What Makes A “Breakthrough”?
This article was originally published in RPM Report
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.
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FDA is building a case for enhanced funding to support the “Breakthrough” therapy program and at the same time is working to educate sponsors on how to use the new program effectively. The 21st Century Cures process floated a proposal for a new “Breakthrough” approval pathway – though that idea has been dropped. Still, it sets up an interesting discussion heading into 2017.
FDA topped 40 new molecular entity approvals in 2014, leaving no doubt that the agency’s drug review group is operating at peak efficiency. But it is still far from clear that drug development is out of the doldrums.
FDA’s second wave of Breakthrough Therapies will look markedly different from the initial flurry of approvals. After an early focus on oncology, FDA is reviewing a more diverse set of applications in the antiviral, vaccines and cardiovascular arenas. And there will be more “breaks” during development as sponsors start to request designations based on earlier data. What will Breakthrough look like in the third year of the program?