Across-Country Variations of Real-World Data and Evidence for Drugs: A 5-European-Country Study

Area of Science:

• Health Economics
• Health Policy
• Pharmacoeconomics

Background:

• Real-world data (RWD) and real-world evidence (RWE) are crucial for informing health technology assessment (HTA).
• Understanding the role and acceptance of RWE in HTA across European countries is essential for evidence-based decision-making.
• Existing literature highlights varying degrees of RWE integration into HTA processes.

Purpose of the Study:

• To describe the role of RWD and RWE in HTA across five European nations.
• To identify barriers hindering the acceptance and utilization of RWE in HTA.
• To propose strategies for the more effective application of RWE in HTA.

Main Methods:

• Comparative analysis of RWD/RWE utilization in HTA across France, Germany, Italy, and Sweden using a standardized template.
• In-depth case study of England's Cancer Drugs Fund as a model for RWD in coverage and managed entry agreements.

Main Results:

• HTA bodies in most surveyed countries acknowledge RWD/RWE for post-launch uncertainties, except in Germany where randomized controlled trials are prioritized.
• Challenges to RWE adoption include data quality, relevance, interoperability, and timing synchronization with HTA agendas.
• The Cancer Drugs Fund model highlights the importance of stakeholder partnerships and pragmatic data use.

Conclusions:

• Sustained investment in national health information systems is vital for generating reliable RWE.
• Developing standardized processes and tools to guide RWE acceptability and application is crucial for its effective integration into HTA.