Rationale for the Use of Subject Level Information for Benefit-Risk Evaluations

• Activity Number: 570 - More Emerging Topics in Benefit-Risk Assessment in Clinical Development Decision Making
• Type: Topic Contributed
• Date/Time: Wednesday, August 2, 2017 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Shihua Wen* and Weili He and Scott Evans and Qi Jiang and Jonathan Norton and Xuefeng Li and George Quartey
• Companies: Novartis and Merck & Co., Inc. and Harvard University and Amgen Inc. and Takeda and FDA and Genetech
• Keywords: benefit-risk assessment ; patient-level

Abstract

Structured benefit-risk (BR) assessment has recently been employed by FDA and EMA during the regulatory review process. Value tree, effect tables, and other quantitative BR assessment methodologies have appeared more frequently in regulatory submission packages. However, most of the BR analyses are based on evaluation of aggregated data (e.g., study level summary data) summarized separately for each important outcome. This approach may not tell the complete story and does not directly describe the benefit-risk experience of patients in the trial. Therefore, it is critical to look into the subject-level data in order to obtain a comprehensive picture of the benefit-risk profile of the medical intervention under evaluation. This presentation will describe the rationale of the use of subject-level BR assessment to demonstrate why it is important, discuss briefly various patient-level BR analysis methods, and illustrate concepts using hypothetical and real data examples.