Dr. Anne R. Pariser

National Center for Advancing Translational Sciences , Bethesda, MD, USA

Dr. Anne Pariser joined NCATS in January 2017 as the deputy director of the Office of Rare Diseases Research (ORDR). As of February 2018, she is the ORDR director and works to advance diagnosis and treatment for rare diseases through research. Pariser comes to NCATS from the Food and Drug Administration (FDA), where she had worked since 2000, mainly on the development of drug and biological products for rare diseases.

Pariser received her bachelor’s degree in chemistry from Bates College in Lewiston, Maine, and her medical degree from Georgetown University School of Medicine in Washington, D.C., where she also completed her training in internal medicine. Pariser is board certified in internal medicine.


DAY 3: September 13, 2019 | 11:30 AM - 11:45 AM

Networks and Tools for Rare Diseases Systems Medicine Research

Anne.R. Pariser, MD, Office of Rare Diseases Research NCATS, NIH, Bethesda, MD, USA

The National Center for Advancing Translational Sciences (NCATS) NIH was established to transform the translational science process so that new treatments for disease can be delivered to patients faster. The Office of Rare Diseases Research (ORDR) within NCATS is dedicated to accelerating the development of treatments for rare diseases. NCATS ORDR has a number of programs intended to improve the research environment for rare diseases so that no disease and no patient will be left behind, regardless of the number of patients living with a rare disorder. ORDR’s programs include the Rare Diseases Clinical Research Network (RDCRN), which includes 20 centers of excellence, each of which studies 3 or more related disorders, and collectively includes more than 200 different rare diseases. The RDCRN provides collaborative awards to further multi-disciplinary rare diseases research, conduct natural history studies and clinical trials, and provide training for young investigators. It also includes a Data Management and Coordinating Center (DMCC), which is focused on developing tools and common processes to collect, standardize and share rare disease clinical research data. Some of ORDR’s other programs and initiatives include the Genetics and Rare Diseases (GARD) information center, grants for clinical trial readiness, and “platform” approaches to advancing gene therapies for rare diseases. NCATS additionally supports programs such as microphysiologic systems, also known as “tissue chips”, and the Therapeutics for Rare and Neglected Diseases (TRND) program that focuses on solving difficult areas of translational research for rare diseases in order to advance the field of rare diseases research.