The increasing digitalization of healthcare data has created novel opportunities to leverage real-world data (RWD) in evaluating health technologies. Regulatory bodies like the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) encourage using RWD to generate real-world evidence (RWE) for decision-making.
Study Objective
This study aimed to inform the value of RWE for European HTA and the criteria driving the acceptance and design of robust external control arms (ECAs) to support HTA decision-making. It focused on five HTA agencies across Europe: AEMPS (Spain), G-BA (Germany), HAS (France), NICE (UK), and AIFA (Italy).
Methodology
The study implemented multiple approaches:
- Targeted literature review to identify technologies using RWE in regulatory submissions
- Analysis of HTA documents containing RWE
- Semi-structured expert interviews with HTA experts
- Review of RWE guidance documents from the included HTAs
Key Findings
- Variability in RWE Acceptance: The acceptance of RWE varied significantly among HTA bodies. NICE (UK) and AEMPS (Spain) had the highest acceptance rates, while G-BA (Germany) and HAS (France) were more cautious.
- Use of RWE: RWE was used for various purposes, including characterizing disease epidemiology, identifying standard of care, and supporting economic models.
- Best Practices for ECA: The study identified eight key categories for designing SATs with RWE-based ECAs, emphasizing representativeness, transparency, and methodological rigor.
- Country Profiles: Country-specific profiles highlighted different approaches to RWE use and acceptance, influenced by local healthcare systems and regulatory frameworks.
Challenges and Limitations
- Limited number of technologies covered
- Representation of each HTA body by a single expert (except for the UK)
- Difficulty in pinpointing the specific impact of RWE on final HTA decisions
Conclusion
RWE is becoming integral to evidence submitted to HTA bodies alongside clinical trials. Its acceptance varies by application and HTA body. Factors like methodological rigor, population comparability, and transparency influence RWE acceptance. Enhanced collaboration among stakeholders is needed to develop integrated evidence-generation plans addressing the needs of patients, healthcare professionals, regulators, and HTA bodies.
