254081 On the Road to Personalized Genomic Medicine in Oncology

Tuesday, November 1, 2011: 4:30 PM

Richard Simon, PhD , National Cancer Institute, Rockville, MD
This presentation will address the validation of prognostic and predictive biomarkers for identifying which patients require treatment and which benefit from specific regimens. I will distinguish analytical validation, clinical validation and medical utility. I will discuss key structural limitations of the vast majority of prognostic marker studies that render them therapeutically irrelevant and how this can be avoided. I will discuss how it is necessary to shift from a retrospective data-dredging “correlative science” posture to a prospective predictive medicine approach. I will present prospective clinical trial designs for co-development of new cancer drugs and companion diagnostics. I will discuss the prospective-retrospective approach to establishing the medical utility of prognostic biomarkers using archived tumor specimens from key clinical trials.

The opportunities for rapid progress to personalized predictive oncology are enormous today. The greatest challenges are only partly scientific. Maximum progress does require better understanding of the key oncogenic mutations and de-regulated pathways. But misunderstanding of the role and nature of properly designed clinical trials in establishing medical utility of diagnostics is also limiting progress.

Learning Areas:
Biostatistics, economics

Learning Objectives:
Describe opportunities and challenges for personalized medicine.

Presenting author's disclosure statement:

Qualified on the content I am responsible for because: Current research interests include Bayesian methods in clinical trial design and analysis, and the development of methods for the analysis of genome sequence and expression data to identify cancer related genes, elucidate their functions, determine the steps of tumor development, identify molecular targets and develop genome based approaches to the prevention, detection, diagnosis and treatment of cancer
Any relevant financial relationships? No

I agree to comply with the American Public Health Association Conflict of Interest and Commercial Support Guidelines, and to disclose to the participants any off-label or experimental uses of a commercial product or service discussed in my presentation.