Rare Disease Institute Partners with Takeda to Standardize Care for Patients with Rare Diseases

Children’s National Hospital and Takeda Pharmaceutical Company Limited (“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 world. 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 people , 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.

For more information on how you can support rare disease research and care at Children's National Hospital, please contact Miriam Selman at [email protected] or 301-502-5827