Real-world evidence in medicines regulation

About a year ago, we entered a new phase in our journey as the EU Medicines Regulatory Network (EMRN) to make full use of data in our regulatory decision making for the benefit of patients and public health.

One year on from establishing DARWIN EU? – the Data Analysis and Real World Interrogation Network – and the value it will bring is becoming clear.

As a physician myself, I could see early on the potential clinical benefits of real-world evidence (RWE). The potential to use big data to get better evidence, to make better decisions and deliver safer and more effective medicines to patients would help us achieve our public health goals in the European Union.

Lead by EMA on behalf of the EMRN, DARWIN EU? is a network of data partners and a suite of tools to analyse data, with the coordination centre at Erasmus University Medical Center Rotterdam.

DARWIN EU? generates RWE to complement the data generated by clinical trials, supporting the decision making of EMA scientific committees and?national competent authorities?in EU Member States, throughout regulatory processes.

A key design feature of DARWIN EU? is that the data are converted into a common data model. In addition, data stays local and is analysed locally by the data partners. This combination of features ensures personal data protection by design and enables big, multi-database studies to be conducted very rapidly.

RWE has been established in the evaluation of medicines safety for decades, it’s not new, but you rarely get to see its impact on the scale and speed of what we witnessed during the pandemic.

During the COVID-19 we proactively set up RWE studies which were critical for learning about the safety of the vaccines when they were rolled out in early 2021 and, subsequently for studying their effectiveness.

The pandemic served as a catalyst for RWE – enabling analysis and demonstrating benefits in a critical public health situation.

It is exciting to watch this development up close, but I truly believe we are just scratching the surface of the potential of RWE. While the benefits will primarily be for patients – by delivering better evidence, enabling better regulatory decisions and safer and more effective medicines – they go beyond medicines regulation.

Also, we are now developing RWE pilots with health technology assessment (HTA) bodies, with payers and with the European Centre for Disease Prevention and Control (ECDC) to establish the value of RWE to support their decision making. We are also participating in a pilot of the future European Health Data Space.

Currently DARWIN EU? has 10 data partners, from eight European countries and has access to 26 million ‘active’ patients, i.e., where data collection is ongoing.

We already have data from Germany, Netherlands, Belgium, France, Spain, UK, Finland, and Estonia, providing a mixture of general practice and hospital data as well as a biobank dataset and a cancer registry. We have initiated four studies using real-world data (RWD), starting to demonstrate the benefits of the network, learning as we go.

Our goal is to onboard another 10 data partners this year and increase up to at least 40 partners in 2025. With additional data partners we will increase the number of patients and the diversity of data available for analysis.

By 2025, DARWIN EU? will be delivering more than 140 studies per year. The number of studies and the speed at which they will be delivered is unprecedented and should transform the volume and quality of clinical evidence available to regulatory decision making.

Of course, any program involving big data on such a large scale – across many countries - comes with its own obstacles and challenges. There is the challenge of change itself, for example, overcoming the misperception that RWE replaces clinical trials. I would like to highlight that RWE complements clinical trials, which remain the gold standard for demonstrating efficacy. However, RWE can fill information gaps and add real world context.

DARWIN EU? makes patient data that already exists in systems available for analysis. If the outcome is better evidence, better decisions, and safer and more effective medicines – isn’t that what patients and healthcare professionals want?

One year on, DARWIN EU? is already showing promise to improve patient care and public health in the EU. Those were our central goals when we started, and I can see bigger things to come. Think about the difference RWE could make in 10 years’ time, or even 20.

And then remember, we have only just started.

• To find out more about the first RWE studies by DARWIN EU? and our open call for data partners, please visit the EMA website: DARWIN EU? has completed its first studies and is calling for new data partners | European Medicines Agency (europa.eu)
• Call for expressions of interest from data holders who wish to be considered as data partners is open. Visit the?DARWIN EU? website?for more information: How to join the network? (darwin-eu.org)
• To see the recording of my LinkedIn live interview on real-world evidence in medicines regulation: https://www.dhirubhai.net/feed/update/urn:li:activity:7052186442625679362

Peter Arlett , Head of Data Analytics and Methods Task Force