Real-World Evidence for Regulatory Decision-Making: Guidance From Around the World

Area of Science:

• Regulatory Science
• Health Policy
• Clinical Trials

Background:

• Growing global interest in using real-world evidence (RWE) for regulatory decision-making on product effectiveness.
• Inconsistent international acceptance of RWE, particularly for pre-market approval.
• Lack of specific guidance for innovative clinical trial designs like registry studies and pragmatic trials.

Purpose of the Study:

• To assess the global regulatory landscape concerning real-world evidence (RWE).
• To identify key regulatory elements including frameworks, data quality standards, and study methods guidance.
• To review existing global frameworks and identify gaps and opportunities for development.

Main Methods:

• Assessed the availability of three key regulatory elements across different regions: RWE regulatory framework, data quality and standards guidance, and study methods guidance.
• Reviewed existing international frameworks and guidance documents.
• Analyzed observed gaps and opportunities for improvement and harmonization.

Main Results:

• Significant global variation exists in the regulatory acceptance and frameworks for real-world evidence (RWE).
• Key elements such as data quality standards and guidance on innovative study methodologies are inconsistently available worldwide.
• Identified gaps in regulatory frameworks and a need for harmonization across different countries.

Conclusions:

• Cross-country collaboration is essential to align RWE policies and establish consistent global frameworks.
• Developing harmonized RWE policies will enhance efficiencies in regulatory decision-making.
• Further development and alignment of RWE policies are needed to support global regulatory approval processes.