Office of Rare Diseases Research (ORDR) Brochure
What is a Rare Disease?
A rare disease is a disease or condition that affects
fewer than 200,000 people in the United States. It is estimated that
more than 25 million people in the United States have a type of rare disease.
What is ORDR?
The Office of Rare Diseases Research (ORDR) was established in
1993 within the Office of the Director of the National Institutes of Health (NIH),
the Federal focal point
for health research. ORDR
coordinates and supports
for rare diseases,
2002, the Congress
and President Bush
established ORDR and its
responsibilities in statute
by enacting and signing
Public Law 107–280, The
Rare Diseases Act of 2002.
What Does ORDR Do?
- Recommends an agenda for conducting
rare diseases research.
- Supports research and training of NIH
rare diseases investigators, together with
NIH Institutes and Centers, at the NIH
Clinical Center hospital and medical
research centers throughout the Nation.
- Supports a rare diseases clinical research
network (http://www.rarediseasesnetwork.org/) and other scientific opportunities.
- Coordinates and encourages cooperation
in rare diseases research.
- Responds to scientific opportunities and
builds international collaborations.
- Supports an extensive scientific conferences
- Cosponsors, with the National Human
Genome Research Institute, the Genetic
and Rare Diseases Information Center.
- Provides information on genetic and
acquired rare diseases in English and
Spanish to patients, their families, health
care professionals, and researchers.
- Compiles yearly reports for Congress and
the public about NIH-supported scientific research plans on rare
What Is the ORDR Web Site?
The Web site provides information about
ORDR-sponsored biomedical research, scientific
conferences, and rare and genetic diseases.
It also serves as a portal to information on
major topics of interest to the rare diseases
The ORDR Web site links to:
- Information on many of the more than
6,000 rare diseases known today
- Contact information for the Genetic and
Rare Diseases Information Center.
- Planned, ongoing, or completed clinical
studies and trials.
- Research funding opportunities from NIH
and other sources.
- Patient recruitment and referral procedures
for the NIH Clinical Center hospital.
- Publications and reports.
- National and international voluntary
health organizations (patient advocacy
- Genetic counseling and testing.
- Information on transportation and lodging
for patients and their families during
diagnosis and treatment.
- Information on ORDR-sponsored scientific
- Other issues of interest to patients with
rare diseases, their families, researchers, health care
professionals, and the general public.
For Information about
Genetic and Rare Diseases
Information Center (GARD)
P.O. Box 8126
Gaithersburg, MD 20988-8126
Toll-free: (888) 205-2311
TTY: (888) 205-3223
International Telephone Access Number:
Office of Rare Diseases Research
National Center for Advancing Translational Sciences (NCATS)
National Institutes of Health
6701 Democracy Boulevard
Suite 1001, MSC 4874
Bethesda, MD 20892
Telephone: (301) 402-4336
Fax: (301) 480-9655
Last Reviewed: March 11, 2009