Rare Disease Drug Development [Video]

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.

Check out the report: https://www.gao.gov/products/gao-25-106774

Get transcript: https://www.gao.gov/assets/880/873051.txt

Learn more about GAO: gao.gov/about
Check out the WatchBlog: gao.gov/blog
Check out our Podcasts: gao.gov/podcast

Follow GAO on Social Media:
Twitter.com/usgao
Instagram.com/usgao
LinkedIn.com/company/us-government
YouTube.com/usgao
Facebook.com/usgao
Flicker.com/photos/usgao

General Information: The U.S. Government Accountability Office (GAO) is an independent, nonpartisan agency that works for Congress. Often called the “congressional watchdog,” GAO investigates how the federal government spends taxpayer dollars.

Mission: Our Mission is to support the Congress in meeting its constitutional responsibilities and to help improve the performance and ensure the accountability of the federal government for the benefit of the American people. We provide Congress with timely information that is objective, fact-based, nonpartisan, non-ideological, fair, and balanced.

Disclaimer: The U.S. GAO does not promote or endorse any non-Government or commercial content appearing on this page.

#GovernmnentAccountabilityOffice #CongressionalWatchdog #USGAO