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What Is A Rare Disease?

In the U.S., any disease affecting fewer than 200,000 people is considered rare. This definition comes from the Orphan Drug Act of 1983 and is slightly different from the definition used in Europe. There are nearly 7,000 rare diseases affecting nearly 30 million Americans. In other words, almost one in ten Americans are suffering from rare diseases.

Besides dealing with their specific medical problems, people with rare diseases struggle to get a proper diagnosis, find information, and get treatment. The rarity of their conditions makes medical research more difficult.

  • A rare disease is defined as any illness, condition, syndrome, disease or disorder affecting fewer than 200,000 people in the US.1
  • There are more than 7,000 known rare diseases impacting 30 Million Americans, that equals 1 in 10 people affected.
  • More than 15 Million are children.1
  • 80% are genetic conditions.2
  • 95% of rare diseases are without an FDA approved treatment or therapy.
  • Those 5% with FDA treatment still struggle with accessing it due to high costs and variability.
  • Patients with rare diseases are frequently misdiagnosed or undiagnosed.
  • Many rare diseases result in premature death of infants & young children or are fatal in early adulthood.2
  • Families & private foundations provide about 3% of ALL medical research funding in the U.S.6
  • Recently, more than 1/3 of all NEW drugs approved by FDA have been for rare diseases; and 47% in 2015.3
  • 90% of healthcare providers must treat the majority of rare disease patients with Non-FDA approved drugs.4
  • There are 100 types of cancer. Approximately 50% of people with cancer are battling a rare cancer. Rare cancers include brain, pancreatic, ovarian, thyroid, and stomach cancers; leukemia and lymphoma; and all pediatric cancers.5
  • There are more Americans who live with a rare disease than ALL of those who have either HIV, Heart Disease or Stroke.
  • 75% of NORD Members are actively funding rare disease medical research.7