Rare diseases are often chronically debilitating and sometimes life threatening. They each affect fewer than 200,000 people nationwide. Rare diseases may seem like a small corner of health care, but together, they affect nearly 10 percent of the US population. That’s about equal to the number of Americans diagnosed with diabetes. Yet most rare diseases are not studied enough. As a result, patients with rare diseases often face years of testing or ineffective treatments.

PCORI recognizes the special challenges that rare diseases, which often are understudied, pose. Learn more about our growing research portfolio.


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