Rare Disease Clinical Research Training Program

Children'S Research Institute, Washington DC

Investigators

Abstract

The goal of the R25 Rare Disease Research (RDR) curriculum is to prepare a multidisciplinary group of rare disease (RD) clinical researchers to perform studies in both genetic and non-genetic rare disorders. This will be accomplished using a one-year 80-hour blended teaching model. An objective of this program is to create online modules, podcast-style lectures, recorded didactic-style lectures, and in-person experiences that enable learners from different health care disciplines and medical specialty backgrounds to actively participate in the curriculum. The curriculum focuses on subjects including RD-specific study design, unique analysis approaches, multi-center recruitment methods, and small cohort design. The field of RDR recognizes that collaborative research and team science are critical to improving therapeutic opportunities and clinical outcomes of the rare disease community. This concept serves as the unifying theme of the curriculum. We also highlight how researchers can fund their work through philanthropy, innovation, entrepreneurship, as well as traditional funding sources. By changing how the next generation of clinical researchers approach study design, recruitment, and funding, we expect to advance health among the approximately 30 million Americans who are estimated to have a rare disease.