Breakthrough therapy: Difference between revisions

Critics have said that the name is misleading and provides companies that obtain a breakthrough designation for a drug candidate with a marketing advantage that may be undeserved.<ref name="JAMAIM">{{cite journal| title =Editor's Note: Would a Breakthrough Therapy by Any Other Name Be as Promising? | journal =JAMA Intern. Med. | date =21 September 2015 | author =Joseph S. Ross | author2 =Rita F. Redberg| volume =175| issue = 11| pages =1858–9| doi =10.1001/jamainternmed.2015.5311| pmid =26390064| pmc =| doi-access =free}}</ref><ref name="NEJM">{{cite journal| title =The FDA Breakthrough Drug Designation: Four Years of Experience | journal =New England Journal of Medicine | date =12 April 2018 | author =Jonathan J. Darrow | author2 =Jerry Avorn| author3 =Aaron S. Kesselheim |volume =378| issue = 15| pages =1444–53| doi =10.1056/NEJMhpr1713338| pmid =29641970| pmc =}}</ref> The FDA acknowledges that the name "breakthrough therapy" may be misleading. It was never meant to imply that these drugs are actually “breakthroughs,” and it does not ensure that they will provide clinical benefit, but still critics complain that they are based on preliminary evidence, including changes in surrogate markers such as laboratory measurements, that often don't reflect "meaningful clinical benefit."<ref name=healthnews />

The FDA guidance states: "Not all products designated as breakthrough therapies ultimately will be shown to have the substantial improvement over available therapies suggested by the preliminary clinical evidence at the time of designation. If the designation is no longer supported by subsequent data, FDA may rescind the designation."<ref>{{cite web|title=Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics|url=https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM358301.pdf|publisher=FDA|page=10|date=May 2014}}</ref>

 

==See also==