Real World Evidence Demonstration Projects and Policy Development at FDA

• Activity Number: 60 - Real World Evidence in Regulatory Decision Making: Past, Current and Future?
• Type: Topic Contributed
• Date/Time: Sunday, July 29, 2018 : 4:00 PM to 5:50 PM
• Sponsor: Health Policy Statistics Section
• Author(s): Diqiong Xie* and David Barrett Martin
• Companies: FDA and FDA CDER OMP
• Keywords: Cures Act; Real Word Evidence; Indications; FDA-Catalyst; FDA My Studies App; Clinical Trial Replication

Abstract

Under the 21st Century Cures Act the Food and Drug Administration (FDA) is required to develop a framework for a program to evaluate the use of real world evidence (RWE) to help support the approval of a new indication for an approved drug or to satisfy post approval study requirements. The effort will result in draft guidance for industry by December 2021. Demonstration projects intended to inform the policy development process have already started. FDA is building infrastructure for distributed pragmatic trials through the FDA-Catalyst program which is part of the Sentinel Initiative. In the IMPACT Afib trial, 80,000 patients with atrial fibrillation identified through claims data have been randomized for an intervention. The FDA My Studies mobile app captured informed consent and patient provided data for linkage to electronic health data. In order to evaluate the potential future role of observational studies, FDA has initiated a project using claims data to replicate 30 clinical trials representing a range of indications, healthcare outcomes, and trial designs. Trial selection considerations and the agreement framework will be presented.