The FDA's "breakthrough" program has been one of the most popular initiatives from the agency in years, yet many industry watchers have been puzzling over the actual value of the new designation for speedy reviews of drugs. Johnson & Johnson ($JNJ) and Vertex Pharmaceuticals ($VRTX)--which are two of the early recipients of the status--provided details about their own positive experiences with their respective breakthrough drugs.
To be clear, many of the benefits of breakthrough status were available before the FDA began granting the coveted designation in January, giving drug developers an inside track to speed their R&D programs to market. Yet whereas before the agency could offer expedited paths to market with regulatory perks such as Accelerated Approval and Fast Track, Breakthrough Therapy status bundles the benefits of those programs and seems to afford developers a higher level of cooperation from FDA staff.
"This has had an enormous impact," Jay Siegel, who heads regulatory affairs for J&J, said, as quoted by Bloomberg. "There is a very proactive role on the part of the FDA. They'll pick up the phone and call us and say 'Have you thought of this way to do this faster? Have you thought about this problem?'"
Vertex President and CEO Dr. Jeffrey Leiden, whose company garnered breakthrough status for the cystic fibrosis compounds VX-809 and Kalydeco, agreed with Siegel. "It's a different kind of conversation," Leiden said at the briefing. "It's iterative. It's continuous. It's pick up the phone if you have a problem.'"
As Siegel told those gathered at a briefing in Washington, D.C., the breakthrough awards for J&J and Pharmacyclics's ($PCYC) blood-cancer contender ibrutinib have helped put the program two years ahead of schedule. If approved as expected later this year or in early 2014, ibrutinib could become a poster child for the new FDA status. Johnson & Johnson filed for approval of initial uses of the therapy earlier this month based on impressive results from Phase II studies, and the therapy is expected to be on its way to becoming a blockbuster before clinical studies of the therapy conclude.
FDA drug chief Dr. Janet Woodcock has made clear that breakthrough status isn't a rubber stamp for an approval. Through July 12, Reuters reported, the agency has awarded 24 breakthrough designations and rejected 18 of them out of 67 total pitches from drug developers. Inevitably, some of those programs will encounter problems during development.
Still, Woodcock and her colleagues at the agency have shown thus far that the status is more than ceremonial and could dramatically reduce timelines for qualifying R&D programs.
Special Report: Dr. Janet Woodcock - The 25 most influential people in biopharma today - 2013