Rare Disease Drug Development

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.