Because Every Person With a Disease Deserves Treatment Options
Join our mission to protect bipartisan incentives that have made rare disease therapies a reality.
NOW LAW
The ORPHAN Cures Act
Congress passed critical legislation to protect research into treatments for the millions of Americans currently suffering from rare diseases and rare cancers. The Task Force thanks bipartisan leaders in the House and Senate for their work to make ORPHAN Cures law.
Rare Disease at-a-Glance
Get a quick glimpse into the staggering numbers that define the world of rare diseases and orphan drugs.
Rare diseases
There are approximately 10,000 different rare diseases.
have an approved treatment
Less than 10% of rare diseases have an FDA approved treatment available.
Americans
Nearly 30 million people in the U.S. have a rare disease.
THE SCALE OF THE PROBLEM
The Urgency of Rare Diseases
An estimated 30 million Americans have a rare disease, which is a disease affecting 200,000 Americans or less. Today, less than 10 percent of Americans with a rare disease have access to an FDA-approved treatment. That means most Americans with a rare disease have no treatment specifically designed to treat their disease.
According to the FDA:
“Drug…development in rare diseases is challenging for many reasons, including the complex biology and the lack of understanding of the natural history of many rare diseases. The inherently small population of patients with a rare disease can also make conducting clinical trials difficult.”
Because of the challenges in rare disease research and development, Congress passed the Orphan Drug Act in 1983 to provide incentives that increased rare disease research. Those incentives have resulted in a 1,576% increase in the number of FDA-approved orphan drugs – from 38 to 600+ therapies to treat more than 1,100 indications. However, a new policy in the Medicare Drug Price Negotiation Program affects those incentives, which are still urgently needed to foster rare disease treatment innovation and address critical unmet needs.
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Watch: The Importance of Protecting Rare Disease Innovation
Learn about the ORPHAN Cures Act, a crucial bipartisan initiative designed to support innovation in rare disease treatments. Hear from experts and advocates Paul Kim, Arya Singh, and Lisa Schlager as they explain why this law is vital to encouraging drug innovation, expanding treatment options, and offering hope to rare disease patients.
