Sources of Safety Data and Statistical Strategies for Design and Analysis in Clinical Trials

• Activity Number: 4 - Safety Evaluation, Data Sources, and Statistical Strategies: Transforming Information into Evidence
• Type: Invited
• Date/Time: Sunday, July 30, 2017 : 2:00 PM to 3:50 PM
• Sponsor: ENAR
• Author(s): Richard C. Zink*
• Companies: JMP Life Sciences, SAS Institute, Inc.
• Keywords: adverse events ; data monitoring committee ; data visualization ; multiplicity ; meta-analysis ; subgroup

Abstract

There has been an increased emphasis on the proactive and comprehensive evaluation of safety endpoints to ensure patient well-being throughout the medical product life cycle. In fact, depending on the severity of the underlying disease, it may be appropriate to plan safety assessments as early as the start of the development program. Statisticians should be intimately involved in this process and contribute their expertise to safety data collection, analysis, reporting, and data visualization. In this talk, we review the challenges associated with the analysis of safety endpoints and describe the safety data that are available to influence the design -- as well as the safety data that are a result -- of one or more clinical trials. We share our recommendations for the statistical and graphical methodologies necessary to appropriately analyze, report, and interpret safety outcomes, and we discuss the advantages and disadvantages of safety data obtained from clinical trials compared to other sources. This work is presented on behalf of the ASA Biopharmaceutical Section Safety Working Group.