“Breakthrough Therapies” Deserve Skepticism, Researchers Say
This article was originally published in The Pink Sheet Daily
Although drugs on the FDASIA-created expedited pathway may promise more than they can deliver, it will be difficult to reduce demand for such treatments awarded the designation, Boston researchers predict in a NEJM piece.
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Agency cites availability of other recently approved therapies in notifying Merck of intent to rescind the designation for grazoprevir/elbasvir in hepatitis C. Sponsor will seek a meeting with FDA to make its case for retaining breakthrough status.
FDA retains the right to pull a breakthrough designation if subsequent data do not back up the promise seen in early clinical results, but a few failures are not expected to unduly hurt the expedited regulatory program.
Dartmouth researchers say that although FDA recommendations can strengthen study designs and improve outcome measures, sponsors often don’t follow agency’s advice or don’t even seek input in Phase III design.