Clinical trials are research studies in which people help doctors find ways to improve health and cancer care. Each study tries to answer scientific questions and to find better ways to prevent, diagnose, or treat cancer. (See What is a Clinical Trial? ¹)

If you or someone you know has cancer, you might want to learn what the best research has to say about its prevention, diagnosis, or treatment. But what constitutes the "best" research? If it's well designed, any clinical trial can produce reliable findings. But reliable findings aren't always definitive.

Research findings that are most likely to set the standard of cancer care usually come from phase III clinical trials that have been randomized and controlled, and that have enrolled enough participants to yield statistically significant results.

This article explains what these terms mean, and why a phase III randomized, controlled clinical trial with a specific number of participants is considered the gold standard in cancer research. With this knowledge, you'll be better able to tell which cancer studies are the most definitive, and therefore the most helpful, in guiding your medical decisions.

Glossary Terms

A clinical study that includes a comparison (control) group. The comparison group receives a placebo, another treatment, or no treatment at all.

A study to compare the results of people taking a new treatment with the results of people taking the standard treatment (for example, which group has better survival rates or fewer side effects). In most cases, studies move into phase III only after a treatment seems to work in phases I and II. Phase III trials may include hundreds of people.

A study in which the participants are assigned by chance to separate groups that compare different treatments; neither the researchers nor the participants can choose which group. Using chance to assign people to groups means that the groups will be similar and that the treatments they receive can be compared objectively. At the time of the trial, it is not known which treatment is best. It is the patient's choice to be in a randomized trial.

Describes a mathematical measure of difference between groups. The difference is said to be statistically significant if it is greater than what might be expected to happen by chance alone. Also called significant.