Negative Phase 3 Randomized Controlled Trials: Why Cancer Drugs Fail The Last Barrier?

Among 16 negative Phase 3 RCTs of new cancer drugs published in major journals in 2016, only 3 were supported by a previous positive Phase 2 trial, while 3 proceeded despite a negative Phase 2, 5 were based on inconclusive Phase 2 data, and 1 had no prior Phase 2 at all. The authors argue that institutional review boards and regulatory authorities should discourage Phase 3 RCTs lacking positive Phase 2 data, and that prospective criteria for defining Phase 2 success should be established to prevent the waste of substantial human and financial resources on trials with a low probability of success.