Our Impact on Clinical Trials

Richard T. Moxley, III, M.D., professor of neurology and pediatrics at the University of Rochester School of Medicine and Dentistry, visits with a patient in the Clinical Research Center participating in a myotonic dystrophy clinical trial. (Vince Sullivan, University of Rochester)

Clinical trials are a necessary step on a treatment’s journey to the medicine cabinet. But the barriers to success in clinical trials are high. A clinical trial’s design and approval can take a long time. Even after completion, a trial may not have enough participants to produce robust results, or the results don’t demonstrate that a treatment will work for everyone who needs it. 

NCATS improves clinical trial practices so more safe and effective therapies can be efficiently tested and developed for the people who need them. We work with our partners to develop trial designs for testing multiple drugs, strategies for enhancing diversity among participants, and best practices for engaging the community at all stages. These approaches allowed major clinical trials for potential COVID-19 treatments to swiftly offer definitive results. 

More innovative clinical trial approaches are on the horizon, especially for rare diseases. We are testing whether biomedical devices that use a patient’s own tissues could speed trial design and execution. We’re also developing a clinical trials approach that tests a single therapy on multiple diseases, as well as an approach for increasing the success of clinical trials for rare diseases.

Explore All Areas of Our Impact