Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases. This allows researchers to ask and answer questions in a way that results in reliable information about the drug and protects the patients. Most clinical trials are classified into one of three phases:
These first studies in people evaluate how a new drug should be given (by mouth, injected into the blood, or injected into the muscle), how often and what dose is safe. A phase I trial usually enrolls only a small number of patients, sometimes as few as a dozen. Cancer patients who enroll in these trials have already tried other treatment options.
A phase II trial continues to test the safety of the drug, and begins to evaluate how well the new drug works. Phase II studies usually focus on a particular type of cancer and typically enrolls fewer than a hundred patients.
These studies test a new drug, a new combination of drugs or a new surgical procedure in comparison to the current standard. A participant will usually be assigned to the standard group or the new group at random (called randomization). Phase III trials often enroll large numbers of people and may be conducted at many doctors' offices, clinics and cancer centers nationwide.
In addition, after a treatment has been approved by the Food and Drug Administration and is being marketed, the drug's maker may study it further in a phase IV trial. The purpose of phase IV trials is to evaluate the side effects, risks and benefits of a drug over a longer period of time and in a larger number of people than in phase III clinical trials. Thousands of people are involved in a phase IV trial.