If You’ve Got a Real Breakthrough, the FDA Wants To Talk
Summary
The company was conservative in its statement, saying that the implications of the action “cannot be determined at this time.” Vertex CEO Jeff Leiden shared a little more with Bloomberg News, saying, “it’s an opportunity to have a different set of interactions and collaborative discussions” with regulators.The FDA was instructed to prioritize reviews of such “breakthrough” therapies through the Food and Drug Administration Safety and Innovation Act (FDASIA) that was signed into law in July.Instead, the company could get a dedicated, cross-disciplinary project team at FDA charged with finding ways to shorten and shrink the clinical trials required to demonstrate that beloranib deserves a place on the U.S. market.The company would then be able to work closely, in collaboration with FDA reviewers and statisticians, to design trials that reflect the latest scientific understanding of the drug and the targeted patient population, Hughes says.FDA history suggests it’s constantly on a swinging pendulum, moving toward a cautious position when safety controversies flare up, and then toward a more permissive stance when it is accused of stalling important new innovations for patients.