Deliver Transformative Medicines to Patients by Leveraging Complex Innovative Designs

• Activity Number: 174 - Patient Centricity at the Heart of Innovative Trial Design
• Type: Invited
• Date/Time: Monday, August 8, 2022 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Amy Xia*
• Companies: Amgen
• Keywords: Complex Innovative Design; Bayesian Hierarchical Model; CID Pilot ; Modeling and Simulation; Design Operating Characteristics

Abstract

Complex innovative designs (CID) can potentially reduce patient burden, reduce cost, shorten cycle time and increase probability of success in drug development so new, transformative medicines can be delivered to patients in a more expedited way. PDUFA VI and 21st Century Cures Act provide exciting opportunities for industry to collaborate with regulatory agencies in promoting and advancing the use of CID. In this presentation, a case study accepted by the FDA CID pilot program will be described. Experiences and considerations from this case study regarding rationale for the design, innovative design elements, evaluation of trial operating characteristics, trial execution, simulation tools being developed, as well as sponsor-regulatory agency interactions will be discussed.