Use of Benefit-Risk, Safety, and Estimand Planning Tools to Optimize Drug Approvability and Labeling

• Activity Number: 518 - Estimand, Causal Inference, and Other Statistical Considerations in Clinical Trials
• Type: Contributed
• Date/Time: Thursday, August 11, 2022 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Susan Mayo*
• Companies: Food and Drug Administration, Center for Drug Evaluation
• Keywords: Benefit-risk value tree; Aggregate safety assessment plan; Estimand framework; Drug development ; Planning

Abstract

In 2021, FDA released benefit-risk and estimand guidance documents, and a paper was published by the ASA Safety Working Group on safety planning. This talk provides an overview of how three recommended planning tools, benefit-risk value tree, aggregate safety assessment plan, and estimand framework can be used in tandem. The benefit-risk value tree (to identify key benefits/risks and how to assess them) and aggregate safety assessment plan (to plan reporting of safety data for safety deliverables required throughout drug development) offer a comprehensive data overview of a drug’s development program. The estimand framework helps establish what is needed during clinical trial design so the trial’s analysis and interpretation are well-aligned with its clinical trial objective. Such planning, like a blueprint, can avoid unnecessary missteps during drug development to position a compound for its best opportunity to demonstrate its benefit-risk balance and substantial evidence of effectiveness. The talk will focus on points in the drug development process where sponsors and regulators may be missing opportunities.