From the U.S. Government Accountability Office, www.gao.gov

Transcript for: Rare Disease Drug Development

Description: Nearly one in 10 Americans have a rare disease, which is
typically defined as any condition affecting fewer than 200,000 people
in the United States. There are up to 10,000 rare diseases, however,
only 5 percent of rare diseases have FDA-approved treatments. GAO found
that FDA has 18 rare disease-specific programs - most initiated since
2019 - intended to help address complexities common to rare disease drug
development. FDA is also implementing a Rare Disease Innovation Hub
intended to expedite the development and approval of safe and effective
rare disease drugs. 

Related GAO Work: GAO-25-106774, Rare Disease Drugs: FDA Has Steps
Underway to Strengthen Coordination of Activities Supporting Drug
Development

Released: November 2024

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Millions of Americans -- many of them children -- have a rare disease.
But only about 5% of the nearly 10,000 identified rare diseases have
Food and Drug Administration-approved treatments. 

Developing rare disease drugs is challenging. The diseases aren't well
understood, and small patient populations can limit clinical trial
results. FDA has 18 rare disease programs to help address such
complexities.

FDA is also implementing a new Rare Disease Innovation Hub intended to
expedite the development and approval of safe and effective rare disease
drugs. One goal of the Hub is to improve coordination between two agency
centers that review drug applications.

Find Out More at GAO.gov

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For more info, check out our report GAO-25-106774 at: GAO.gov