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
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…
September 1, 2023
Yesterday the Federal Trade Commission (FTC) announced the decision to settle its Amgen-Horizon merger challenge. The settlement will give the Commission the relief it needs to protect competition, while allowing rare disease patients to enjoy the deal's pro-competitive benefits without delay. BIO…
June 30, 2023
The Centers for Medicare & Medicaid Services (CMS) today released its guidance for the Drug Price Negotiation Program (Negotiation Program) under the Inflation Reduction Act of 2022 (IRA). John Murphy, BIO’s Chief Policy Officer, made the following statement:  "As we have stated in the…
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.