State of the Science in Patient-Focused Drug Development with Statistics Leading Innovation: Where Have We Been, and Where Are We Going?

• Activity Number: 552 - State of the Science in Patient-Focused Drug Development with Statistics Leading Innovation: Where Have We Been, and Where Are We Going?
• Type: Topic Contributed
• Date/Time: Thursday, August 11, 2022 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Joseph C. Cappelleri* and Mallorie Fiero* and Shing M Lee* and Jessica Roydhouse*
• Companies: Pfizer Inc and US Food and Drug Administration and Columbia University and Menzies Institute for Medical Research, University of Tasmania
• Keywords: clinical trials; dose-response; estimands; oncology; patient experience; treatment effect

Abstract

Patient-focused drug development (PFDD) is increasingly important for regulators, payers, and pharmaceutical companies. This panel session brings together experts from academic, industry, and regulatory institutions to discuss critical methodological issues and new developments in the field of patient-centered measurement science – and, specifically, how statistics can serve as a foundation for innovation and progress. The panel will seek to answer the following broad questions: What is the state of the science in PFDD? Where have we been and where are we going? In addressing these questions, each panelist will give a 15-minute presentation on a specific, targeted topic on PFDD. The panel will cover the following central topics in PFDD: 1) sharing updates on standards for patient experience data in oncology clinical trials; 2) designing and analyzing trials with patient experience data: Using the ICH E9 (R1) estimand framework; 3) communicating evidence about the knowledge and perception of treatment assignment; and 4) incorporating the patient experience in early phase dose-finding trials. Audience participation and questions will be encouraged through discussion.