Breakthrough therapy designation was introduced by the FDA in 2012 to expedite the drug development process for promising novel therapies for serious or life-threatening conditions. In 2013, of 28 breakthrough therapy designations publicly announced, 12 were for oncology indications, and the first breakthrough therapy designations to gain regulatory approval (obinutuzumab [Roche/Genentech’s Gazyva] and ibrutinib [Janssen Biotech/Pharmacyclics’ Imbruvica]) were for chronic lymphocytic leukemia (CLL). These designations have been awarded for both hematological and solid tumor types, including multiple myeloma, non-small-cell lung cancer, malignant melanoma, and breast cancer. Breakthrough therapy designation therefore appears set to become an important accolade in oncology drug development and, as such, has received close attention from drug developers. Because the breakthrough therapy designation program is aimed at quickening drug review times, therapies may achieve earlier regulatory approval compared with standard review, a key advantage in increasingly competitive and fragmented oncology markets.
The breakthrough therapy designation program potentially impacts drug developers planning for novel therapies; if an agent gains regulatory approval more quickly, considerations such as appropriate clinical trial design to secure favorable market access and optimal positioning in the treatment algorithm will need to be considered at an earlier stage of drug development.