Companion Diagnostics in Oncology Drug Development

• 1 unit
• Quarter Offered: Fall
• Instructor: Ramez N. Eskander, M.D., Professor, UC San Diego SOM & Moores Cancer Center

Course Description & Learning Objectives

In solid tumor oncology, companion diagnostics (CDxs) are playing an increasingly important role in identifying treatments by enriching for patients who are likely to benefit from a novel drug. The United Stated (US) Food and Drug Administration (FDA) is currently placing a larger emphasis on CDxs development in an effort to ensure safe and effective use of therapeutic products. In 1998, Trastuzumab was the first drug to ever obtain US FDA approval together with a CDx assay examining HER2 expression. Using real world examples (i.e. development of Mirvetuximab Soravtansive and its CDx Folate Receptor alpha expression (FOLR1)) this course will demonstrate the importance of thoughtful clinical trial design and CDx development from the perspective of a clinical trialist. The course will additionally highlight areas of vulnerability during CDx development that can ultimately affect drug approval and access.

Our understanding of cancer biology has resulted in a dramatic shift in oncology drug development. In an era of “precision oncology” it is critically important to understand how to both design and interpret clinical trials that are focused on enriching patient response. This course is timely and relevant as it will explain CDx development in oncology and regulatory implications with practical clinical examples.

Learning Outcomes & Skills Acquired from the Course

• Clearly define what a CDx is in oncology drug development.
• Be able to design a clinical trial incorporating a CDx.
• Explain to both colleagues and patients what a CDx “means” in cancer care.
• Discuss limitations to CDx development in oncology
• Understand the definition of a CDx from a regulatory perspective
• Define the differences between US FDA and European Medicine Agency (EMA) approach to CDx development
• Incorporate US FDA guidance documents in drug development and clinical trial design (what is a high risk, class III device?)
• Understand regulatory implications of developing a CDx. Does the CDx have to be approved for the drug to be approved?
• Pragmatic approach to clinical trial design, with predictive biomarker assays to enrich the clinical population with responders
• Understand prospective-retrospective study design for CDx development with clinical examples
• Describe how do CDxs fit into accelerated approval drug development programs