Does The Orphan Drug Act Need A Tune-Up?

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

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