Research Process
The research process is a slow and careful one. Research begins in the laboratory where theories and hypotheses are tested. Once proven to work in the lab, research moves to the preclinical phase.
Preclinical research is an essential step in the process of understanding the complexity and mechanisms of disease. This type of research allows scientists and clinicians to create models of disease states to uncover the behavioral and cellular processes responsible for the development of disease. As the precursors and course of action of a disease are more fully understood, preclinical research is then used to test and determine the safety and efficacy of treatment theories. The next step in research is the clinical phase.
Phase I clinical research is the first step in testing a new treatment in humans. These studies are usually small (10-15 participants) and designed simply to show that a treatment is safe in humans. In drug studies, the dose of the drug is incrementally increased to find the best dose.
Phase II clinical research studies are further tests of safety in a larger population. In addition, researchers are beginning to evaluate the effectiveness of the treatment. There are usually further refinements of the dose of a drug. Phase II studies are not designed to prove that a treatment works.
Phase III clinical research studies are large studies designed to prove the effectiveness of a treatment and further confirm their safety. They are usually placebo-controlled, meaning that the new treatment will be compared to current standard therapy plus placebo. What is a placebo? A placebo is an inactive, harmless substance that is compared to active study drug. One group of patients receives the study (investigational) drug, while a second "control" group will receive a standard treatment along with placebo. Often the participant, the physician, and the study coordinator are "blind" to which group is getting the actual drug and which is receiving the placebo. The "blind" study method prevents bias on the part of the patient, the doctor and the study coordinator.
Once a Phase III study is complete, the data are analyzed and submitted to the FDA for approval of the drug or therapy.
Phase IV clinical research studies evaluate FDA-approved drugs for new indications. For example, a drug has been approved for high blood pressure. Investigators notice that patients on the medication for high blood pressure and who also have congestive heart failure show an improvement in their heart failure. Investigators then may do a Phase IV study of the drug in heart failure patients to see if their hypothesis is correct. If correct, the drug would then also be approved for congestive heart failure.
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