Clinical trials are well-designed studies that collect information about new treatments for diseases and disorders. Most of the time, this means medications, but clinical trials can also test other things, such as stem cell therapies, surgical techniques, tests for diagnosis, medical devices, as well as others. In particular, clinical trials focus on administering an experimental therapy in humans, as opposed to animals, which are conducted first in the lab (known as pre-clinical research).
Without clinical trials, doctors and other prescribing healthcare providers (such as nurse practitioners or physician’s assistants) cannot prescribe medications or recommend other medical treatments. These studies are needed to understand two important types of information: 1) that the treatment is effective (also called efficacious), ie, that it really works and 2) that the treatment is safe for use in humans.
In addition to helping patients by making the best possible treatments available, clinical trials also advance scientific understanding of a disease or disorder.