Because Every Person With a Disease Deserves Treatment Options

Join our mission to protect bipartisan incentives that have made rare disease therapies a reality.

The ORPHAN Cures Act

Bipartisan, federal legislation to protect research into treatments for the millions of Americans currently suffering from rare diseases and rare cancers.

Rare Disease at-a-Glance

Get a quick glimpse into the staggering numbers that define the world of rare diseases and orphan drugs.
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Rare diseases

There are approximately 10,000 different rare diseases.

< 0 %

have an approved treatment

Less than 10% of rare diseases have an FDA approved treatment available.

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Americans

Nearly 30 million people in the U.S. have a rare disease.

Watch: Drugs Treating Rare Disease & Medicare Drug Price Negotiation

Paul Kim, an attorney with more than 30 years of experience in federal health policy, including at the FDA, explains the problem with the narrow exclusion for orphan drugs in the Medicare Drug Price Negotiation Program.

The overly narrow exclusion undermines longstanding incentives for medical innovation to treat rare diseases and thereby jeopardizes access to future treatments for persons living with rare diseases.