Clinical Research on Rare Diseases

Clinical investigators in rare disease research regularly face challenges distinct to the study of uncommon disorders. Rare diseases often provide opportunities to study human physiology and biomedical science from distinctive perspectives. It is estimated that over 7000 rare diseases affect an estimated 25-30 million people with a rare disease (8-12% of population). The Orphan Drug Act was approved in 1983 to provide funds that encourage developing optimistic drugs to treat, prevent, or diagnose rare diseases or conditions affecting less than 200,000 persons. The scientific methodology and logistics of clinical research for rare diseases have changed drastically in the past two decades. Understanding pathophysiology of the disease has been increased and many new treatments have been developed.

  • Orphan products clinical Trials Grants Program
  • Biomedical
  • Research on new drugs

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