Perspective: A path forward for rare disease policy

The rare disease policy calendar in Washington has been packed. Highlights have included an advisory committee meeting for a Barth syndrome treatment from Stealth BioTherapeutics, a meeting about FDA’s Rare Disease Innovation Hub convened by the Reagan-Udall Foundation, the NORD Breakthrough Summit, BIO’s Patient & Health Summit, and a Haystack Project congressional briefing. These all happened in the past two weeks, and I’ve probably left a few out.

Conversations on and off the podiums suggest rare disease policy in the U.S. has reached a crossroads, buffeted by two opposing winds. ...