Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age.
The effort is modeled on that of the Cystic Fibrosis Foundation in establishing CF centers of excellence to improve patient care.
“Given the increasing survival rates of patients with rare diseases and our enhanced ability to diagnose them, the handful of patients we started with has become an entire population,” Dr. Marshall Summar, the institute’s director, said in an interview with BioNews Services.
“In the 1980s, someone born with Down syndrome survived an average 20 years; now it’s in the 50s. Survival for cystic fibrosis in the 1980s was in the teens, now it’s 30 to 40 years,” said Summar, chief of genetics and metabolism at Children’s National as well as NORD’s chairman of the board. “As we bring in new therapies and diagnose better, we’ve increased the cohort of genetic-based patients.”
Summar, interviewed on the sidelines of NORD’s Oct. 16-17 Rare Summit in Washington, said that in some ways, the concept of such an institute goes back to where medical genetics started.
“Now it’s spread across the entire medical spectrum,” he explained. “We have created this group of patients who need chronic care and who use rare disease therapies. We have patients in their 70s, 80s, and even 90s as well as newborns — and some who haven’t even been born yet.”
The Rare Disease Institute currently operates out of Children’s National itself, though it recently acquired 12 acres of land at the former Walter Reed Army Medical Center along Georgia Avenue in northwest Washington. The property, which was vacated in 2011, includes the Armed Forces Institute of Pathology building, a large parking lot, and a 300-seat lecture hall.
Summar said he expects to be operating at the new location by fall 2018.
“This is a 50-year-old-plus building, and we want to renovate it,” he said, estimating that Children’s National will invest “several hundred million dollars” into the project.
At its core, he said, are 14 physicians — all clinical geneticists — as well as 15 genetic counselors, two biochemical nutritionists and four clinical care coordinators. The Rare Disease Institute already treats an estimated 8,000 patients a year. According to a Children’s National press release, the testing and development of new therapies will be a key institute focus.
“The geneticists at Children’s National will use systematic data models that track patient outcomes and treatment regimens to create an ever-expanding clinical knowledge base,” it said. “The institute also intends to eliminate patient care gaps by providing training specific to rare diseases for clinicians, genetic counselors, nurses, researchers and allied health professionals.”