egulatory science bridges the gap between medicines research and the practical application of its findings in regulatory decision-making. At a time of accelerating healthcare innovation, advances in regulatory science are essential to provide the rigorous scientific and technical frameworks necessary for evaluating the quality, benefits, and risks of increasingly complex medicines. To ensure that the promises of innovation translate into the timely availability of safe and effective medicines for the patients who need them, regulators, researchers, and developers need to keep fostering excellence in regulatory science.

Regulatory science is a multidisciplinary field of fundamental and applied research that provides the tools, standards, and methodology to support medicines research and development (R&D) as well as the evaluation of these products. Regulatory science complements the disciplines of regulatory affairs and regulatory law, with the latter focusing on compliance with and enforcement of requirements. Regulatory science aims to provide a resilient and adaptable regulatory framework that ensures the decision-making process remains consistent, evidence-based, and transparent and, at the same time, aligns with growing scientific insights and developments in such fields as pharmacology and pathophysiology, novel methodologies in trial design and biostatistics, benefit-risk assessment, and post-marketing surveillance. Promoting and achieving excellence in regulatory science is central to the mission of the European Medicines Agency (EMA) and that of the broader network of European medicines regulators (EMRN).

At the EMA, medicines evaluation has been conducted within a clearly defined regulatory framework since its inception in 1995, now 30 years ago. But during that time, the framework and the science guiding it have never been static. With the evolution of regulatory science, EMA’s role has expanded from a traditional gatekeeper assessing the benefit-risk balance of new medicines to a proactive enabler of innovation that continues to ensure the safety and efficacy of medicines while also facilitating medicines development and timely access for patients. Together with other agencies, in the EU network and globally, EMA strives to provide a regulatory environment that supports innovation and the development of new and better medicines to meet human and animal health needs.

Supporting the broadening of EMA’s role in medicines development, regulatory science provides an ecosystem for change that ensures that pharmaceutical R&D and regulatory decision-making continuously adapt to scientific progress. EMA systematically identifies and addresses gaps in the regulatory science areas supporting its activities through a continuous process of several structured feedback mechanisms. As part of this effort, the recently published 2025 update of the Regulatory Science Research Needs highlights key areas for research across several domains including clinical trials, regulatory system evolution, developments for specific medicines, and animal health. By monitoring and analyzing its regulatory activities (for instance, EMA’s experience with scientific advice for advanced therapies, authorization of adapted COVID-19 vaccines, and accelerated regulatory approval of cancer treatments), EMA identifies what works well and what needs further work. Results can provide valuable information for developers to refine medicines research and for regulators to adapt their tools to the use of new research methods and medicine platforms and the characteristics of different patient populations. For example, through its committees and working groups, EMA prepares and updates regulatory guidelines that provide a harmonized approach to help medicines developers prepare their marketing authorization applications (for instance, on the development of medicines containing genetically modified cells) and issues opinions on the acceptability of novel methods used during medicines R&D (for instance, the use of a digital endpoint in clinical trials in Duchenne muscular dystrophy), which ultimately supports both medicines innovation and people with unmet medical needs.

Essential to fostering improvement in regulatory science is proactive engagement with all stakeholders. For many years, EMA has engaged in regulatory science research by contributing to external research projects, such as those set up under the European research framework in general and the Innovative Health Initiative (IHI) in particular. Examples of recent achievements of such projects are VAC2VAC, resulting in forgoing the pyrogen tests in rabbits, and INNODIA, for establishing a master protocol now widely used for accelerating diabetes research. Such projects have mainly been carried out by large consortia comprised of public and private stakeholders who work together in the pre-competitive R&D space to achieve common objectives to advance public health. EMA’s participation benefits the consortia’s outputs but also directly supports the agency in its mission to foster scientific excellence and advance regulatory science. This is further realized through a range of activities, including among others the qualification of novel methodologies for medicine development, issuing of letters of support, collaborating on scientific publications, and hosting or contributing to scientific and stakeholder workshops.

To drive progress in research and in the collaboration between academic researchers and regulators in the field of regulatory science, in 2025 EMA and the Heads of Medicines Agencies (HMA) launched the European Platform for Regulatory Science Research. The platform introduces a new mechanism for regular and iterative engagement between researchers and regulators to foster collaboration between academic research groups and regulatory scientists working at regulatory agencies and in the not-for-profit sector in the EU and beyond. Together, they can identify pressing research gaps in regulatory science and how to work together to address them. This will not only improve the overall understanding of research needs but also support the relevance and usefulness of research outputs so that they can be effectively applied in the work of regulators and developers.

Finally, to foster regulatory science progress with medicines developers, EMA hosts regular meetings between regulators and industry stakeholder organizations. This allows for constructive exchanges on general updates, on specific processes, and on opportunities for continuous improvement. In addition, individual pharmaceutical companies and regulators help to capture innovative and disruptive technologies, and anticipate the research methods and scientific expertise needed to assess future medicines, in portfolio and technology meetings.

We are at the next threshold of significant change in medicines development and regulation, with the ongoing revisions of the EU pharmaceutical legislation, which is intended to improve availability, accessibility, and affordability of medicines across Member States, while promoting innovation and environmental sustainability in Europe. It is essential for developers and regulators to have the best tools for bringing new medicines to patients and to foster an environment conducive to keeping pace with scientific and technological advances. By promoting and continuing to foster excellence in regulatory science, we can help ensure that the promises of medicines research innovation are translated to the availability of better developed, high-quality, safe, and effective medicines for patients.