Time to raise the bar: Transition rate of phase 1 programs on anticancer drugs

Anticancer drug research and development is a high-risk endeavor. Among investigational drugs with oncology as the lead indications that entered phase 1 trials between 2003 and 2011 (n = 489), only one in eight (13.2%) of phase 1 anticancer drugs were eventually approved (Hay et al., 2014). The recent decade has witnessed a shift of focus in anticancer drug development from cytotoxic drugs to molecular targeted agents (MTAs) and immuno-oncology therapies. Responding to this shift, the development paradigm and regulatory process for investigational anticancer drugs also underwent significant changes.