In an opinion piece in the New England Journal of Medicine, Food and Drug Administration officials describe the breakthrough therapy designation authorized by the Food and Drug Administration Safety and Innovation Act (FDASIA) as “the initiation of a new paradigm for investigational drugs undergoing development in a setting of extensive mechanistic understanding of disease pathogenesis.”

The breakthrough therapy designation can be granted to drugs for serious or life-threatening diseases or conditions where there is preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The designation has been granted to several investigational therapies for cancer, and the designation has resulted in an expedited development and review process for some products.

At policy roundtable meetings convened by NCCS, the breakthrough therapy designation has been discussed by FDA officials, drug developers, and patient advocates. The conversations have focused on the preliminary clinical evidence necessary to trigger a breakthrough therapy designation and the benefits that flow to developers and patients from such designation.