Data analytics and methods

In 2022, EMA continued its transformation towards a data-driven organisation that supports decision-making in the EU medicines regulatory network by building capability and capacity in the analysis of data.

Significant progress was made in enabling the use and establishing the value of Big Data in the development, authorisation and supervision of medicines in Europe.

EMA data governance

As part of its transformation and to strengthen its data governance, in 2022 EMA established an internal Data Board to steer the organisation on data matters. Its initial workplan foresees a data strategy and an improved data governance framework to connect with network data governance.

Big Data Steering Group workplan 2022-25

Last year, the Big Data Steering Group (BDSG), which was set up in 2020 by EMA and the Heads of Medicines Agencies (HMA), endorsed its third workplan that detailed key actions to be delivered between 2022–25. Its aim was to further enhance the integration of data analysis into the evaluation of medicinal products by regulators. The workplan laid out deliverables and timelines including for areas like DARWIN EU®, data quality, data discoverability, and EU network skills. A synchronised review of its mandate and the EU Network Data Board terms of reference was launched aimed at improved network data governance.

The BDSG 2022 report provides a summary of the key activities and achievements of the BDSG in 2022 and shows that significant progress in the transformation to data-driven regulation continued during the year.

Raw data analysis

One of the initiatives in the work plan was the launch of a pilot project to assess whether the analysis of ‘raw data’ from clinical trials by regulatory authorities improves the evaluation of marketing authorisation applications for new medicines and therefore holds promise for faster and better medicines access for patients. There are several potential benefits the analysis of raw data might bring, including faster evaluation through fewer questions being put to applicants and a better definition of the target treatment population.

The pilot, launched in July 2022, will run for up to two years and will include approximately ten regulatory procedures submitted to EMA. Since its launch, EMA received a first submission of an initial marketing authorisation application including individual patient data from clinical studies and numerous applicants have indicated their interest for 2023 and beyond.

Learnings from the pilot will help the EU medicines regulatory network take an informed decision on the optimal place of raw data in regulatory assessment and decision-making.

Discoverability and quality of data – key to data-driven regulation

Data quality is critical to realise the full potential of data-driven regulation and supports the trust of patients and healthcare professionals. Two guidance documents were released in 2022.

• The draft Data Quality Framework for EU medicine regulation aims to set out quality criteria for data used in medicine regulation to ensure they are fit for purpose to support benefit-risk decisions. The public consultation was open until 18 November 2022.
• Following a public workshop, a list of metadata describing real-world data sources and studies has been finalised and published to help pharmaceutical companies and researchers identify and use such data when investigating the use, safety and effectiveness of medicines.
• The draft good practice guide for the use of the planned EU metadata catalogue of real-world data sources was the first regulatory guide produced worldwide to focus on the discoverability of real-world data, supporting systematic integration of real-world evidence in medicines regulation. The public consultation was open until 16 November 2022.

DARWIN EU® becomes operational

In February 2022, EMA started working with Erasmus University Medical Center Rotterdam to establish the Coordination Centre for the Data Analysis and Real World Interrogation Network (DARWIN EU®). The vision of DARWIN EU® is to give EMA and national competent authorities in EU Member States access to trustworthy real-world evidence – for example on diseases, patient populations, and the use, safety and effectiveness of medicines, including vaccines – throughout the lifecycle of medicinal products.

The role of the Coordination Centre is to develop and manage the network of real-world healthcare data sources across the EU and to conduct scientific studies requested by medicines regulators and other stakeholders.

The first set of data partners to collaborate with DARWIN EU® was selected in October 2022. The data available to these partners will be used for studies to generate real-world evidence that will support scientific evaluations and regulatory decision making on medicines. The Big Data Steering Group workplan foresees more than 140 DARWIN EU® studies per year by 2025.

Big data in veterinary medicines

When it comes to veterinary medicine, EMA and the HMA adopted a Veterinary Big Data strategy to 2027 outlining their vision for fostering data-driven, digital innovations in the veterinary medicines’ domain in the EU.

Building upon key objectives of the recently implemented Veterinary Medicinal Products Regulation (Regulation (EU) 2019/6), the strategy aims to converge traditional regulatory practice with innovative digital solutions. The strategy proposes to identify relevant use cases, existing and additional data sources, critical infrastructure and methods to enable an environment that encourages innovation in the development of new veterinary medicines for the benefit of animal and human health and welfare. The new strategy impacts different business areas, such as pharmacovigilance, the fight against antimicrobial resistance (AMR), environmental risk assessment, regulatory submission, innovation of veterinary medicinal products development and demonstration of efficacy/effectiveness.