Children’s National Hospital Rare Disease Institute and Takeda Partner to Standardize Care for Patients With Rare Diseases

The following is an excerpt from a press release from Takeda. Read the press release in its entirety here.

Children’s National Hospital and Takeda Pharmaceutical Company Limited announce 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.
“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.”
Read the press release in its entirety here.