The contribution of real-world evidence (RWE) to regulatory submissions as supportive or substantial evidence has increased in the pre-marketing authorization space over the last decade. An extensive series of guidance documents have been put forth by the United States (US) Food and Administration (FDA) on the use of real-world data (RWD) and RWE in regulatory submissions, with several guidance themes emerging. In this white paper, we discuss key takeaways from a series of FDA guidance and review selected case studies of RWD/RWE to highlight best practices and learnings that sponsors can implement when planning and managing their regulatory submissions.

Recent FDA guidance emphasizes that real-world data (RWD) / real-world evidence (RWE) used in regulatory submissions must be submitted electronically and adhere to the approved data standards for such submissions. Not adhering to these guidelines on data standards may have implications on the regulatory submission and review process. Therefore, aligning RWD/RWE with the FDA’s requirements is crucial to facilitate a streamlined regulatory review for submissions containing RWD/RWE.

This White Paper aims to: 

• Summarize relevant FDA guidance documents
• Describe currently accepted data standards
• Summarize key elements of the data submission
• Discuss key challenges that companies may encounter for RWD