Use of Real-World Data to Support US Regulatory Submissions  

Use of Real-World Data to Support US Regulatory Submissions

In July 2024, the Food and Drug Administration (FDA) finalized recommendations on using real-world data (RWD) and real-world evidence (RWE) in regulatory submissions. The FDA has increasingly recognized the importance of RWE in enhancing the regulatory process for drugs and medical devices. RWE refers to data collected from various sources outside of traditional clinical trials, including electronic health records, insurance claims, patient registries, and wearable health devices. This guidance aims to clarify how RWE can support regulatory submissions, particularly in evaluating safety, effectiveness, and post-market surveillance. 

Key Points from FDA RWE Guidance

  • Definition and Scope: RWE encompasses data derived from real-world settings, which can provide insights into patient experiences, treatment patterns, and outcomes. The guidance outlines scenarios where RWE can complement clinical trial data, such as in drug approvals, label expansions, and post-market assessments 
  • Methodological Considerations: The guidance emphasizes the need for rigorous methodologies when utilizing RWE. Key considerations include: 
    • Relevance: Consideration of availability, timeliness, and generalizability 
    • Reliability: Consideration of accrual, quality, and integrity of data, along with sufficient numbers of representative patient populations 
    • Data Quality: Ensuring data are accurate, complete, and representative 
    • Statistical Approaches: Employing appropriate statistical methods to address potential biases and confounding factors 
    • Validation: Confirming the findings through validation studies or comparisons with randomized controlled trial data 
      • Regulatory Uses: RWE can be leveraged in various regulatory contexts: 
        • Pre-Market Submissions: Supporting evidence for effectiveness and safety during the approval process 
        • Post-Market Studies: Monitoring ongoing safety and effectiveness in the general population 
        • Labeling Changes: Providing data that may justify modifications to product labeling based on real-world usage 
      • Stakeholder Engagement: The FDA encourages collaboration with stakeholders, including industry, academia, and patient advocacy groups, to enhance the quality of RWE studies. Additionally, engaging with the FDA early in the development process can help align RWE strategies with regulatory expectations  
      • Limitations: While RWE presents significant opportunities, it also has limitations, including potential biases inherent in nonrandomized data, variability in data sources, and challenges in data integration. The guidance underscores the necessity of addressing these limitations in any submission 
      • Future Directions: The FDA is committed to evolving its frameworks for the integration of RWE into regulatory decision-making. This includes ongoing research, stakeholder engagement, and the development of standardized approaches for RWD collection and analysis 

      The FDA’s guidance on RWE highlights its potential to enrich the regulatory landscape, allowing for more comprehensive evaluations of medical products. By adhering to methodological rigor and engaging with diverse stakeholders, the integration of RWE can lead to improved patient outcomes and a more responsive healthcare system. To learn more about using RWE in regulatory submissions, please contact us at Magnolia Market Access.