EMA - Publication of Article on Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value

26th November 2021

EMA - Publication of Article on Real-World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value

Enabling the use of real-world evidence (RWE) and establishing its value for regulatory decision-making on the development, authorisation and supervision of medicines in Europe by 2025: this is the vision of European regulators as outlined in an article from Peter Arlett, Head of Data Analytics and Methods at EMA, Jesper Kjær, Director of Data Analytics Centre at the Danish Medicines Agency, Karl Broich, President of the Federal Institute for Drugs and Medical Devices (BfArM), and Emer Cooke, EMA's Executive Director, published in Clinical Pharmacology & Therapeutics.

The authors emphasise that delivering this vision, anchored in the Network Strategy to 2025, will support the development and use of better medicines for patients.

The creation of the Data Analytics and Real World Interrogation Network (DARWIN EU) will be key to delivering this vision. This EU-wide network will allow to access and analyse healthcare data from across the EU. It will be launched in early 2022 with the establishment of a coordination center to on-board data partners and to drive the conduct of studies requested by medicines regulators and, at a later stage, also requested by other stakeholders.

The article explains plans to establish methods and standards for high-quality collection and use of RWE, in cooperation with stakeholders including patients, healthcare professionals, industry, regulatory and public health agencies, health technology assessment bodies, payers, and academia.

According to the authors, it will be important to advance the debate on the value of RWE compared to randomised clinical trials (RCTs), the gold standard to demonstrate efficacy of a medicine. The vision is that RWE and RCTs should be seen as complementary, each having strengths and weaknesses, with their relative importance depending on the regulatory question. A rigorous and systematic approach to learning from doing will help to identify and establish the use-cases in regulatory decision-making for which RWE will add most value.

In this context, EMA has also contributed to an article that examines when and how RWE was used to support marketing authorisation applications for new products and extensions of indications, submitted to the Agency in 2018 and 2019. The retrospective analysis shows that 40% of initial marketing authorisation applications and 18% of applications for extension of indication for products currently on the market contained RWE. The article describes the characteristics of RWE included in these applications and identifies areas where further research is required.  

Both articles aim to support transformation to data-driven regulatory decision-making and to advance patient-centered access to better medicines. They are available through open access:

• Real‐World Evidence in EU Medicines Regulation: Enabling Use and Establishing Value 

·         Marketing Authorization Applications Made to the European Medicines Agency in 2018–2019: What was the Contribution of Real‐World Evidence?  

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