Achieving Regulatory Approval Using Real-World Evidence: Challenges and Considerations

• Activity Number: 467 - Alternative Data Sources Utilization in Clinical Development
• Type: Topic Contributed
• Date/Time: Wednesday, August 10, 2022 : 2:00 PM to 3:50 PM
• Sponsor: Health Policy Statistics Section
• Author(s): Tae Hyun (Ryan) Jung*
• Companies: US FDA
• Keywords: Real-World Evidence ; Real-World Data; Regulatory Science; Registry; Observational study; FDA

Abstract

The 21st Century Cures Act of 2016 contains provisions to help accelerate medical product development and bring new innovations and advances to patients who need them faster and more efficiently. As real-world data (RWD) presents great potential to accelerate drug development, real-world evidence (RWE), the clinical evidence generated from the analysis of RWD, has received substantial attention in recent years. On July 16, 2021, FDA approved a new use for Prograf (tacrolimus) based on a non-interventional (observational) study providing RWE of effectiveness. This approval reflects how a well-designed, non-interventional study relying on fit-for-purpose RWD can be considered adequate and well-controlled under FDA regulations. This talk will overview the regulatory considerations from statistical perspective. Then it will discuss the challenges and importance of getting a quality fit-for-use RWD for submission.