12 April 2016 - This survey study examines how well physicians understand the US FDA’s statutory definition of a “breakthrough” therapy, and whether the term breakthrough affects their perceptions of a drug’s efficacy.

Before US patients can use new prescription drugs, the US FDA reviews the clinical trial results to confirm that benefits outweigh harms for the indication. Numerous pathways expedite drug development and approval for serious or life-threatening conditions. For example, since 2012, the FDA can designate a drug as a “breakthrough therapy” if preliminary clinical evidence—such as an improvement in a pharmacodynamic biomarker—suggests an advantage over existing options.

Through April 2015, the FDA designated 76 “breakthrough” drugs, and the term is routinely used in press releases and prescribing resources.

For more details, go to: http://jama.jamanetwork.com/article.aspx?articleID=2512772