US Food and Drug Administration's Advancing Real-World Evidence Program: Initial Experience

Area of Science:

• Regulatory Science
• Health Informatics
• Data Science

Background:

• Real-world data (RWD) availability and analysis have advanced significantly.
• Robust real-world evidence (RWE) can now be generated to support regulatory decision-making.
• Cross-discipline collaboration is crucial for maximizing RWE's potential in regulatory settings.

Purpose of the Study:

• To address the need for increased confidence in RWE-based regulatory approaches.
• To establish a new pathway for regulatory interactions concerning RWE.
• To foster expertise and collaboration in the use of RWE for regulatory purposes.

Main Methods:

• Leveraging advances in RWD availability and analytical methodologies.
• Implementing collaborative, cross-discipline approaches.
• Establishing the FDA's Advancing RWE Program for regulatory interactions.

Main Results:

• Increased generation of robust RWE.
• Enhanced confidence in RWE-based regulatory submissions.
• A new FDA program facilitating RWE discussions.

Conclusions:

• The Advancing RWE Program provides a vital platform for regulatory engagement on RWE.
• Cross-discipline collaboration is key to realizing the full potential of RWE in regulatory science.
• Continued development of RWE methodologies will further support regulatory decision-making.