Phase I trials in oncology: Pros and cons of current designs

October 21, 2021

Demystifying different types of Bayesian and frequentist designs that may be used for first-in-human or other types of phase I trials in oncology

THURSDAY, OCTOBER 21 – 5:00 – 6:00 PM CET | 11:00 AM – 12:00 PM EDT



Historically, phase 1 trials in oncology have mostly used the 3+3 design for dose finding, even though many alternatives have been proposed over the years. That design is useful mainly for its simplicity, but it suffers from disadvantages that can be circumvented by designs which provide for more flexibility and an enhanced performance in terms of various desirable outcomes.

  • In this webinar, our goal is to demystify different types of Bayesian and frequentist designs that may be used for first-in-human or other types of phase 1 trials in oncology.
  • We will start by reviewing the recent literature describing general trends in the design of phase 1 trials over the past few years.
  • We will then summarize the most salient features of Bayesian and frequentist designs in this setting.
  • Finally, we will formulate recommendations for use of phase-1 designs in different situations and touch upon the inclusion of expansion cohorts in phase 1 studies.
  • The interested registrant should notice that this webinar will be followed by a similar one dedicated to phase 2 trials, which will cover different statistical caveats, including early consideration of randomization, issues related to sample-size calculation and the use of biomarkers.


  • Be updated on current trends in phase-1 designs in oncology
  • Gain insights into the advantages and disadvantages of various Bayesian and frequentist approaches to the design of phase 1 trials


Elisabeth (Els) Coart, PhD, is Principal Biostatistician, Consulting Services, IDDI.


• Els graduated in Bio-engineering at KU Leuven in 1995, got her PhD in Molecular Genetics in 2003 at Ghent University, and a Master’s degree in Biostatistics also at Ghent University, in 2007.
• After some years of experience in research and clinical biostatistics in diagnostic companies, Els joined IDDI in 2011, where she conducted research related to biomarkers in Alzheimer’s disease and validation of surrogate endpoints in advanced colorectal cancer. In 2015, Els joined the team of consultants at IDDI, led by the company founder, Marc Buyse. She served as Director, Consulting Services, from 2017 to 2019 and now focusses on consultancy activities.
• As a consultant, Els has helped a large number of pharmaceutical and biotechnology companies to design, implement and analyze clinical trials for drug development, with a focus on oncology and biomarkers. She is particularly interested in early-phase trials using frequentist and Bayesian methods, as well as phase trials with biomarker-driven designs. Moreover, given her strong background in assay development she is IDDI’s expert in analytical and clinical validation of in-vitro diagnostics.
• Els has published more extensively in Alzheimer’s disease, biomarkers, and oncology.


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: Phase I trials in oncology: Pros and cons of current designs

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