Real World Evidence Position Statement

The Scottish Medicines Consortium (SMC) has published a Position Statement on the use of Real-World Evidence (RWE). This guidance explains when and how the data generated from everyday clinical practice can strengthen the evidence base for the appraisal of new medicines in Scotland and support decision-making committees to reach informed decisions. The full document is now available in the Making a Submission section of the SMC website.

Why is RWE important?
Randomised controlled trials (RCTs) remain the gold standard for investigating a medicine’s efficacy, but they aren’t possible in every circumstance; for example, in rare conditions, small paediatric populations or situations where a treatment is already the only option available. In such cases, early-phase, single-arm or open-label studies often form part of the evidence base.

That’s where RWE adds values since it can:

• Fill evidence gaps when traditional studies are too small or can’t run long enough to capture long-term data.
• Show how a medicine performs in real practice. For instance, patients treated in NHS Scotland or a similar population, rather than the narrower cohort enrolled in a global study.
• Strengthen health economic models by providing up-to-date information on resource use, adherence and quality-of-life outcomes.

What does the SMC Position Statement on RWE cover?

• Clear definitions of Real-World Data (RWD) and RWE.
• Methodological standards from choosing the right data source to handling confounding and missing data, selecting appropriate study design and relevant outcomes investigated and adequate reporting of these.
• Transparency standards outlining RWE studies proper management of RWD, such as data provenance, governance and quality.
• When RWE provides the greatest impact, such as rare diseases, long-term outcomes and situations where RCTs are not feasible or ethical. Or supporting non-RCT data, such as phase I/II single-arm or open-label studies.
• Economic-evaluation expectations, such as the inclusion of scenario analyses that explore the real-world impact of a medicine.

This Position Statement is effective immediately for all future submissions. SMC will keep the document under review and update it as international best practice evolves.

If you have any questions or want to discuss how RWE fits into an upcoming submission Contact us