Director's Messages

NCATS’ acting director highlights crimps in the translational pipeline and approaches for developing new treatments twice as fast over the next decade.

NCATS Acting Director Dr. Joni Rutter promotes Rare Disease Day at NIH and shares two key areas of rare diseases research that need to be addressed.

NCATS is committed to raising awareness about rare diseases and identifying treatments to address this urgent public health issue. The annual Rare Disease Day at NIH event held this year on March 1, offers an opportunity to advance this mission.

NCATS quickly pivoted many of its existing platforms, technologies and programs to address COVID-19 in March. The translational science approaches to COVID-19 similarly can speed solutions for many diseases and future pandemics.

New COVID-19 data integration projects show the power of data sharing to address a public health crisis like the COVID-19 pandemic. NCATS’ new online resources describe NCATS work that brings a translational science approach to addressing COVID-19.

Christopher P. Austin, M.D., shares how his encounter with an end-stage ALS patient shaped his understanding of the personal and public health benefits of translating scientific discoveries into new treatments for people with rare diseases

For millennia, physicians have diagnosed human ailments by categorizing symptoms and then treating patients according to these preformed categories. While this approach has been enormously valuable in allowing health care providers to effectively identify, understand and manage many diseases, every doctor knows that each patient’s disease is unique in presentation, course and response to treatment.

Officially, any disease that affects fewer than 200,000 people in the U.S. is classified as “rare,” and drugs that treat these diseases as “orphans.” Useful as these terms are, they can be misleading and even detrimental to efforts to understand, diagnose and treat people living with these diseases...

Anyone embarking on an unfamiliar journey will want a map to help plan needs, anticipate problem spots and formulate contingency plans. This is all the more true if the journey being contemplated is known to take many years, be fraught with hazards and rarely be completed successfully...

There are about 7,000 diseases officially defined as “rare,” or affecting fewer than 200,000 people in the U.S.; only a few hundred of these diseases have any approved treatment. Added together, these disorders are anything but rare: They affect 25 million people in the U.S., and approximately 50 percent of these patients are children...