A clinical trial is defined as research using human volunteers (also called participants) that is intended to add to medical knowledge. However, long before a treatment is tested on a participant it is studied for many years in a laboratory. Once researchers determine that the new method is likely to succeed and is a better medical solution than current treatment options, researchers are required to present a “clinical trial protocol” (or study methodology) to the Food & Drug Administration (FDA) for rigorous review and approval. Additionally, by federal regulation, every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB), an independent committee of physicians, statisticians, community advocates, and others. The IRB is charged with ensuring that all clinical trials are ethical and that the rights, safety and well-being of the participants are protected.
Once FDA and IRB approved, a small pilot study where laboratory discovery is applied to patients is conducted, officially beginning the first phase of a clinical trial. Studies begin by enrolling a small number of patients to determine feasibility. As data is collected and there are a signs of benefit, larger scale studies with more patients are added. Clinical trials may compare a new medical approach to a standard one that is already available or to a placebo.
Once enough data is collected, it is presented to the FDA where the information is scrutinized. If the FDA believes, based on the evidence presented, that the therapy is safe and effective it will be approved for medical consumers.