Woodcock: Breakthrough designation mostly about efficacy

FDA awards or denies breakthrough designation requests primarily based on convincing clinical evidence of substantial efficacy or lack thereof, Janet Woodcock said Friday at a conference organized by Friends of Cancer Research and the Brookings Institution. Woodcock is director of the agency's Center for Drug Evaluation and Research.

In a review of the breakthrough program's first two years, FDA found that a 50% reduction of a key risk -- for example, disease progression -- would suffice to merit a breakthrough designation, she said. ...