The report begins by clarifying what is meant by real-world data (RWD) and real-world evidence (RWE). RWD refers to health-related information collected outside of traditional clinical trials (for example from electronic health records, insurance claims, registries).
RWE is the insight derived from analysing RWD to understand how treatments and care perform in “real-world” settings, helping inform decisions around patient care, policy and regulation.
The report emphasises why RWD/RWE is especially important in the rare-disease context: because rare-disease populations often fall outside the design of large clinical trials (small numbers, heterogeneity, complex patient journeys), RWD can fill gaps—giving insight into disease progression, patient characteristics, treatment outcomes, care pathways, and the “experience” of living with a rare condition.
You can read these insights from a roundtable discussion by clicking below.
