WHAT IS Orphan Disease?

In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people. The total number of Americans living with a rare disease is estimated at between 25-30 million across as many as 7,000 rare diseases. This estimate has been used by the rare disease community for several decades to highlight that while individual diseases may be rare, the total number of people with a rare disease is large. 


eStudySite has participated in several investigative clinical trials of various Orphan Diseases including:

  • Alport Syndrome
  • Familial Amyloidotic Polyneuropathy
  • Hereditary Hemochromatosis
  • Beta-thalassemia
  • Primary Hyperoxaluria Type 1

Qualified subjects participating in a trial receive study-related medical evaluations and the investigative study medication at no cost and may be eligible to receive financial compensation for time and travel cost.


If you know someone suffering from Orphan Disease who may want to learn more or volunteer, you can let us know by clicking the button below or visit the Rare Diseases Clinical Research Network (RDCRN) at: https://ncats.nih.gov/rdcrn


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