WASHINGTON and CAMBRIDGE, Mass., March
1, 2021 /PRNewswire/ -- Children's National Hospital and
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK)
("Takeda") today announced the creation of the Rare Disease
Clinical Activity Protocols (Rare-CAP) program, which will
establish a networked system for the development, dissemination and
curation of protocols to help standardize the process of diagnosis
and care for patients with rare diseases.
Rare-CAP will be led by the Rare Disease Institute at the new
Children's National Research & Innovation Campus that opened in
February 2021 on the grounds of the
former Walter Reed Army Medical Center. A $3.85 million commitment over five years from
Takeda will help launch and sustain the program. Rare-CAP will
leverage several novel features to serve as a protocol platform
that reduces barriers for researchers, clinicians and patients in
determining appropriate diagnosis and clinical care for rare
disease patients. This includes ongoing input from patients and
families and an open "wiki" format for near real-time updates from
vetted contributors to enable access to the latest, real world
data.
Rare diseases impact hundreds of millions of families around the
world1. Currently, no overarching model exists for
uniform standards of care and diagnosis. It can take years for
patients to receive a correct diagnosis for rare diseases. Even
then, care protocols are often hard to find, amplifying the pain
and anguish that families already face in their challenging medical
journeys.
"Just as Wikipedia revolutionized how we think of the
encyclopedia, the Takeda commitment will help harness technology to
revolutionize access to care standards for rare disease," said
Marshall Summar, M.D., founding
director of the Children's National Rare Disease Institute and
chief of the Division of Genetics and Metabolism at the hospital.
"We are extremely grateful for Takeda and their support of our work
to unify rare disease research and real-world clinical experience
in a way that's never been done before."
"With a rare disease typically affecting fewer than 1 in 200,000
people2, there are unfortunately few standardized care
protocols in place once a diagnosis is confirmed," says
Tom Koutsavlis, M.D., Takeda head of
U.S. Medical Affairs. "We're pleased to be part of this important
effort with Children's National to help patients get faster access
to the optimized treatment for their particular rare disease
diagnosis, which is critical in helping them achieve better health.
In addition, this partnership underscores our commitment to driving
continuous innovation and personalized care for patients with rare
diseases."
The Children's National Rare Disease Institute is a
first-of-its-kind center focused exclusively on advancing the care
and treatment of children and adults with rare genetic diseases. It
is the first NORD Designated Clinical Center of Excellence and
provides a medical home for patients and families seeking the most
advanced care and expertise for rare genetic conditions that remain
largely unknown to the general medical community. Takeda's
commitment represents one of the institute's founding
partnerships.
Takeda aspires to transform the treatment of rare diseases in
immunology, gastroenterology, neurology, oncology, hematology,
metabolic and lysosomal storage disorders. These rare diseases can
have symptoms that vary widely and progress very differently from
person to person, which means that people affected by these
diseases are frequently misdiagnosed.
About Children's National Hospital
Children's National Hospital, based in Washington, D.C., celebrates 150 years of
pediatric care, research and commitment to community. Volunteers
opened the hospital in 1870 with 12 beds for children displaced
after the Civil War. Today, 150 years stronger, it is among the
nation's top 10 children's hospitals. It is ranked No.1
for newborn care for the fourth straight year and ranked
in all specialties evaluated by U.S. News & World Report.
Children's National is transforming pediatric medicine for all
children. In 2021, the Children's National Research &
Innovation Campus will open, the first in the nation dedicated to
pediatric research. Children's National has been designated three
times in a row as a Magnet® hospital, demonstrating
the highest standards of nursing and patient care delivery. This
pediatric academic health system offers expert care through a
convenient, community-based primary care
network and specialty care locations in the D.C.
metropolitan area, including the Maryland and Northern Virginia suburbs. Children's National
is home to the Children's National Research
Institute and Sheikh Zayed Institute for Pediatric
Surgical Innovation and is the nation's seventh-highest
NIH-funded children's hospital. It is recognized for its expertise
and innovation in pediatric care and as a strong voice for children
through advocacy at the local, regional and national levels.
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About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a
global, values-based, R&D-driven biopharmaceutical leader
headquartered in Japan, committed
to discover and deliver life-transforming treatments, guided by our
commitment to patients, our people and the planet. Takeda focuses
its R&D efforts on four therapeutic areas: Oncology, Rare
Genetic and Hematology, Neuroscience, and Gastroenterology (GI). We
also make targeted R&D investments in Plasma-Derived Therapies
and Vaccines. We are focusing on developing highly innovative
medicines that contribute to making a difference in people's lives
by advancing the frontier of new treatment options and leveraging
our enhanced collaborative R&D engine and capabilities to
create a robust, modality-diverse pipeline. Our employees are
committed to improving quality of life for patients and to working
with our partners in healthcare in approximately 80 countries.
For more information, visit https://www.takeda.com.
1 World Health Organization, Priority diseases and
reasons for inclusion, 6.19;
https://www.who.int/medicines/areas/priority_medicines/Ch6_19Rare.pdf
accessed 2/17/21
2 National Institutes of Health, Genetic and Rare
Diseases Information Center: rarediseases.info.nih.bov/diseases,
accessed 2/17/2021
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SOURCE Children's National Hospital