Because Every Person With a Disease Deserves Treatment Options
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
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
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.
Get Involved
Support the Save Rare Treatments Task Force and our advocacy efforts.
Resources
Read more about relevant research and insights related to The ORPHAN Cures Act and rare diseases.