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Brooks et al. Rare Dis Orphan Drugs J 2024;3:15 Rare Disease and
DOI: 10.20517/rdodj.2023.27
Orphan Drugs Journal

Perspective Open Access

The division of rare diseases research innovation at
the national center for advancing translational

sciences, NIH: mission, history, and current
research activities

P. J. Brooks, Alice Chen Grady, Stephen Groft, Linda Ho, Joanne Lumsden, Meera Shah, Eric Sid, Yanji
Xu, Ainslie Tisdale, Jim Dickens, Dominique Pichard, Tiina Urv

Division of Rare Diseases Research Innovation, National Center for Advancing Translational Sciences (NCATS), National
Institutes of Health, Bethesda, MD 20892, USA.
Correspondence to: Prof./Dr. P. J. Brooks, Division of Rare Diseases Research Innovation, National Center for Advancing
Translational Sciences (NCATS), National Institutes of Health, 6701 Democracy Boulevard, Suite 206, Bethesda, MD 20892,
USA. E-mail: pjbrooks@mail.nih.gov

How to cite this article: Brooks PJ, Grady AC, Groft S, Ho L, Lumsden J, Shah M, Sid E, Xu Y, Tisdale A, Dickens J, Pichard D, Urv
T. The division of rare diseases research innovation at the national center for advancing translational sciences, NIH: mission,
history, and current research activities. Rare Dis Orphan Drugs J 2024;3:15. https://dx.doi.org/10.20517/rdodj.2023.27
Received: 8 Aug 2023 First Decision: 16 Sep 2023 Revised: 17 Nov 2023 Accepted: 27 May 2024 Published: 3 Jun 2024

Academic Editors: Jacques S. Beckmann, Daniel Scherman, Daniel O’Connor Copy Editor: Fangling Lan Production Editor:
Fangling Lan

Abstract
The mission of the NCATS Division of Rare Diseases Research Innovation (DRDRI), formerly known as the Office of
Rare Diseases Research, is to advance rare diseases research to benefit patients. DRDRI is part of the National
Center for Advancing Translational Sciences, one of the 27 components of the US National Institutes of Health.
DRDRI facilitates and coordinates NIH-wide activities involving rare diseases research, as well as directly
supporting rare diseases research activities. These activities include the development and maintenance of a
centralized database on rare diseases; collaboration and coordination with organizations focused on orphan
products development and rare diseases research across the globe, advising the Office of the NIH Director on
matters related to NIH-sponsored research involving rare diseases; and responding to information and policy
requests about rare diseases within the NIH. DRDRI also supports various rare diseases research activities,
including the Rare Diseases Clinical Research Network, rare disease-related conference grants, and assessment of
the costs of untreated rare diseases. In addition, several of the projects DRDRI is supporting are “many diseases at
a time” translational approaches for rare diseases, which emphasize leveraging commonalities across multiple rare

© The Author(s) 2024. Open Access This article is licensed under a Creative Commons Attribution 4.0
International License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, sharing,
adaptation, distribution and reproduction in any medium or format, for any purpose, even commercially, as
long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and
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