Acceptability of Using Real-World Data to Estimate Relative Treatment Effects in Health Technology Assessments:...

Area of Science:

• Health Technology Assessment
• Real-World Evidence Research
• Comparative Effectiveness

Background:

• Randomized controlled trials (RCTs) are the standard for evaluating new treatments but have limitations.
• Nonrandomized studies (NRS) using real-world data (RWD) are increasingly used to complement RCTs.
• Concerns exist regarding the acceptance of NRS evidence by health technology assessment (HTA) agencies.

Purpose of the Study:

• Identify barriers to the acceptance of NRS evidence by HTA agencies.
• Propose steps to increase the acceptability of NRS in future HTA.
• Highlight the role of NRS in HTA when RCT evidence is limited.

Main Methods:

• Expert opinion from academic, HTA, and industry settings.
• Critical assessment of existing studies on real-world evidence.
• Identification of barriers across stakeholder groups (HTA agencies, evidence generators, external stakeholders).

Main Results:

• Barriers include lack of methodological guidelines for RWD, deviations from best practices, and data access issues.
• Facilitating steps involve improving RWD quality, integration, and accessibility.
• Wider use of demonstration projects and enhanced HTA guidelines are recommended.

Conclusions:

• NRS provide crucial evidence for HTA when RCT data is insufficient.
• Addressing identified barriers can improve the utilization of NRS in HTA.
• Enhancing RWD infrastructure and guidelines will increase NRS acceptability.