Does Real-World Evidence have a role in Precision Oncology?

Fri, April 28, 2023, 09:00 – 10:00AM (PDT), 12:00 – 1:00PM (EST)

Abstract: 

The FDA instituted a program for Accelerated Approval in 1992, which allowed for approvals on the basis of surrogate endpoints for drugs treating serious conditions that filled an unmet medical need. The Food and Drug Administration Safety Innovations Act, passed in 2012, allows accelerated approvals for appropriate drugs and indications by evaluating the effects of drugs on surrogate markers. More recently, the FDA has established three additional pathways to speed the review process for emerging therapies. These changes prompted innovations in trial design with master protocol and seamless designs. Immune checkpoint inhibitors (ICIs) have yielded promising therapies for patients experiencing refractory cancers. Trials evaluating ICIs made extensive use of phase Ib, enrolling hundreds and even more than one thousand patients into dose expansion cohorts following dose-escalation spanning multiple tumor types. This represents a departure from conventional drug development strategies, for which dose expansion cohorts were used in roughly 25% of phase trials. Moreover, in 2021 two drugs, Atezolizumab and Durvalumab, were voluntarily withdrawn from accelerated approvals for PD-L1 inhibition in advanced or metastatic bladder cancer. This presentation considers the statistical implications of expansive, uncontrolled early phase trials and discusses the potential role for real-world evidence in this setting.

Speaker:

Dr. Hobbs completed a doctoral degree in biostatistics at the University of Minnesota and then joined The University of Texas MD Anderson as an Assistant Professor of biostatistics. He was promoted to Associate in 2017, and then recruited to Cleveland Clinic to found a Section of Cancer Biostatistics. He joined The University of Texas Dell Medical School in August 2020 as a tenured Associate Professor. The Eastern North American Region of International Biometric Society selected his thesis paper for the John Van Ryzin Award in 2010. In 2016, Dr. Hobbs was selected by The University of Minnesota for the Emerging Leader Award, an honor bestowed on alumni on the basis of impactful contributions within 10 years of graduating from one of The School of Public Health’s 20 programs. Recognized as an expert in clinical oncology research methodology, in 2017 Dr. Hobbs was invited to lead the publication of National Cancer Institute’s Clinical Trials Design Task Force with the goal of providing national, consensus recommendations for first-in-human cancer drug trials that use seamless designs. In 2018, he co-founded Telperian Inc. where he currently contributes in the role of scientific advisor. In 2020, he was invited to contribute to an article for Nature Reviews Clinical Oncology describing the current state of tumor agnostic trials. In 2021, Dr. Hobbs was invited to review the landscape of basket trials in the Journal of Clinical Oncology.

Keywords: Bayesian, clinical trials, real-world data, seamless design, cancer 

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