Experiences with learning and confirming in drug and biological development

Abstract

Since the 1997 landmark article by Dr. Lewis Sheiner in Clinical Pharmacology & Therapeutics, biopharmaceutical development in phases I-IIA has become more targeted toward learning (i.e., establishing proof of concept), then subsequently confirming that regulated standards are met. The purpose and importance of the learning-proof-of-concept phase is subjective but typically uses traditional statistics (which were developed for use in confirming). Two examples from development are presented to illustrate learning in practice. Suggestions for how to improve and embed the learn-confirm concept and how to enhance the contributions of clinical pharmacology and statistics are considered.