Toolkit to Prepare for Clinical Trials
In addition to supporting preclinical trial research, your group can work with key stakeholders to lay the foundation for successful clinical trials.
Toolkit to Prepare for Clinical Trials
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 toward 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 U.S. Food and Drug Administration (FDA).
Establish Clinical Sites
A Center of Excellence Network provides multiple sites for clinical trials.
Help Industry With Clinical Trial Design
Learn the type of information you can share with Industry to enhance clinical trial design.
Participate in Initial Industry-FDA Meetings
Find out ways to ensure you are invited by the Industry Sponsors to meetings with the FDA.
Perform Patient Preference Studies
Share patient preference study data with the FDA for development of benefit-risk assessment.
Give Input on the Informed Consent Process
Providing the patient perspective on the informed consent process may increase patient participation.