CQS Workshop Series 2013
"Statistical issues in evaluating the efficacy of personalized medicine"
Presenter: Motomi Mori, Ph.D., Professor and Head, Division of Biostatistics, Department of Public Health & Preventive Medicine; Director, Biostatistics Shared Resource, Knight Cancer Institute
Oregon Health & Science University, Portland, OREGON USA
Collaborators: Yiyi Chen1,2, Byung S. Park1,2, Jeffrey W. Tyner2,3, Marc M. Loriaux2,3, Stephen Spurgeon2,3, Brian J. Druker2,3
1Division of Biostatistics, Department of Public Health & Preventive Medicine; 2Knight Cancer Institute
3Division of Hematology/Oncology, Department of Medicine
Oregon Health & Science University, Portland, Oregon 97239, USA
With the advance of high-throughput genetics/genomics technology, the concept of personalized medicine, i.e., therapies tailored to the molecular and genetic profile of an individual patient, is becoming a realistic possibility. In this presentation, we will discuss statistical issues involved in evaluation of the clinical utility of “personalized medicine” using an example of a novel in-vitro screening assay developed by cancer researchers at OHSU. The in-vitro kinase inhibitor assays evaluate sensitivity of primary leukemia cells against a panel of small-molecule kinase inhibitors (i.e., molecularly targeted drugs). It is hypothesized that the assay results can help identify molecularly targeted drugs to which a patient is most likely to respond. To test this hypothesis in a phase II trial setting, we will propose several phase II trial designs and evaluate their operating characteristics through simulation. We will present the results of the simulation study and discuss general statistical issues/challenges involved in testing a personalized medicine strategy. These include trial design, patient eligibility, selection of endpoints and control group, randomization, sample size and analysis strategies.