Real-world evidence and its importance in medicines development

What is real-world evidence (RWE)?

Real-world evidence (RWE) is the evidence derived from the analysis and/or synthesis of real-world data (RWD). RWD is an overarching term for data on the effects of health interventions (such as benefits, risks or resource use) that are not collected in the context of conventional randomised controlled trials (RCTs). Instead, RWD is collected both prospectively and retrospectively from observations of routine clinical practice. It may include clinical and economic outcomes, patient-reported outcomes and health-related quality of life.

RWD can be obtained from many sources including patient registries, electronic medical records, and observational studies. Find more about existing RWD sources here and generating RWD here.

For more information about the terms used on this website, see the GetReal glossary.

How can RWE be used in medicines development?

RWE has an important role in decision-making related to authorisation and access to new medicines and reimbursement. It can be used for:

  • understanding ‘real-world’ settings such as treatment populations, patterns of care and the burden of disease
  • assessing the effectiveness of current therapies using existing data
  • refining or supplementing evidence from conventional trials of new medicines
  • providing new evidence of relative effectiveness of new medicines.

RWD has conventionally been used to understand and characterise treatment populations, patterns of care and burden of disease. It is also used to describe the natural history of disease, project long-term outcomes, and define and validate new measures of outcome of relevance to patients.

RWE of the effectiveness of existing therapies will usually be available to help plan studies of new medicines. In some situations it may be possible to generate new RWE (‘early’ estimates) of the effectiveness of a new medicine, in time to inform reimbursement decision-making.

Why is RWE important in medicines development?

In most European healthcare systems, decision-making related to (national and regional) reimbursement and access to medicines by local healthcare payers and providers has become more prominent. RWE can be used to support these decisions.

Decision making for marketing authorisation of new medicines is traditionally supported by randomised controlled trials (RCTs), recognised as the ‘gold standard’ for establishing safety and efficacy. However, RCTs are undertaken in an idealised care environment and they may only measure efficacy in restricted populations, defined by trial eligibility criteria.

Healthcare payers, including reimbursement decision makers, wish to know the relative effectiveness of a new medicine compared with the current standard treatment in patients eligible for the new medicine in their population (the ‘treatable population’). They will ask questions such as:

  • Where will this medicine be used in the ‘care pathway’ for these patients?
  • Is the treatable population similar to the trial population, both in terms of socio-demography and in treatment history?
  • Is the treatable population currently receiving similar care to the comparator group in the trial?
  • Do the measures of health outcome (safety and efficacy) reported in the trial form a sufficient evidencebase on which to make a decision? Do they capture relevant patient and clinician experience?

RCTs alone may not provide sufficient evidence of relative effectiveness. To support decision-making:

  • RCTs may be combined in new types of meta-analyses to enable a wider range of comparisons with alternative therapies.
  • Statistical analyses may be applied to RCT data to estimate the efficacy in specific sub-populations or populations with different characteristics.
  • Studies (randomised or non-randomised) of the effectiveness of new medicines in less restricted populations may be carried out in usual care ‘real-world’ settings.
  • Statistical analyses may be used in advance of trials to help refine the trial design and optimise the results.

Both for authorisation and for reimbursement of new medicines RWD / RWE can be used to refine or supplement the evidence available from conventional trials.

GetReal and RWE

The focus of GetReal is on the use of RWE in medicine development, before and around the time of marketing authorisation and reimbursement. It is primarily focused on the potential use of RWE to provide evidence on relative effectiveness of new medicines. For more about GetReal, see here.