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 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…
August 12, 2021
President Joe Biden today announced a slew of drug pricing reforms, including changes that would radically alter the widely successful Medicare program for seniors and people with disabilities. Rich Masters, BIO’s Chief Public Affairs and Advocacy Officer, made the following remarks about the plan…
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