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Multiple Perspectives on the Need for Real-World Evidence to Inform Regulatory and Health Technology Assessment
Marieke S Jansen1, Olaf M Dekkers1,2,3, Saskia le Cessie1,4
1Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands.
Pharmacoepidemiology and Drug Safety
|January 8, 2025
Summary
Real-world evidence (RWE) needs for health technology assessment (HTA) and regulatory decisions share common contextual factors but differ in specific questions addressed. Understanding these nuances is key for effective RWE utilization in healthcare.
Area of Science:
- Health Economics and Outcomes Research
- Regulatory Science
- Evidence-Based Medicine
Background:
- Real-world evidence (RWE) is increasingly integral to regulatory and health technology assessment (HTA) decision-making.
- Perspectives on the relevance and application of RWE can vary significantly across different healthcare domains.
- Previous research has explored RWE in regulatory contexts, necessitating a deeper understanding of its role in HTA.
Purpose of the Study:
- To identify factors influencing the need for RWE in HTA decision-making.
- To confirm and enrich these factors through stakeholder engagement.
- To evaluate the similarities and differences in RWE requirements between regulatory bodies and HTA agencies.
Main Methods:
- A scoping review of 118 articles was conducted to identify factors for RWE in HTA.
- Semi-structured interviews with stakeholders were performed to validate and expand upon literature findings.
- A comparative analysis integrated review and interview data to explore RWE needs in both regulatory and HTA settings.
Main Results:
- The HTA review identified 45 factors influencing RWE needs, categorized into questions addressable by RWE and contextual factors.
- Stakeholder interviews corroborated the literature-derived factors, highlighting alignment in contextual influences.
- Differences emerged in question-related factors; RWE is a direct input for HTA, unlike its role in regulatory benefit-risk assessment, with HTA requiring broader scope and specific details.
Conclusions:
- Contextual factors driving the need for RWE are largely consistent between regulatory and HTA decision-making processes.
- Variations in RWE needs primarily stem from the specific questions each domain seeks to address.
- RWE serves distinct purposes in regulatory submissions (e.g., orphan drugs, risk minimization) versus HTA (e.g., comparator selection, implementation evaluation).
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