The Influence of Clinically and Statistically Meaningful Differences in Risk Benefit: A Case Study

• Activity Number: 206
• Type: Roundtables
• Date/Time: Monday, August 10, 2015 : 12:30 PM to 1:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Tammy Massie*

Abstract

This discussion will focus on the evolution of the benefit-risk assessment approaches in the licensure process. This case study will examine recently approved allergenic sublingual immunotherapy (SLIT) products, including the steps taken during the review of the products, as well as creation of a more formalized development and adoption of the structured benefit-risk assessment approaches as part of PDUFA V. In 2014, three SLIT products were approved for the treatment of grass- or ragweed pollen--induced allergic rhinitis. These approvals were a result of interaction among pharmaceutical sponsors, external stakeholders, the Allergenic Product Advisory Committee (APAC), and an FDA review team who identified the potential benefits and risks of these novel products and developed a path forward. As part of these efforts, statisticians worked closely with the agency's multidisciplinary review team to develop criteria for efficacy that would ensure an acceptable benefit-risk profile. The speaker will present this case study developing the assessment tools and share lessons learned that may be of value to colleagues in industry, academia, and other government agencies.