Accelerating and optimising the assessment of medicines

Streamlining and simplifying assessment processes

Throughout 2025, EMA made significant efforts to streamline and simplify medicines assessment processes, enhancing efficiency while maintaining the rigorous scientific standards on which regulatory decisions depend. Overall, these initiatives reflect the Agency's continued commitment to reducing administrative burden and improving predictability for all stakeholders, ultimately accelerating patient access to safe and effective medicines.

A cornerstone of these efforts has been the medicines assessment report revamp project, which has modernised the structure and content of assessment documentation to improve clarity for decisionmakers and stakeholders. Specifically, key assessment templates were updated, and a more streamlined collaboration between assessment teams was introduced by enabling the co-authoring of documents. This initiative complements broader work to harmonise and optimise procedural aspects of the evaluation process.

In 2025, the Group for Internal Rules on Extensions of Clock Stops (GIREX) project achieved significant progress in shortening the time for applicants to respond to questions during the assessment process, or 'clock-stop' extension. The average clock-stop duration in 2025 was 159 days, down 46 days from the peak in 2022. This constitutes approximately a 22% reduction from 2022.

In addition, the pre-submission interaction group (Pre-SIG) project is also in the process of reshaping interactions between EMA, rapporteur teams and applicants during the pre-submission phase. This initiative aims to enhance submission predictability and dossier maturity by fostering earlier dialogue and setting clearer expectations, through mechanisms including joint pre-submission meetings with the EMA product team and the rapporteurs, reducing the likelihood of procedural issues that could delay assessments. The work of this group will build towards the implementation of the new pharmaceutical legislation. Additional streamlining measures implemented during 2025 include:

• A single rapporteurship pilot programme for biosimilar medicines: In 2025, EMA started piloting the appointment of a single CHMP rapporteur for less complex marketing authorisation applications for biosimilar medicines. The pilot was launched in March 2025 and will last for two years. The aim of the pilot is to make optimal use of the available expertise in the network, without lowering the quality of our assessment.
• New variations guidelines, developed with support from EMA and the national competent authorities in 2025, will facilitate quicker and more efficient processing of variations, benefiting both marketing authorisation holders and regulatory authorities. The guidelines will support the implementation of the new European Commission Variations Regulation that came into force in January 2025.
• The revamp of the Good Pharmacovigilance Practices (GVP) inspection programme has also enabled more efficient use of regulatory resources with the more extensive application of risk criteria when defining inspection frequency. The improved risk assessment model focuses inspection resources on high-risk and high-impact areas, enabling faster responses to emerging safety concerns while maintaining robust oversight of pharmacovigilance systems. The project also delivered substantial simplification in the way information is exchanged between EMA and NCAs by moving the GVP Inspection programme onto a shared platform directly available to all NCAs. This change facilitates online collaboration and more efficient use of both network and EMA resources.

Collectively, these initiatives represent a comprehensive approach to simplifying the regulatory environment, ensuring that EMA's assessment processes remain fit-for-purpose in an evolving medicines landscape while upholding strict European Union safety standards.

A new era for clinical trials in the EU

The year 2025 marked the full implementation of the Clinical Trials Regulation (CTR), following a three-year transition from the Clinical Trials Directive. Since 31 January 2025, all clinical trials in the EU operate under a single, unified legal framework. The regulation streamlines processes for trial authorisation and supervision, ensuring all sponsors follow harmonised procedures regardless of the location in the EU where the trial will be conducted. This strengthens Europe's position as an attractive place for clinical research.

Central to this transformation is the CTIS, which serves as the single-entry point for sponsors and regulators to submit, assess, and oversee trials across the EU. Managed by EMA, CTIS has received more than 13,000 initial applications since its launch in 2022, (2,844 in 2025) with more than 10,600 trials authorised by EU Member States.

In April 2025, the WHO designated CTIS as a primary registry in its International Clinical Trials Registry Platform (ICTRP) Registry Network. This designation confirms that CTIS meets high standards for data quality, accessibility, and governance, supporting global data sharing and reinforcing trust in clinical research. It also aligns EU clinical trials with requirements for publication in leading medical journals, further integrating them into the global research ecosystem. Throughout the year, work progressed on simplifying business rules and developing a long-term modernisation roadmap for the system.

CTIS is the single platform for information about ongoing trials. To improve access for doctors and patients to this information, EMA launched a new clinical trial map as part of the CTIS public website. With this interactive tool, users can search for ongoing trials by location and medical condition using lay language. Search results offer investigators' contact details, allowing patients to consult with their doctors and potentially enquire about enrolment on a trial. The trials map was initially launched in English and was expanded to 22 additional EU/EEA languages later in the year. Five more languages will be added in 2026.

The trials map was one of the initiatives developed under the Accelerating Clinical Trials in the EU (ACT EU) initiative. ACT EU is a collaboration between the European Commission, EMA, and NCAs, aimed at transforming how clinical trials are initiated, designed and run to promote the EU as a centre for clinical research. In 2025, the initiative published a three-year analysis of clinical trial applications in the EU/EEA which showed that the CTR transition was successfully completed, with 5,088 trials transferred to CTIS from the Clinical Trials Directive. The analysis also found that CTIS received around 200 new initial applications per month between January 2023 and January 2025, including about 80 multinational trials.

Looking ahead, the EU has set ambitious targets: aiming to add an additional 500 multinational authorised clinical trials over five years, from January 2026 until the end of 2030, and ensuring two-thirds of trials start recruiting within 200 days of application submission, compared to 50% today. These objectives build on ACT EU efforts and complementary initiatives such as CTR Collaborate, COMBINE, and MedEthicsEU, which aim to harmonise procedures and reduce administrative burden across the clinical research ecosystem.

A fast-track approach for authorising strategic multinational clinical trials, FAST-EU, was also launched under the leadership of the Heads of Medicines Agencies (HMA) at the end of 2025. This initiative is fully aligned with the shared goal of making Europe a more attractive destination for clinical research and improving timely access to innovative medicines for patients. EMA will support this effort with technical expertise and by facilitating processes through CTIS.

EMA continued piloting the use of individual patient-level data from clinical studies in marketing authorisation evaluations, aiming to gain a deeper understanding of the evidence, thereby improving its regulatory decisions. The pilot learnings will inform preparations in anticipation of systematic submissions of such data in the future.

Enhancing the useof real-world evidence

High-quality clinical evidence is at the heart of well-informed decisions on medicines. While clinical trials remain central, experts can use real-world data (RWD) from routine healthcare settings to generate real-world evidence (RWE). The use of RWE helps regulators address knowledge gaps and complement the evidence picture gained from clinical trials by enhancing their understanding of the use, safety and benefits of medicines.

Fully operational since 2024, DARWIN EU® is now the main pathway to generate RWE. DARWIN EU® provides the structure, data and tools to access relevant and reliable RWE on diseases, populations and medicine use and performance across Europe. In 2025, DARWIN EU® onboarded three new data partners, bringing the total to 33. These partners can provide data from over 180 million people across 16 European countries. Seven additional data partners were at an advanced stage of onboarding by the end of the year.

The number of studies conducted via DARWIN EU® continued to grow. In total, 67 studies were performed during the fourth year of operation of DARWIN EU®, which includes 52 studies newly initiated in 2025. Forty studies were completed in 2025. For example, a study on antibiotic prescribing practices across the EU helped inform and support the CHMP's May 2025 recommendations to revise how azithromycin is used, aiming to optimise its clinical use and minimise antimicrobial resistance. A study on respiratory syncytial virus (RSV) highlighted its impact on vulnerable populations such as infants and older adults and the unmet clinical need in certain groups, guiding EMA in preparing future vaccine effectiveness studies. Other notable work includes a study that supported the PRAC in confirming that the widely used antibiotic doxycycline is not associated with an increased risk of suicidality.

The results from the studies are shared with the relevant EMA committees and stakeholders to support the evaluation of medicines and are publicly disclosed in the HMA-EMA RWD study catalogue.

Leveraging the power of data for public and animal health – empowering safe and responsible use of artificial intelligence

Artificial intelligence (AI) is a transformative technology with immense potential across the medicinal product lifecycle, from drug development to pharmacovigilance.

In March 2025, the CHMP issued the first Qualification Opinion (QO) on an innovative development methodology based on AI. The tool, called AIM-NASH, helps pathologists analyse liver biopsy scans to determine the severity of metabolic dysfunction associated steatohepatitis (MASH). This marked the first time that EMA considered data generated with the assistance of an AI-based tool to be scientifically valid to support an application for marketing authorisation.

In view of the technology-driven explosion of data, we need to be strategically aligned across the EU in terms of data governance, management and AI-powered analysis tools to transform data into tangible benefits for public and animal health.

Peter Arlett, EMA's Head of Data Analytics and Methods Task Force and co-chair of the Network Data Steering Group (NDSG)

In May 2025, HMA and EMA published a joint workplan, Data and AI in medicines regulation to 2028. It sets out how the European medicines regulatory network plans to maximise data use, exchange and interpretation, improve access to data and evidence generation, and leverage use of artificial intelligence for better decision-making. It also provides a framework to address new legislation and initiatives in the EU, notably the new pharmaceutical legislation, the European Health Data Space (EHDS), the Interoperable Europe Act and the AI Act. A new Network Data Steering Group (NDSG), combining the former Big Data Steering Group and the Network Data Board, was set up to oversee the implementation of the workplan.

We are excited to join forces to harness data and AI to improve public and animal health across the EU and realise the vision of the network in its strategy to 2028. Through collaboration, stakeholder engagement, training and guidance, we aim to drive impactful outcomes throughout the workplan.

Karl Broich, president of the German Federal Institute for Drugs and Medical Devices (BfArM) and cochair of the Network Data Steering Group (NDSG)

As regulators continue to deepen their understanding of how best to harness the potential of AI, significant effort was invested across the regulatory network in 2025 to collect and prioritise AI use cases and explore existing or potential solutions for implementation. This work will inform the development of the Knowledge Mining and AI use cases roadmap in 2026.

A group focused on AI with industry stakeholders was established to facilitate open dialogue with industry on the development and use of AI in the medicines lifecycle.

Collaboration with stakeholders and partners on AI continued in 2025 at the international level and under the umbrella of ICMRA. At the European Union level, EMA is chairing the EU Agencies Network Working Group (EUAN WG) on AI.

The NDSG worked with the US FDA on a set of ten guiding principles for good AI practice in the lifecycle of human and veterinary medicines. The principles give broad guidance on AI use in evidence generation and monitoring, from early research and clinical trials to manufacturing and safety monitoring. The principles were published in early January 2026.

Still in the area of AI, EMA published in July 2025 its first AI Observatory Report which compiles the European medicines regulatory network's experience with AI during 2024 in enhancing productivity, automating tasks, and supporting data-driven decisions across a medicines lifecycle. In addition, the Agency published a compilation of examples of AI use in medicine regulation, as well as a horizon scanning short report, based on the review of scientific literature and EU-funded projects, which helps identify gaps, challenges and opportunities for integrating AI in medicine regulation.

Product master data is essential for strengthening the interoperability of the Network's data assets. The NDSG recognised the Product Management Service (PMS) as the shared source of product master data for all EU medicinal products, supporting EU-wide use cases (Medicinal Product Master Data for Better Regulation and Better Health – NDSG recommendations for human Product Master Data implementation and data management).