Preclinical studies can help scientists learn how a potential therapy interacts within living systems before being tested in humans. Testing is carried out using cell and animal models of the disease, samples of human or animal tissues, and possibly computer simulations. When a therapy has the potential to be both safe and effective in humans, a Sponsor, often from the pharmaceutical industry, will begin to move forward towards clinical trials.

In addition to supporting preclinical studies, multiple opportunities exist for your group to help prepare for clinical trials. Data from your patient registry and natural history study can help Sponsors design clinical trials. Established clinical centers for your disease may serve as sites for the clinical trial. Your patient group can also perform patient preference studies and participate in initial meetings between the Sponsor and the FDA.