Top Three Challenges Facing Orphan Drug Candidates

May 17, 2017 9:00:00 AM / by John Kouten, CEO posted in drug development, rare diseases, orphan drugs, Precision medicine, FDA approval process


Prior to the passage of the 1983 Orphan Drug Act, just 34 drugs were approved to treat rare diseases, leaving small patients populations with few options for their rare diseases. That number has since skyrocketed with the implementation of the Orphan Drug Act. In 2016 alone, the FDA designated 333 drugs in development as orphans and approved 39 products, including both novel medicines and new orphan uses of approved drugs. Since the Orphan Drug Act took effect, The FDA has designated 3,600 orphan drugs and approved 552 orphan drugs.
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