Staff Profile: Anne R. Pariser

Anne Pariser
Anne R. Pariser, M.D.


Office of Rare Diseases Research

National Center for Advancing Translational Sciences

National Institutes of Health

Email Anne R. Pariser


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. Most recently, she served as associate director for knowledge management in the FDA’s Office of Translational Sciences, which is part of the Center for Drug Evaluation and Research (CDER). Prior to serving in that role, Pariser was the associate director for rare diseases in the Office of New Drugs (OND) at CDER, where she established the Rare Diseases Program (RDP) in 2010 to support, facilitate and accelerate the development of therapeutics for rare diseases. Before founding RDP, she was a medical officer and team leader in OND, where she worked almost exclusively on the review and regulation of products for rare genetic disorders. Pariser has been involved in numerous collaborations within the FDA and with drug developers, governmental agencies, patient groups and other stakeholders to further the development of treatments for rare diseases. In addition to her government service, she works as a physician volunteer in the Arlington Free Clinic.
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.

Research Topics

Pariser’s research interests include the advancement of regulatory science to facilitate the efficient development of rare disease therapeutics, natural history studies, and the use of biomedical informatics to promote high-quality data capture to inform rare disease research and programs. 

I Am Translational Science Feature

Watch Pariser explain how translational science can help advance therapies for rare diseases.

Watch the rest of the I Am Translational Science video series.

Selected Publications

  1. Clinical trial safety population size: analysis of drug approvals for rare and common indications by FDA Center for Drug Evaluation and Research.
  2. A role for plasma cell targeting agents in immune tolerance induction in autoimmune disease and antibody responses to therapeutic proteins
  3. Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction
  4. Investigational New Drug applications: a 1-year pilot study on rates and reasons for clinical hold
  5. Important role of translational science in rare disease innovation, discovery, and drug development