22 August, 2022

EMA wants to enable generation and use of RWE for regulatory decision-making

The role and use of real-world data (RWD) and real-world evidence (RWE) accros the different stages of medicine development is evolving.

July 2022, the European Medicines Agency (EMA) has endorsed a statement calling for international collaboration to enable the generation and use of real-world evidence for regulatory decision-making. One of the reasons is the EMA wants to enable new medicines to reach the market more quickly.

Real world data is observational information, which has not been collected in the context of a clinical trial. Researchers can obtain this type of information from patient files, claim data and disease registrations, but can also be supplied by patients using, for example, wearables, blood pressure monitors and questionnaires. EMA wants to use the data generated by such apps to assess, for example, how a patient experiences the treatment.

EMA will use these new forms of evidence in addition to the information from Randomised Controlled Trials (RCTs). In the US, manufacturers are already using analytics based on real world data to expand drug registrations. The real-world data will not replace RCT, but will be used in addition.

Why using RWE

  • The integration of science and technology in drug development;
  • better scientific evidence of new drugs;
  • a greater role for patients;
  • being able to respond quickly to new healthcare needs, such as the COVID-19 pandemic;
  • provide ‘a learning assessment system’ so that the EMA can continue to adapt to changes in drug development.


In the past, the EMA primarily reviewed large clinical trials, where the patient went to the hospital and received pre-described treatment. In recent years, EMA has received completely different files. This as result of the continued breakthrough of drugs against rare diseases, the introduction of gene therapy and the increasing use of biomarkers in defining a disease.

Diseases are increasingly broken down into small pieces, with the result that the groups of patients are also becoming smaller and smaller. With holding on to the classic setup with large RCTs, it would be difficult to admit medicines in the long run.

The actual drug development is increasingly done by universities and biotech companies with large investors behind them. These new players more often get stuck in the rules, as a result of which they fail to get their products from the lab to the clinic. In addition, EMA will also involve patients more in the process. 

Due to the higher complexity of using real-world evidence, EMA will invest more in expert networks. 

With the changes in the admission procedure, EMA wants to ensure that the EU remains an attractive place for drug development, in the interests of patient health and innovation.


Related Courses

 Read more about Real-World Data and Real-World Evidence at GetReal Institute.

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