Clinical trials are research studies in human volunteers and patients that help us find new ways of preventing, treating and diagnosing human diseases using novel drugs or biological therapies. These investigational treatments must be thoroughly tested in highly controlled environments for safety and efficacy before they are approved for general use by a nation’s regulatory health authority (such as the Food and Drug Administration [FDA] in the United States). Clinical trials can also be performed to evaluate methods of screening for diseases and improving a patient’s quality of life.

Before approval, a new therapy or diagnostic application must successfully complete several phases of clinical trials to prove safety, effectiveness and optimal dosing or procedures. The phases of these trials progressively involve more patients in a greater range of dose levels as the trials prove safety. Later trials typically use techniques such as blinding or randomization to ensure that the results are not biased. Initiation of each clinical trial must be approved by the appropriate health authority (such as the FDA). Additional safety monitoring is also provided by institutional review boards (IRBs) at the clinics that are running the trials and trial-specific data safety monitoring boards (DSMBs).

Before agreeing to participate in a clinical trial, a volunteer or patient should discuss his/her decision with family and a physician. Those participating in studies have a right to receive information about the trial (in the form of an informed consent document) and a right to withdraw from the study at any time and for any reason.