1. Pink Sheet

Does The Orphan Drug Act Need A Tune-Up?

 
• By Derrick Gingery

A better description of the US FDA’s regulatory flexibility may be necessary, but tinkering with the foundational law for rare disease drug development could open the door for more substantial and unwanted changes.

Rare Disease Day webinar screenshot
Frank Sasinowski, second from left, discusses the Orphan Drug Act during a Rare Disease Day event hosted by The Hill. • Source: Screenshot of Event Livestream

Rare disease advocates are wondering whether the Orphan Drug Act should be amended to better describe regulatory flexibility, a concept that is well-known in the abstract, but more difficult for the US Food and Drug Administration to define and apply.

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