Rare Diseases & Orphan Drugs

Approximately 7,000 rare diseases are known to exist, and approximately 30 new ones are identified each year. Also known as orphan diseases, they are defined as those which affect fewer than 200,000 patients in America. And while it might be true that each rare disease only affects a small number of patients, collectively, millions of lives are depending on the development of innovative cures and medicines.

Press Releases
July 17, 2024
July 17, 2024 – BIO President and CEO John F. Crowley today released the following statement after the U.S. Food & Drug Administration (FDA) announced a plan to establish a Rare Disease Innovation Hub that will utilize new and collaborative approaches to expedite the development and approval of…
June 20, 2024
BIO President and CEO John F. Crowley today released the following statement after a bipartisan group of Senators, Sen. Bob Casey (D-PA), Sen. Markwayne Mullin (R-OK), Sen. Sherrod Brown (D-OH), and Sen. Susan Collins (R-ME), introduced the Creating Hope Reauthorization Act to reauthorize the Rare…
February 15, 2024
The Creating Hope Reauthorization Act of 2024 was introduced in the House of Representatives this week. BIO CEO and President, Rachel King, made the following statement following the bill's introduction: "BIO strongly supports the House introduction of the Creating Hope Reauthorization…
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