Anne R. Pariser, MD, Director of the Office of Rare Diseases Research, National Center for Advancing Translational Sciences at the NIH provides an overview of the Office of Rare Diseases Research at the NIH.
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) was officially established in fiscal year 2012 to transform the translational science process so that new treatments and cures for disease can be delivered to patients faster. NCATS, one of 27 Institutes and Centers (ICs) at NIH, strives to develop innovations to reduce, remove or bypass costly and time-consuming bottlenecks in the translational research pipeline in an effort to speed the delivery of new drugs, diagnostics and medical devices to patients.
NCATS focuses not on specific diseases, but on what is common among them and the translational science process. The Center emphasizes innovation and deliverables, relying on the power of data and new technologies to develop, demonstrate and disseminate improvements in translational science. In these ways, NCATS is serving as an adaptor to enable other parts of the research system to work more effectively. NCATS complements other NIH ICs, the private sector and the nonprofit community.
NCATS’ organization of divisions and offices spans the entire spectrum of translational science. Through programs in its Division of Pre-Clinical Innovation, the Center drives advances in early stages of the translational process, from target validation to first-in-human studies. Through its Division of Clinical Innovation, NCATS supports clinical and translational research, creating and sharing the expertise, tools and training needed to develop and deploy effective treatments in people. Our cross-cutting programs in rare diseases, translational technologies, strategic alliances and other emerging areas address common scientific and organizational barriers to enable faster and more effective interventions that tangibly improve human health.