Leveraging Real-World Evidence: A Paradigm Shift in Regulation.

Area of Science:

• Regulatory Science
• Medical Device Development
• Real-World Data Analytics

Background:

• Regulatory frameworks for real-world data (RWD) collection are rapidly advancing.
• The Food and Drug Administration (FDA) has a history of accepting real-world evidence (RWE) for specific regulatory decisions.
• Recent progress focuses on using RWE for premarket regulatory assessments.

Purpose of the Study:

• To examine the evolving role of RWE in regulatory decision-making processes.
• To review key concepts in RWE study design for generating valid scientific evidence.
• To facilitate the use of RWE in supporting marketing authorizations for medical devices.

Main Methods:

• Review of current regulatory frameworks and methodologies for RWE collection.
• Analysis of hybrid study designs integrating randomized clinical trial elements with RWE.
• Case study examination of the Bioventus Observational Non-Interventional EXOGEN Studies (BONES) program for orthopaedic and trauma devices.

Main Results:

• Evolving methodologies and regulatory frameworks enhance the use of RWE.
• Hybrid study designs are pivotal for accelerating RWE adoption in regulatory submissions.
• Careful planning enables the generation of robust RWE for device effectiveness and safety evaluation.

Conclusions:

• RWE plays an increasingly significant role in regulatory decision-making.
• Methodological advancements and hybrid designs are crucial for leveraging RWE.
• The successful application of RWE supports marketing authorization and enhances understanding of device performance.