Assessing treatment efficacy in early-phase oncology trials

February 3, 2022

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

THURSDAY, FEBRUARY 3 – 9:00 – 10:00 CET



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.

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.

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



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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