The regulatory pathway for nearly every product developed to treat a rare disease begins and ends with FDA's Office of Orphan Product Development.
FDA Orphan Designations At Record Levels; Resources Need To Keep Up
The development of drugs and biologics for rare diseases has reached a regulatory milestone that underscores industry’s interest in the orphan drug market. FDA’s Office of Orphan Product Development is now granting one new orphan drug designation, on average, every day of the year. That activity underscores the success of the Orphan Drug Act, but creates a new problem: Finding the funding to support it.
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