Assessing treatment efficacy in early-phase oncology trials

February 3, 2022

early phase oncology trials

Join IDDI experts to get updated on efficacy assessment in early-phase trials and more!


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

Errata: At 8’17’’ and 8’57’’, the speaker mentions “safety”, when in fact what was meant was “efficacy”. The speaker apologizes for these mistakes.


Everardo Saad, MD, Medical Director at the International Drug Development Institute, IDDI.


• He has nearly 20 years of experience in Medical Oncology and clinical-trial design.
• He graduated in Medicine and trained in Internal Medicine in Sao Paulo, and did his fellowship in Medical Oncology at the University of Texas M.D. Anderson Cancer Center, in Houston.
• After practicing for several years, he shifted his professional career towards education and research in Medical Oncology, and has a special interest in clinical trial methodology, the assessment of endpoints, and the development of novel therapies for cancer patients.

Presentation Webinar: Assessing treatment efficacy in early-phase oncology trials

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