National Organization for Rare Disorders(NORD)

About the National Organization for Rare Disorders

NORD was founded in 1983 by Abbey Meyers and a coalition of rare disease patient advocates who lobbied Congress to pass the Orphan Drug Act — legislation that provided incentives for pharmaceutical companies to develop treatments for rare diseases that would otherwise not be commercially viable. The Orphan Drug Act has enabled the approval of over 1,000 rare disease treatments since 1983.

NORD is a federation of more than 300 patient organisation members covering hundreds of rare diseases, and a resource for patients with conditions that have no dedicated organisation.

What they do

Orphan drug advocacy: NORD continues to monitor and advocate around the Orphan Drug Act, fighting efforts to weaken the Act while also addressing concerns about excessive pricing of orphan drugs. The balance between incentivising development and ensuring access is a central tension in NORD's work.

FDA engagement: NORD advocates for accelerated approval pathways and patient-centred drug development for rare diseases.

Patient assistance: NORD's Patient Assistance Programmes help uninsured and underinsured rare disease patients access medications they cannot afford.

Information: NORD's Rare Disease Database contains information on over 1,200 rare disorders, written in plain language, covering symptoms, causes, diagnosis, treatment, and organisations.

Key programs and resources

• Rare Disease Database: Plain language disease information for over 1,200 conditions
• Patient Assistance Programmes: Medication access support
• OrphanHub: Information on orphan designation and rare disease research
• Annual Rare Disease Summit: Policy conference bringing together patient advocates, researchers, and policymakers

Who they serve

People with any of the approximately 7,000 known rare diseases, their families, and healthcare providers.

Why it matters

For people with rare diseases — conditions that may have only dozens or hundreds of known patients in the US — NORD often provides the only accessible information and advocacy infrastructure. The Orphan Drug Act that NORD helped pass has enabled life-changing treatments for conditions that would otherwise have been neglected by the pharmaceutical industry.