What Makes A “Breakthrough”?

 
• By Michael McCaughan

FDA is still not ready to draw any clear line defining what qualifies for the new “Breakthrough” therapy designation, but most successful applicants have presented data showing more than a 50% improvement versus standard therapy on some important endpoint.

There is not and probably never will be a bright line to define what qualifies for FDA’s “Breakthrough Therapy” designation, but one early rule of thumb is that a compound should show at least a 50% improvement over standard therapy on the primary endpoint used in early efficacy trials.

Center for Drug Evaluation & Research Director Janet Woodcock presented an analysis of the first two years of the “Breakthrough”...

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