Director
Office of Rare Diseases Research
National Center for Advancing Translational Sciences (NCATS) National Institutes of Health (NIH)

Anne Pariser, M.D. is the director of the Office of Rare Diseases Research (ORDR) at the National Center for Advancing Translational Sciences (NCATS) NIH. ORDR is dedicated to accelerating rare diseases research to benefit patients, through rare diseases programs such as the Rare Diseases Clinical Research Network, Genetic and Rare Diseases Information Center (GARD), and the NCATS Toolkit for Patient-focused Therapy Development. Important translational science research initiatives for rare diseases at ORDR include establishing best practices and tools for good quality natural history studies, data standards and sharing initiatives, the development of diagnostic support tools, and rare diseases therapeutics development, as well as translational and basic science research grants and collaborative programs.  Dr. Pariser came to NCATS in 2017, and before this, she worked for 16 years at the US Food and Drug Administration Center for Drug Evaluation and Research, where she founded the Rare Diseases Program in FDA CDER’s Office of New Drugs in 2010 and served as a Medical Officer and Team Leader for rare diseases drug and biologics product development, review and regulation. Dr. Pariser has 20 years of experience in rare diseases research, and her current research interests include “many diseases at a time” research approaches, such as platforms for gene therapies and other rare disease product development, and informatics approaches to diagnosis.