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FDA Orphan Designations At Record Levels; Resources Need To Keep Up

 
• By Kate Rawson

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.

The regulatory pathway for nearly every product developed to treat a rare disease begins and ends with the Food & Drug Administration’s Office of Orphan Product Development.

That office reviews requests from sponsors for orphan drug designation at the front end of development, and – should the...

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