As part of the 2012 US Food and Drug Administration (FDA) Safety and Innovation Act, Section 9021 created a new regulatory review designation—breakthrough therapy—that would expedite the development and FDA review of as-yet unapproved drugs and biologic agents intended to treat serious or life-threatening diseases and for which preliminary clinical evidence indicates that the drugs may offer substantial improvement over existing therapies. This legislation was likely enacted to promote innovative drug development and to respond to critics of the FDA regulatory review process, who criticize it as being slow despite research demonstrating the opposite.2 Reflecting these intentions, the term developed by Congress and adopted by the FDA for this new review designation—breakthrough therapy—is aspirational.
Ross JS, Redberg RF. Would a Breakthrough Therapy by Any Other Name Be as Promising?. JAMA Intern Med. 2015;175(11):1858-1859. doi:10.1001/jamainternmed.2015.5311