Webinar: Randomization and the Limits of Precision Oncology

August 26, 2021

Drug development in oncology is undergoing several changes brought about by precision medicine, which makes it appealing to move efficacy assessment to earlier-phase trials and questions the need for randomized trials. Indeed, some remarkably efficacious drugs have even been approved based on uncontrolled phase I or II trials.

With very few exceptions, we challenge the view that the expected benefits from new drugs are generally sufficient to forgo randomization to a standard-of-care arm. Apparently improved outcomes in a single-arm early trial may be due at least in part to the prognostic nature of the target and to selection bias, rather than result from a true effect of therapy. Moreover, the predictive role of biomarkers cannot be ascertained in a definitive way without randomization to a control arm.

On the other hand, randomization often raises scientific, ethical and economic issues when precision medicine is understood in a very strict sense, according to which each tumor presents a unique molecular landscape in a manner that precludes the formation of groups of patients with a similar disease.

The challenge for the scientific community moving forward will be to strike the right balance between eliciting high-level evidence, such as that originating from randomized trials, and leveraging the full potential of precision medicine.


Everardo Saad is the medical director at IDDI and a member of its team of consultants. He is a medical oncologist who trained at the University of Texas M.D. Anderson Cancer Center and developed a special interest in clinical-trial methodology. After several years of clinical practice, he shifted his career toward consultancy in clinical research. He has over 15 years of experience in design and analysis of clinical trials for pharmaceutical/biotech companies and academic groups. He has published more extensively in the area of efficacy endpoints in oncology.


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