Breakthrough therapy-designated oncology drugs: are they rightfully criticized?

The US Food and Drug Administration (FDA) has four expedited programs, including the breakthrough therapy designation (BTD). Recently, this program has been criticized. In this feature, we determine whether BTD oncology drugs were truly a breakthrough, based on the outcome of a validated instrument to measure clinical benefit. We find that only a few drugs were likely a breakthrough, indicating that the success rate of the BTD program is somewhat low. Despite this, we believe that programs for fast drug approval do have a place in the current regulatory practice and that the necessary efforts for their improvement should be further explored, especially considering the remaining unmet medical need for patients with cancer.