The Good Practice Guide for the use of the HMA-EMA Catalogues of Real World Data (RWD) sources and studies has been developed to provide regulators, researchers (including academia and pharmaceutical industry) and other interested stakeholders with recommendations on the use of the catalogues from the perspective of the data user. Specifically, the guide focuses on describing the steps and best practices on identifying a suitable data source, when planning a study.
For the users submitting data to the Catalogues (i.e.: data holders and investigators submitting study data) a user guide has also been published to help users navigate the Catalogues in this context. The user guide provides descriptions of the data fields and definitions, as well as guidance on how to submit and maintain a record in the Catalogues. The user guide is available on the Catalogues website https://catalogues.ema.europa.eu
The Catalogue of RWD studies, replacing the former EU PAS Register, aims to promote transparency of observational studies in the context of the medicine regulation activity. A central repository where observational research is registered benefits the scientific community and promotes the use of Real World Evidence (RWE).
Real-world data (RWD) and real-world evidence (RWE) are increasingly used in the scientific evaluation of medicines to support regulatory decision-making. Conducting RWD research and interpreting study findings pose unique challenges and require a good understanding of associated methods, terminologies as well as deep knowledge of data source characteristics and of the healthcare system organisation in the respective countries. However, information on RWD sources is often lacking or not standardised. Such information, commonly provided as metadata1 or “data about data”, offers context about the data and includes details on a dataset’s purpose, location, key-variables, generation, format, and ownership. As mentioned in the Data Quality Framework (DQF) for EU medicines regulation, metadata are essential to understand the meaning of data and to assess the fitness for purpose (e.g. quality) of a dataset for a specific purpose. The HMA-EMA (Heads of Medicines Agencies-European Medicines Agency) Catalogues of RWD sources and studies along with the EMRN DQF and the Draft ‘Data Quality Framework (DQF) for EU medicines regulation: application to Real-World Data’ (hereafter referred to as RW-DQF) can help to further address these aspects.
Having access to a standard and electronic set of complete and accurate metadata for data sources can support the identification of data sources suitable for a specific study, facilitate the description of the data sources planned to be used for a specific study or research, and help to properly assess the evidentiary value of the study results. Metadata are often published in data catalogues, which have the purpose of making data discoverable and assessable for fitness for purpose without revealing the raw data themselves. The Heads of Medicines Agencies/European Medicines Agency (HMA/EMA) joint Big Data Task Force recommended “to promote data discoverability through the identification of metadata” as part of its priority recommendation III: “Enable data discoverability. Identify key meta-data for regulatory decision making on the choice of data source, strengthen the current European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) resources database to signpost to the most appropriate data, and promote the use of the FAIR principles (Findable, Accessible, Interoperable and Reusable) (HMA/EMA, 2020).
The RWD Catalogues provide elements that cover information on type of data present, as well as their extensiveness. The Catalogues also provide the elements to be included in the table of data sources recommended by the HARmonized Protocol template to Enhance Reproducibility (HARPER). These data elements cover:
Setting: country(-ies), region(s), type of data source, care setting. For sensitive variables, depending on country- and data holder-specific data privacy rules, not all items may be published in the catalogues due to privacy concerns.
Population: total and active population size, percentage of the population covered by the data source in the catchment areas, description of the population for which data are not collected, age groups, social and demographic information, lifestyle factors, family linkage, availability of data on pregnancy and neonates, trigger for registration and de-registration, median time between first and last records for all individuals and active individuals.
Exposure: availability of data on prescriptions and/or dispensing, advanced therapy medicinal products (ATMPs), contraception, vaccines, other injectables, medical devices, procedures, medicinal products and indication and biomarker data.
Outcomes: availability of data on hospital admission or discharge, intensive care unit (ICU) admission, death (mortality) and cause of death, clinical measurements, genetic data, patient-generated data, health care utilisation, diagnostic codes, specific diseases with disease information collected.
Time elements: date when the data source was established, first collection date and last collection date, median time between the first and the last available records for unique individuals captured in the data source and for unique active individuals.
Link to the Catalogue of RWD studies also allows for identification of studies that have been performed with the same data source, and subsequently for an evaluation of its suitability to answer the research question of interest.