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
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…
December 7, 2023
Today, the Biotechnology Innovation Organization released a poll that shows voters overwhelmingly support the ORPHAN Cures Act, a recently introduced bill that would incentivize rare disease drug development.   The ORPHAN Cures Act would fix a provision in the…
September 18, 2023
Representatives John Joyce, M.D. (R-PA) and Wiley Nickel (D-NC) today introduced the Optimizing Research Progress Hope And New Cures (ORPHAN Cures Act). BIO President and CEO, Rachel King, made the following remarks: "This legislation is a welcome and bipartisan fix to the Inflation Reduction Act…
Join BIOAction
Become an Advocate for Biotechnology

Sign-up to join BIOAction, BIO's grassroots advocacy program. You will be notified about relevant policy issues where we need you to contact your lawmaker.