This NIH center has brought 44 drugs for rare diseases to market — and isn't slowing down

In just the 12 years since its establishment, the National Center for Advancing Translational Sciences has brought 44 FDA-approved drugs for rare diseases to the market — and it shows no signs of slowing down.

NCATS as one of the 27 centers making up the National Institutes of Health uniquely focuses on research and treatments for 10,000 diseases, especially those that are rare or have unmet needs. It "has played a pivotal role in tackling ongoing challenges in research to deliver new and innovative treatments to patients faster," according to a description from Manatt Health, which hosted a webinar on the center March 15.

During the webinar, NCATS Director Joni Rutter, PhD, spoke to the need to innovate even faster and more efficiently during an age of rapidly evolving health needs. Extensive research and science are understandably involved, but a key component to accelerating drug discovery, especially for rare diseases, is involving key stakeholders. 

"NCATS has played a really critical role for the rare disease community by focusing on what rare diseases have in common," Dr. Rutter said. "It's important to understand we have more than 7,000 rare diseases and less than 600 currently have an FDA approved treatment … and by really fostering partnerships across all stakeholders in the rare disease space … we have huge potential for the whole to be greater than the sum of its parts."

Two approaches Dr. Rutter said the center uses for accelerating drug development are its use of artificial intelligence and other technology to help find drugs and determine the best starting point to begin to develop new drug candidates and speed up chemistry. It also uses drug repurposing to cut development time down from 10 to 15 years to just one or two. 

"We're applying these tools to enable preclinical drug development for things like pain and addiction, cancer metastases, ALS and many more diseases as well," Dr. Rutter said. "These are the kinds of approaches that create these efficiencies and advance translational science more quickly."

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