We are living in an exciting era in which the gold standard in clinical development of antitumor drugs is changing at an astonishing pace, after roughly half century of stagnation and relative inefficacy in providing more and better drugs to our patients. New study designs have emerged, such as basket and umbrella clinical trials, as well as dynamically evolving multiple-arm phase 1b/2 trials with hundreds of recruited patients and specific exploratory cohorts of different tumor types and pivotal trials with associated sub-studies. These novel designs coupled with the recent awareness and sensitization of regulatory agencies in improving the current system inefficacy are changing the paradigm of clinical cancer research. As opposed to a classically based succession of phase 1 trials for dose finding and administration schedule, followed by phase 2 trials to check for antitumor activity and, lastly, phase 3 trials in which the drug was compared against the standard therapy for superiority and drug approval, it is now becoming a reality that the typical objectives, in early- and late-phase investigation, are now obtained in a time continuum during the course of the initial trials that seek to be much more informative.
However, this revolution is insufficient. We cannot conform in our objective of developing new molecular entities with different mechanisms of action, which we aim to be better tolerated and more active, if we are not able to reduce costs, development time, and drug accessibility to our cancer patients. Therefore, we still have the need for a disruptive change, which would shift the current focus to the free, informed decision and choice of the patient from the hand of his oncology practitioner, through a more libertarian clinical drug development system that is nowadays frankly intervened. In this sense, some initiatives of globalization and clinical data sharing, as Targeted Agent and Profiling Utilization Registry (TAPUR) and CancerLinQ, are heading in the right direction.