Innovative Oncology Trial Designs In Practice

Clinical development of anti-cancer therapeutics faces particularly high rates of failure, even in the context of low success in drug development as a whole. (1) One major contributing factor is the uncertainty about the treatment effect or expected benefit. In a 2012 article, Gan et al (2) analyzed over 250 published oncology trials. The authors found little correspondence between the expected and observed benefits. More importantly, the expected benefits consistently overestimated the observed benefits often by a large margin.

New clinical development strategies are urgently needed to improve the probability of success. One promising approach, recently championed by FDA Commissioner Scott Gottlieb (3), is the adaptive clinical trial. These more flexible approaches to trial design – from exploratory designs that more effectively target the maximum tolerated dose, to population enrichment methods that support precision medicine, have the potential to improve efficiency and reduce risk. On the following pages, you will see examples of how Cytel's strategic consulting group has helped clients create and apply innovative trial designs within exploratory and confirmatory oncology development.