Real-world evidence, often called RWE, is evidence generated from data collected in routine healthcare settings. It helps show how a health technology performs outside controlled clinical trials.
In HTA, real-world evidence can support the evaluation of medicines, medical devices, diagnostics, digital health solutions and healthcare interventions.
Clinical trials remain essential, but they do not always reflect everyday clinical practice. Real-world evidence helps decision-makers understand how a technology may perform in real patients, real healthcare systems and real treatment pathways.
It can provide information about effectiveness, safety, adherence, persistence, healthcare resource use, patient outcomes, quality of life and budget impact.
In simple terms, real-world evidence helps answer an important question:
What happens when this technology is used in routine clinical practice?
Why is real-world evidence important?
HTA agencies and payers often need to make decisions with some level of uncertainty.
Clinical trials are designed to test efficacy and safety under controlled conditions. However, trial populations may be narrower than real-world populations. Follow-up periods may be limited. Comparators may not always match local clinical practice.
Real-world evidence helps reduce uncertainty by providing additional context.
It can show how patients are treated in practice, how diseases progress over time, how healthcare resources are used and how outcomes differ across patient groups.
For healthcare systems, this information can be highly relevant because reimbursement decisions are not made in theory. They are made within real budgets, real pathways and real patient populations.
How does real-world evidence support reimbursement?
Reimbursement decisions often require evidence that is relevant to the local healthcare context.
A payer may want to understand how many patients are eligible for treatment, how the technology will be used, what costs may be generated and whether expected outcomes are realistic in routine practice.
Real-world evidence can help support these questions.
It may provide data on local epidemiology, treatment patterns, disease burden, healthcare resource utilisation, adherence, persistence, safety and patient outcomes.
This can be especially important when clinical trial evidence is limited, when there is no direct comparison with the local standard of care or when long-term outcomes remain uncertain.
Real-world evidence can also support post-launch monitoring, managed entry agreements and reassessment after reimbursement.
What sources of data are used for real-world evidence?
Real-world evidence can be generated from different types of real-world data.
These may include electronic health records, hospital databases, claims data, patient registries, pharmacy data, disease registries, observational studies and patient-reported outcomes.
Each data source has strengths and limitations.
Electronic health records can provide clinical information about diagnosis, treatment and outcomes. Claims data can help analyse healthcare resource use and costs. Registries can be valuable for long-term follow-up, rare diseases or specific patient populations.
Patient-reported outcomes can capture quality of life, symptoms, functioning and treatment experience directly from patients.
The choice of data source depends on the research question, the decision context and the quality of the available data.
What are the challenges of using real-world evidence?
Real-world evidence can be powerful, but it must be generated carefully.
Because real-world data are not usually collected in controlled research environments, they may include missing information, inconsistent coding, selection bias or confounding.
This means that methodology is critical.
A strong real-world evidence study should have a clear research question, appropriate data sources, transparent methods, suitable statistical analysis and careful interpretation of uncertainty.
For HTA and reimbursement, credibility matters. Decision-makers need to understand not only the results, but also how reliable those results are.
Real-world evidence should therefore be fit for purpose, scientifically robust and aligned with the needs of payers and HTA agencies.
Final thoughts
Real-world evidence plays an increasingly important role in HTA and reimbursement. It helps bridge the gap between clinical trial evidence and routine healthcare practice.
By providing insight into real patients, real treatment patterns and real healthcare systems, RWE can support stronger market access strategies and more informed decision-making.
At Clevidence, we support organisations with real-world evidence planning, healthcare data analytics, observational study design, evidence generation strategy, HTA support and value communication.