FDA Commissioner Robert Califf highlights need for ‘creative approaches’ for rare disease therapies

The FDA’s top of­fi­cials con­tin­ue to call for new ways to reg­u­late up-and-com­ing med­i­cines for rare and ul­tra-rare dis­eases.

“We’re about to see a tsuna­mi of ther­a­pies for a rare and ul­tra rare dis­ease, and I don’t think any of us think the cur­rent path­ways are op­ti­mal,” FDA com­mis­sion­er Robert Califf said dur­ing a Q&A at the Com­pas­sion­ate Use and Preap­proval Ac­cess con­fer­ence host­ed by New York Uni­ver­si­ty on Tues­day.

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