Collaborative Program to Evaluate Real World Data for Use in Clinical Studies and Regulatory Decision Making

Area of Science:

• Health Informatics
• Regulatory Science
• Clinical Research

Background:

• The 21st Century Cures Act enables the FDA to use real-world data (RWD) for regulatory decisions.
• Limited guidance exists on the comparative quality of different RWD types.
• Assessing RWD quality is crucial for its effective integration into drug development and post-market surveillance.

Purpose of the Study:

• To compare the quality of various real-world data (RWD) sources against traditional clinical trial data.
• To evaluate electronic health record (EHR) clinical data, EHR billing data, and linked healthcare claims data.
• To provide guidance on the relative quality of different RWD types for regulatory use.

Main Methods:

• The ACE-RWD program conducts comparative quality assessments of RWD sources.
• Ancillary studies are performed in parallel or subsequent to parent clinical studies.
• Data from EHR clinical, EHR billing, and healthcare claims are compared to clinical trial data.
• Health Level Seven (HL7) Fast Healthcare Interoperability Resources (FHIR®) standard is anticipated for data extraction.

Main Results:

• Quality assessment of EHR clinical data is underway.
• Quality assessment of EHR billing data is underway.
• Quality assessment of linked healthcare claims data is underway.
• Comparative analysis against traditional clinical trial data is ongoing across multiple therapeutic areas.

Conclusions:

• The ACE-RWD program will yield insights into the relative quality of diverse RWD sources.
• Findings will inform the appropriate use of RWD in regulatory submissions and post-approval studies.
• This research supports the 21st Century Cures Act's goal of modernizing regulatory pathways using real-world evidence.