Assessing treatment efficacy in early-phase oncology trials


One of the major recent trends in drug development in oncology, particularly with highly targeted therapy and immunotherapy, is a great concern with efficacy assessment in early phases of development. This was not frequent before the era of precision medicine. As a result, phase 1 trials now routinely have efficacy endpoints and even sample-size justification related to efficacy, particularly in expansion cohorts.

In this webinar, we will:

– Present a historical overview of efficacy assessment in early-phase trials
– Highlight some recent regulatory trends in that respect
– Discuss the tension between the clinically appealing and statistically sound methods to assess the efficacy of novel treatments in phase 1 trials, single-arm phase 2 trials, and randomized phase 2 trials