Drug Firms Praise New “Breakthrough” FDA Pathway

Johnson & Johnson and Vertex Pharmaceuticals, two companies whose drugs have received “breakthrough therapy” designation, have spoken out in support of the FDA’s new breakthrough program, which could shave years off of the approval process.



Receiving breakthrough status has meant that J&J and Pharmacyclic’s blood-cancer therapy ibrutinib is now two years ahead of schedule, according to Jay Seigel, head of regulatory affairs for J&J.

Ibrutinib, if approved, will become the first in a class of oral medicines targeting a protein called Bruton’s tyrosine kinase, and after impressive results from Phase II studies, the therapy is on track for blockbuster status even before all clinical studies of the drug have concluded. Siegel claimed in a briefing in Washington that the new regulatory program “has had an enormous impact” on the approval process, saying that “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?’”

Dr Jeffrey Leiden, CEO of Vertex Pharmaceuticals, also spoke at the briefing; Vertex’s cystic fibrosis compounds Kalydeco and VX-809 both received breakthrough designation. Leiden agreed with Siegel on the merit of the new program, describing it as “a different kind of conversation. It’s iterative, it’s continuous. It’s pick up the phone if you have a problem.” Siegel commented that being able to talk in real time to FDA made the approval process “immeasurably smoother.”

The Director of the FDA’s drugs division Janet Woodcock said in the discussion that the new pathway was designed to prioritise new science, especially targeted therapies that are linked to certain genetic mutations. However she also pointed out that getting onto the expedited pathway did not mean approval was guaranteed. Reuters reports that as of the 12th of July the agency has received a total of 67 applications, awarding 24 breakthrough designations and rejecting 18.

Despite the praise given to the program, Siegel said at the briefing that there were still issues to be resolved. One of these was the uncertainty around whether foreign regulators would approve products which had undergone fewer clinical trials due to their breakthrough designation. “Our hope is that foreign regulators will catch up,” Siegel said. Another issue still to be worked out was “how to bring payors on board,” as insurers may not be willing to pay for drugs with less clinical trial data to back them up.


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