Statistical Methods for Benefit Risk Assessment

• Activity Number: 647
• Type: Contributed
• Date/Time: Thursday, August 8, 2013 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Lanju Zhang*+ and Bo Yang
• Companies: Abbvie and Abbvie Inc
• Keywords: Benefit Risk

Abstract

Benefit risk assessment is a fundamental element of drug development, critical for both the pharmaceutical companies and regulatory agencies. Traditionally the benefit of a drug will be summarized, during submission, in the integrated summary of efficacy (ISE) and the risk in the integrated summary of safety (ISS). These two sets of evidence of the drug will be weighed by experts of the advisory committee and compared to evidence of available drugs on market for the same indication. A decision of approval or rejection will be based on the votes of these experts who synthesize and compare the benefit and risk evidence in their minds. This process obviously suffers from great subjectivity. Therefore, a quantitative approach to this process is pressingly called for and numerous attempts have been made to this goal, for example, the ratio of number needed to treat and number needed to harm, benefit-less risk, clinical utility index, or multi-criteria analysis. However, the use of these methods is rare because of their limitations. In this talk, we will briefly review the pros and cons of these methods and points out some new directions.