Pragmatic Considerations for Endpoint Selection and Display in Benefit-Risk Assessment
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*Bennett Levitan, Janssen Research & Development, LLC  Zhong Yuan, Janssen Research & Development, LLC 

Keywords: benefit-risk assessment, endpoint, composite, clinical trials

While there are numerous approaches for drug benefit-risk assessment, most share the need for a structured approach to select the endpoints included in the assessment. The requirements for endpoints in benefit-risk are not always considered when designing clinical trials, and in some cases may not align. Approaches for benefit-risk endpoint selection include using a single composite endpoint that combines benefits and harms, a pair of endpoints that compares an efficacy composite to a safety composite, and characterization of the full set of benefits and harms. These approaches vary in their ease of interpretation, potential for double-counting, mixing of events with varying clinical impact and in their ability to show core tradeoffs. This presentation will compare and provide pragmatic examples of these approaches for setting up and displaying results of a benefit-risk assessment.