Clinical Trial Readiness (CTR) for Rare Diseases, Disorders and Syndromes

Clinical trials are key to developing and evaluating new treatments for rare diseases. Scientists, however, often do not have enough information about the symptoms and biology of rare diseases to design clinical trials. We worked with the Eunice Kennedy Shriver National Institute of Child Health and Human Development to create the Clinical Trial Readiness (CTR) for Rare Diseases, Disorders and Syndromes grants to address some of the obstacles scientists face, including gaps in our understanding of a rare disease’s natural history and a lack of suitable biomarkers or clinical outcome measures.

Through these grants, we seek to encourage rare disease research by supporting efficient and effective movement of candidate therapies or diagnostics toward clinical trials and to increase their chance of success. These grants are modeled after a grant program at the National Institute of Neurological Disorders and Stroke.