Many Faces of Subgroup Analysis in Clinical Drug Development

• Activity Number: 285
• Type: Invited
• Date/Time: Tuesday, August 11, 2015 : 8:30 AM to 10:20 AM
• Sponsor: ENAR
• Author(s): Ilya Lipkovich* and Alex Dmitrienko
• Companies: Quintiles and Quintiles
• Keywords: Subgroup analysis ; exploratory data analysis ; reproducibility ; statistical learning

Abstract

Subgroup analyses (SA) in pharmaceutical industry is somewhat poorly defined area that may range from hypothesis testing in purely confirmatory settings to biomarker/subgroup discovery in exploratory settings. In real clinical practice hybrid SA strategies are often employed combining elements of both confirmatory and exploratory SA. In this talk we try to delineate distinct types of SA and present some features of principled SA focusing on criteria for assessing the validity of exploratory subgroup analysis such as reproducibility, type I error control, and complexity control. These principles are illustrated with several key methods for exploratory subgroup analysis that emerged in such diverse fields as multiple testing, machine learning, and causal inference using clinical data examples.