Breakthrough status is reserved for new drugs and biological products intended for “rare” and “serious or life-threatening” diseases, with preference for products that are “genetically targeted.” The concept stems from informal discussions several years ago between stakeholders and agency officials about how to deal with “a new drug or drug combination for a severe illness that shows remarkable activity very early in clinical development,” says Jeff Allen, executive director of Washington, DC–based Friends of Cancer Research. These ideas were shared with influential members of the US Congress, who incorporated them into the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA), known as the Prescription Drug User Fee Act V (PDUFA V; Nat. Biotechnol. 30, 733–734, 2012).
The criteria for granting breakthrough status are not entirely evident, and the FDA does not grant such designation unless companies request it. Companies welcome the opportunity to collaborate more closely with the FDA. Alessandro Riva, global head of oncology development and medical affairs at Novartis in Basel, referring to LDK378, points out that breakthrough status “includes all of the fast-track program features, as well as more intensive FDA guidance.”
This is a preview of subscription content, access via your institution
