemed clinically safe. Because of these requirements this testing process must be accomplished. This is also why you only see large corporations involved in these kinds of studies.