Stringent rules govern clinical trials, which are monitored by the National Institutes of Health and the U.S. Food and Drug Administration.
Before the FDA approves a clinical trial, scientists perform laboratory tests and studies in animals to determine a potential therapy’s safety and efficacy. If these studies show favorable results, the FDA gives permission for the medication or device to be tested in humans.
The website clinicaltrials.gov, maintained by the NIH U.S. National Library of Medicine, is the primary resource to learn about current and upcoming clinical trials in the U.S. and internationally.
Starting at the home page, users of the site can search for clinical trials by disease or condition, location, and key terms, such as a drug or investigator name.
The federal government requires trials to be registered and to include detailed information on the site about eligibility criteria, site location, primary outcome measures, and other information.
The Rare Diseases Clinical Research Network (RDCRN)
The Rare Diseases Clinical Research Network (RDCRN) program, supported by the NIH National Center for Advancing Translational Sciences, advances medical research on rare diseases by providing support for clinical studies, collaboration, study enrollment, and data sharing.
The RDCRN comprises 20 individual clinical research consortia and a Data Management and Coordinating Center. Each consortium focuses on at least three related rare diseases or conditions, participates in multiple studies, and actively involves patient advocacy groups as research partners.
ResearchMatch is an organization that brings together people trying to find research studies and clinical trials, and researchers associated with registered IRB-approved studies on clinicaltrials.gov who are looking for volunteers.
National Patient Advocacy and Rare Disease Organizations
National Patient Advocacy and Rare Disease Organizations are a good source of information about clinical trials and clinical research being conducted for a specific disease.
The NORD website provides a library of information for more than 1,200 rare diseases, including national and international advocacy and research organizations for each disease.
Also, the National Institutes of Health (NIH) Genetic and Rare Disease Information Center (GARD) website contains an easy-to-use search tool that lists national and international advocacy organizations for specific diseases.
Disease Specialists and Clinicians
Disease specialists and clinicians are often knowledgeable about clinical trials and research currently in the pipeline for specific diseases. Frequently, researchers conducting clinical trials are medical doctors who may also see patients who are not enrolled in a study. These researchers may be able to refer you to a colleague who sees patients for your disease.
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