A new European platform for advancing regulatory science research

Launching in 2025, the European Platform for Regulatory Science Research will bring together academia, regulators and other stakeholders to accelerate collaborative regulatory science research solutions.

Regulatory science is essential for addressing issues and gaps encountered by stakeholders in medicines research, development and evaluation. Research in regulatory science delivers solutions by providing better methodologies for medicines research and development, and improved tools for evaluating medicines based on outcomes that better align with patients’ needs. It drives progress by identifying gaps in the evolving regulatory system and developing new approaches for generating evidence to support decision-making. Tangible impacts of regulatory science research include the revision or creation of new regulatory guidelines, or the qualification of novel methodologies such as biomarkers to support their use in medicines development.

Regulatory science research is multidisciplinary and involves a diverse range of methods, including wet-lab and desk-based research, text and data analysis, and techniques from mathematical and social sciences. Such research is being conducted in academic centres, but also by, and often in collaboration with, regulators, industry and patients (Supplementary Fig. 1). Collaboration and dialogue between these stakeholders is needed to identify and address emerging regulatory science research questions, keeping the field in step with innovation, and enhancing its impact on regulatory standards and decision-making.

However, while regulators and academia currently collaborate in various ways, and with differing degrees of intensity, the regulatory science research landscape is fragmented, with limited cross-national collaborations and no centralized, Europe-wide system in place4. Researchers less familiar with regulatory practices might not be fully aware of how their work could have an impact on regulatory practice and/or how to design it so that it could lead to the development of, or changes to, regulatory guidelines, policy changes or the qualification of novel tools for medicines development.

Facilitating more systematic exchange, collaboration and cross-pollination on research questions, methods and translational approaches will help address regulatory science challenges. And importantly, the anticipated new pharmaceutical legislation in Europe, which will be the largest reform of EU medicines regulation in decades, is providing momentum for efforts to rethink and expand working models. Our ambition is to engage researchers in regulatory science through initiatives that build on the European Medicines Agency (EMA) Regulatory Science Strategy to 2025 and the new European Medicines Agencies Network Strategy to 2028.

Here, we introduce the European Platform for Regulatory Science Research, a new initiative jointly led by the EMA and Heads of Medicines Agencies (HMA) in the EU that is set to launch in 2025, with the aim of strengthening and expanding regulatory science research with increased application potential.

The European Platform for Regulatory Science Research

The European Platform for Regulatory Science Research is designed to foster collaboration among academic researchers and regulatory scientists from Europe and beyond on key topics in the field. Its overarching goal is to advance and accelerate regulatory science research, focusing on questions of common interest, informing research on these key questions, increasing the usefulness of research outcomes and supporting the translation of solutions into the work of regulators and medicines developers. The platform is expected to enable researchers to amplify the impact of their research on medicines development and regulatory decision-making, facilitate projects and collaborations with researchers from other groups and regulators, and also to increase their awareness of initiatives and support from regulators. Both EU nationals and non-EU nationals are invited to join, as the platform aims to foster collaboration not only within the EU but also beyond.

A multi-step structured process informed the platform’s development, including (1) mapping of the academic regulatory science researcher landscape, (2) consultations within the EMA and the European regulatory network, (3) development of a draft concept paper describing the proposal, (4) a public consultation on the draft concept paper (available here), (5) a public event to introduce the platform and its objectives (recording and presentation available here), (6) a first call for expression of interest to invite stakeholders to become involved (available here), and (7) refinement of the proposal based on the public engagement input. The full proposal for the European Platform for Regulatory Science Research is described in the public draft concept paper (available here)5.

The platform’s agenda will consist of a combination of cross-cutting topics and specific case studies and research projects. Cross-cutting topics will include identifying regulatory science research gaps and finding topics of common interest, discussing methods and best practices for regulatory science research, and discussing how to improve the translation and impact of research outcomes. These will be informed by discussions on specific research topics and projects, as case studies, with the aim of generating lessons and best practices for a wider group of researchers and regulators.

Discussion topics will encompass a range of research gap types, including knowledge, conceptual, theoretical, methodological, empirical, practical and population gaps, spanning human and veterinary domains. Topics will include data sources, such as how to access and leverage available clinical data and catalogues of real-world evidence, and how to use European Public Assessment Reports (EPARs) to extract detailed information on medicines’ benefit–risk assessment, which researchers can use to analyse regulatory decision-making processes and assess the scientific basis behind them. Topics will also include the evaluation and use of new methods; for example, how to develop new statistical methods, or how to conduct document analyses that are fit for purpose, including the use of data science and artificial intelligence.

Other topics of common interest and case studies to inform cross-cutting discussions include research into methods to improve acceptance and efficiency of platform trials or research on topics related to the new draft pharmaceutical legislation, such as platform technologies. These discussions will involve experts of the corresponding committees or working parties.

The translation of regulatory science research tools into real-world regulatory applications is crucial for advancing progress. One avenue for this is qualification, through which innovative tools such as new biomarkers or models for medicines development are evaluated for their potential use in regulatory decision-making. Qualification and complementary pathways for fostering translation into regulatory applications will be discussed in platform meetings.

To support topic finding for regulatory science research, the EMA published its first list of regulatory science research needs in 2021 to encourage researchers and funding organizations to consider these questions in their work and programmes. This list is periodically updated, with the 2024 update available here. Topics from this list can be selected for discussion in the platform.

As regulatory science research is conducted not only by researchers in the academic sector and regulatory agencies, the platform’s steering group will also invite patients, healthcare professionals, pharmaceutical industry representatives, research funders, and health technology assessment (HTA) representatives to participate as observers. Over time, the involvement of these stakeholder groups may be expanded.

The platform will launch with a one-year pilot phase, and two to three meetings convening platform participants will be organized in this first year to discuss selected questions, case studies, research gaps, and priority topics. In addition, topic-centric discussions can be organized. After this one-year pilot phase, the platform and events may be adapted.

A call to drive progress in regulatory science research

The EMA and the European regulatory network are now inviting academic and not-for-profit researchers to join this new platform. A call for expression of interest to participate is open throughout the year (available here). A second call for expression of interest for patients, healthcare professionals, industry, and HTA bodies to participate as observers in the steering group will be opened later in 2025. The platform’s first steering group meeting is scheduled in the first quarter of 2025, followed by the platform’s inaugural meeting in the second quarter.

The EMA and the European regulatory network look forward to strengthening their collaboration with the research community and stakeholders to drive advancements in regulatory science and deliver solutions that benefit patients and the public across Europe and beyond.