New guidance enhances usability of HMA-EMA real-world data Catalogues

The European Medicines Agency (EMA) and Heads of Medicines Agencies (HMA) have published updated guidance and tools to support the use of the HMA-EMA Catalogues of real-world data (RWD) sources and studies. These updates aim to strengthen the generation of high-quality, reproducible real-world evidence (RWE).

To improve the discoverability and quality of RWD for regulatory decision-making and public health research, EMA and HMA have released a revised Good Practice Guide, a new User Guide, and an updated List of Metadata for the HMA EMA RWD Catalogues. Developed in line with the European Medicines Regulatory Network (EMRN) Strategy and the Big Data Steering Group (BDSG) workplan, these updates are based on stakeholder consultation and one year of practical experience since the Catalogues' launch in February 2024.

The initiative addresses a long-standing challenge in the RWE space: the lack of standardized, transparent, and reproducible information on RWD sources and studies. As the EMA explained in a recent LinkedIn post,

“One major challenge has been the lack of transparency and reproducibility of RWD studies and standardized information on RWD sources. To address this and to promote transparency, exchange of information and collaboration, EMA and the HMA launched two electronic catalogues for RWD sources and studies.”

Catalogues support transparency and data discovery

Since their launch in 2024, the Catalogues have seen steady growth, with over 3000 observational studies now registered and more than 245 RWD sources documented. These include data from general practice electronic health records (EHRs), hospital registries, biobanks, and health insurance claims.

The Catalogues serve two main purposes. They offer an open-access platform for registering RWD studies in advance, helping to promote transparency and reproducibility in research. At the same time, they serve as a central resource for identifying relevant data sources to support studies on how medicines are used, how effective they are, and whether they are safe. Users can also access study protocols and reports from initiatives such as DARWIN EU (Data Analysis and Real-World Interrogation Network) directly through the Catalogues.

While registration is mandatory for studies requested by EMA or national competent authorities, over half have been submitted voluntarily, highlighting strong engagement across the research community. As EMA noted, the Catalogues, “serve as an open-access resource to pre-register RWD studies in Europe and beyond, enhancing transparency and reproducibility.”

Good practice guide enables structured assessment of RWD

The Good Practice Guide for the use of the HMA EMA Catalogues of real-world data sources and studies (version 2.0, adopted February 2025) provides practical recommendations for identifying and evaluating RWD sources. Designed for regulators, academic researchers, and industry users, the guide outlines how to assess data relevance and quality for a specific research question.

Specifically, the guide supports users from the perspective of the data user. It describes key steps and best practices for selecting suitable data sources when planning a study, including how to assess relevance, accessibility, governance, and data quality. It also provides a six-step process for study planning, from searching databases using PICOT criteria to reviewing previous study usage and metadata on coding systems and representativeness. As the guide explains,

“Data quality can be defined as, ‘fitness for purpose for users’ needs in relation to health research, policy making and regulation, and the degree to which data reflect the reality which they aim to represent.’”

The guide also supports broader use cases, such as benchmarking data sources, assessing study protocols and reports, and evaluating study design elements. It is aligned with key regulatory frameworks and methodologies, including the Structured Process to Identify Fit-for-Purpose Data (SPIFD), target trial emulation, and the HARPER protocol template.

User Guide provides submission and maintenance guidance

While the good practice guide focuses on data users, the User Guide of the HMA EMA Catalogues of real-world data sources and studies is designed for users submitting data to the Catalogues, specifically, data holders and study investigators. It provides essential guidance on how to navigate the submission process, ensuring accurate and consistent entry of metadata.

The guide includes detailed descriptions and definitions for each data field, covering everything from administrative and methodological aspects to coding systems, linkage capabilities, and data quality indicators. It also offers step-by-step instructions on how to submit and maintain a record in the Catalogues, supporting regular updates and ensuring traceability over time.

By helping contributors align with common standards and regulatory expectations, the guide plays a key role in maintaining the overall quality and usability of the Catalogues for the broader research and regulatory community.

Updated metadata list improves standardization and interoperability

The updated List of metadata for the HMA-EMA Catalogues of real-world data sources and studies serves as the technical foundation for both catalogues. It captures detailed descriptors for RWD sources and studies across administrative, methodological, and data quality domains. Metadata elements cover key content areas such as exposure, outcomes, sociodemographics, and biomarker data, as well as quantitative descriptors like population size and observation periods.

For studies, metadata includes information on design, objectives, target populations, interventions, and data quality checks. Standardized terminologies such as ICD-10, MedDRA, and SNOMED are used throughout to support comparability and interoperability. According to EMA, “We’ve gathered feedback showing high satisfaction with the clarity and detail of the information available. We’ve also received valuable suggestions for further improvements, notably a request for additional guidance.”

The metadata structure supports alignment with regulatory frameworks and EU-level initiatives, including the European Health Data Space and DARWIN EU, where data quality, interoperability, and transparency are essential.

A transparent framework for RWE in regulation

Together, the Good Practice Guide, User Guide, and Metadata List provide a structured, harmonized framework for the effective use of RWD in medicines regulation. They reinforce the HMA-EMA Catalogues as a central resource for study registration, data discovery, and assessing data suitability.

EMA emphasized its commitment to continuous improvement, stating:

“We’re committed to expanding and improving the catalogues to further support regulatory decision-making.”