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A rare disease is defined as any illness

condition, syndrome, disease, or disorder

affecting fewer than 200,000 people in the U.S.

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THERE ARE

7,000

KNOWN RARE DISEASES

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IMPACTING

30 MILLION

AMERICANS

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THAT EQUALS

1 in 10

PEOPLE AFFECTED

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and this is the

1 PLACE

for ALL effected to come

for TOOLS,

RESOURCES,

and SUPPORT

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  • 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

The content of this report has been obtained through government: FDA’s Orphan Drug Product designation database, the National Institutes of Health (NIH), and Institute of Medicine (IOM) and industry sources based on the latest information. Report current as of 6/14/2016.

  1. FDA Office of Orphan Products Development (OOPD).
  2. “Rare Diseases and Orphan Products: Accelerating Research and Development” Committee on Accelerating Rare Diseases Research and Orphan Product Development Board on Health Sciences Policy.
  3. U.S. FDA Novel New Drug Summaries (2011-2015); calculated average from each report’s data.
  4. Frattarelli, D.A., et al., Off-label use of drugs in children. Pediatrics, 2014. 133(3): p. 563-7.
  5. Memorial Sloan Kettering Cancer Center’s Cycle for Survival website;  https://www.cycleforsurvival.org/about-cycle-survival ; June 2016.
  6. The Financial Times Limited 2016;  “Private foundations: Searching for the sweet spot” by Sarah Murray, 6/24/2016, http://www.ft.com/cms/s/0/57c46446-2d51-11e6-bf8d-26294ad519fc.html#axzz4DjtDphuB.
  7. NORD Membership Survey; taken 2014.

©2016 NORD. All rights reserved. NORD is a registered trademark of National Organization for Rare Disorders. NORD is a 501(c)(3) charity.  NRD-1008

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

The content of this report has been obtained through government: FDA’s Orphan Drug Product designation database, the National Institutes of Health (NIH), and Institute of Medicine (IOM) and industry sources based on the latest information. Report current as of 6/14/2016.

  1. FDA Office of Orphan Products Development (OOPD).
  2. “Rare Diseases and Orphan Products: Accelerating Research and Development” Committee on Accelerating Rare Diseases Research and Orphan Product Development Board on Health Sciences Policy.
  3. U.S. FDA Novel New Drug Summaries (2011-2015); calculated average from each report’s data.
  4. Frattarelli, D.A., et al., Off-label use of drugs in children. Pediatrics, 2014. 133(3): p. 563-7.
  5. Memorial Sloan Kettering Cancer Center’s Cycle for Survival website;  https://www.cycleforsurvival.org/about-cycle-survival ; June 2016.
  6. The Financial Times Limited 2016;  “Private foundations: Searching for the sweet spot” by Sarah Murray, 6/24/2016, http://www.ft.com/cms/s/0/57c46446-2d51-11e6-bf8d-26294ad519fc.html#axzz4DjtDphuB.
  7. NORD Membership Survey; taken 2014.

©2016 NORD. All rights reserved. NORD is a registered trademark of National Organization for Rare Disorders. NORD is a 501(c)(3) charity.  NRD-1008

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