Real World Evidence to Support Accelerated Approval Processes

• Activity Number: 438 - Real-World Evidence in Clinical Trial: New Era of Informed Decision Making Session
• Type: Invited
• Date/Time: Wednesday, August 2, 2017 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Sebastian Schneeweiss and Jessica Franklin*
• Companies: Harvard Medical School and Harvard Medical School
• Keywords: Real world data ; accelerated approval ; phase 2/3 trial ; Clinical trial design

Abstract

Real world data (RWD) in concert with randomized trials add to an integrated evidence generation system throughout the drug life cycle. This presentation will focus on how real world evidence (RWE) can inform phase 2/3 trial design and its key role in adaptive pathways and accelerated approval. Regulators explicitly look for RWE to contribute to incremental evidence development as soon as medications are marketed via accelerated approval pathways. The presentation speaks about RWE that is fit for decision making, i.e. is meaningful evidence, valid evidence, expedited evidence, and transparent evidence. The presentation covers methodological aspects as well as database considerations.