Discover how real-world evidence (RWE) addresses long-term treatment effects, fills evidence gaps, and enhances decision-making in health technology assessments (HTA).
Table of Contents
Question
Select the focus of real world evidence in recent health technology assessment dossiers
A. Effectiveness of the treatment
B. Effectiveness of the comparator
C. Long term treatment effect
Answer
C. Long term treatment effect
Explanation
The focus of real-world evidence (RWE) in recent health technology assessment (HTA) dossiers is C. Long-term treatment effect.
RWE is increasingly used to address critical gaps left by randomized controlled trials (RCTs), particularly in evaluating long-term outcomes of health technologies. RCTs often lack extended follow-up periods or real-world applicability, especially for complex therapies like advanced therapeutic medicinal products (ATMPs) or medical devices. HTA bodies leverage RWE to:
- Assess durability of treatment effects beyond trial timelines.
- Reassess technologies post-market to validate safety and effectiveness in diverse populations.
- Support conditional reimbursement decisions requiring evidence of sustained benefits.
For example, RWE from registries and observational studies is prioritized for therapies with accelerated approvals, where long-term data from RCTs are unavailable. This aligns with initiatives like NICE’s Early Value Assessment (EVA), which emphasizes real-world outcomes for technologies in early adoption phases.
While RWE also informs treatment effectiveness (A) and comparator effectiveness (B), these are often secondary to addressing uncertainties about long-term impacts. For instance, European HTA agencies frequently use RWE to extend evidence horizons for rare diseases or therapies with limited trial data.
The emphasis on long-term treatment effects reflects RWE’s role in bridging evidence gaps and ensuring HTA decisions account for real-world clinical and economic outcomes over time.
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