David A Pearce
Sanford Research, USA
Title: Need for patient registries for rare disease clinical trials and how to integrate them into the electronic medical record
Biography
Biography: David A Pearce
Abstract
Rare diseases affect an estimated 350 million people worldwide, or 1 in 10 people, and approximately 7000 different rare diseases exist throughout the world today. These are striking numbers, especially considering that 90% of those affected with a rare disease are treated with off-label prescriptions, and less than 1% of rare diseases have an FDA approved drug. Families affected with rare diseases often have little support to turn for information about treatments, support or current research. Researchers and physicians studying rare diseases frequently lack the amount of patient information needed to further their research, in turn inhibiting development of new therapeutics for treatment of rare diseases. Clinical trials for rare diseases are challenging because the patient population is so small, and participants are difficult to find. In 2010, Sanford Research started the Coordination of Rare Diseases at Sanford or CoRDS to try and curb the lack of centralized, collated and available patient information. CoRDS serves as a national rare disease registry that connects rare disease patients with researchers, physicians, patient advocacy groups, other rare disease families and genetic counselors. The ability to have a database of rare disease patient information is key to accelerating research in rare diseases that affect only a few people worldwide