Vanderbilt University Medical Center has collaborated with Children’s National Hospital in Washington, D.C., to develop the Rare Disease Clinical Activity Protocol Program, or RareCAP, a growing online repository of clinical protocols designed to offer quick, practical guidance on the care of patients with rare diseases.
The repository, now approaching 100 protocols, is intended to save physicians the work of digging through journal articles and other sources to determine the best approach to caring for patients with disorders that most physicians seldom encounter.
“If you’re in in the emergency department and you’ve never seen someone with a particular rare disease, our goal is that you can come to this platform and find what you need to know immediately to treat this person in an acute care setting,” said Colleen Lawrence, PhD, an assistant director at the Vanderbilt Institute for Clinical and Translational Research (VICTR) and the project manager for RareCAP.
Jennifer Micham, PhD, RareCAP curator at VUMC, adds, “An ED physician can go right to seeing the topics under emergency management and zero in on exactly what he or she needs to know. Here is a site that is easy to use, simple to access, and at your fingertips.”
The protocols are written in bullet-point format and use language geared toward busy clinicians. In addition to guidance for the acute care setting, the protocols cover chronic management and include links to more in-depth open-source information from the web. Though designed with clinicians primarily in mind, patients and their families will also find the repository useful.
The protocols are being written by volunteer contributors whose work is vetted by Micham and a RareCAP medical advisory board. Contributors to the platform, as they read protocols written by other contributors, can add comments and citations and suggest changes.
“The message to the VUMC community,” Lawrence said, “is to use the platform if you need it; consider contributing your expertise; and, for patients with rare diseases, encourage your physician to become a contributor.”
Globally, rare diseases, often defined as those affecting fewer than 1 in 200,000 people, are estimated to affect more than 300 million people. There are an estimated 7,000 to 8,000 rare diseases.
RareCAP was conceived by Marshall Summar, MD, formerly with Children’s National, and a member of the VUMC faculty from 1990 to 2010. RareCAP is funded by a grant to Children’s National from Takeda Pharmaceuticals, with headquarters in Tokyo. The medical advisory board is led by Debra Regier, MD, chief of genetics and metabolism at Children’s National.
For more information or to become a RareCAP contributor, visit the project website.
