Introductory Overview Lecture 6: Personalized Medicine

• Activity Number: 275
• Type: Other
• Date/Time: Tuesday, August 6, 2013 : 8:30 AM to 10:20 AM
• Sponsor: ASA
• Author(s): Stephen J. Ruberg*+
• Companies: Eli Lilly & Company
• Keywords: tailored therapeutics ; bootstrapping ; recursive partitioning ; Type 1 error ; drug development ; subpopulation

Abstract

Personalized medicine refers to how a medical intervention (e.g. drug/biologic treatments, surgery) can be tailored to individual patients based that patient's individual characteristics. In pharmaceutical drug development, a pharmaceutical company must have a clinical development program that provides substantial evidence that the treatment is safe and effective in a specific subpopulation of patients. This can be a very challenging undertaking.

This talk will discuss practical considerations for developing a tailored therapeutic, considering trial design and analysis issues. Some novel proposals will be made that can help both pharmaceutical companies and regulatory agencies identify the right subpopulation of patients using recursive partitioning techniques. Due consideration must be given to pre-specification of analysis methodologies, control of Type I errors when looking across multiple subpopulations. Permutation distributions are utilized to adjust for multiplicity and bootstrapping is utilized to obtain unbiased estimates of treatment effects in subpopulations of interest. Some real-life examples will be provided.