Rare Disease Stats


  • US$262bn and 20% of total worldwide prescription sales by 2024*
  • Over 7,000 rare diseases identified 
  • 25M to 30M patients in the United States have a rare disease 
  • Average of 5 YEARS and 7 SPECIALISTS to accurately diagnose a child with a rare disease 
  • US$147,308 is the mean orphan drug cost to USA patients in 2017*





SOURCE: Evaluate Pharma Orphan Drug Report 2018