Introductionby Emer Cooke

Benefits for EU patients

Notable approvals include the first medicine to treat early Alzheimer's disease, a nasal spray adrenaline for allergic reactions, a treatment for tumours associated with von Hippel-Lindau disease, and two new antibiotics for severe infections. Cancer was the leading therapeutic area, with 28 recommendations for oncology products. We also approved Durveqtix, a new gene therapy for haemophilia B, Qalsody for a rare motor neurone disease (ALS), and the first vaccine to protect adults from Chikungunya, a virus transmitted to people by mosquitoes.

There were also 28 recommendations for new biosimilar products, covering a wide range of diseases, including several types of cancer, osteoporosis, macular degeneration and diseases that involve an abnormal immune response like plaque psoriasis, ulcerative colitis and Crohn's disease. This is certainly good for European patients, as biosimilars make treatments more accessible and can provide broader access to potentially life-changing medicines.

Progress for animal health

In 2024, EMA also recommended 25 veterinary medicines for marketing authorisation – the highest number ever. Among them, 14 were vaccines, including seven that had been developed through a biotechnological process.

With bird flu continuing to be a public health concern in 2024, EMA's veterinary medicines committee, the CVMP, recommended a new vaccine against the strains of highly pathogenic avian influenza that were circulating last year. EMA works with public health authorities in the EU and worldwide to monitor the risk and prepare to respond in the event of a public health emergency.

EMA also certified the first veterinary vaccine platform technology master file (vPTMF) in November 2024. Such platforms play an important role in animal and public health preparedness because they can be adapted rapidly, thus speeding up the development and approval of new veterinary vaccines in the EU in response to emerging diseases.

A coordinated approach to shortages

In 2024, medicine shortages remained a high priority on the public health agenda. The reasons for shortages are always multifaceted but a specific case of difficult supply-and-demand dynamics emerged in 2024 with shortages of GLP-1 receptor agonist medicines. Increased demand for these medicines for both diabetes and obesity, coupled with significant supply challenges, jeopardised treatment programmes for many patients.

To address this issue, we launched coordinated actions across the EU. We stepped up communications to influence prescribing and consumption behaviours, ensuring that those who needed the medicines received them. When it was most needed, we also oversaw the redistribution of stocks among EU Member States to prevent patients from missing their medications. By closely monitoring the market situation, including the status of current and anticipated shortages, we could see where our mitigation actions were effective.

At the end of 2024, we organised the soft launch of the European Shortages Monitoring Platform (ESMP), introducing a limited set of functionalities for marketing authorisation holders. A full launch followed in January 2025. This platform enables the fast exchange of information between regulators and pharmaceutical companies to support the management of shortages in the EU.

On the communications side, we hosted a media seminar towards the end of the year, bringing together EU journalists and EMA experts. By providing greater access to our experts, we aim to bridge the knowledge gap and prevent behaviours that contribute to medicine shortages.

Digitalisation: AI enablingour work

AI is influencing all parts of our lives today and continues to bring dramatic changes to all industries with important applications for medicines regulation. We continue to explore how AI systems can be used to bring benefits to patients, our broader regulatory system and to the work our scientists do in a safe, transparent, ethical and unbiased way.

We are proud of our proactivity in the AI field. With the HMA, we published our 2023-2028 multi-annual AI work plan early in 2024, to help the European Medicines Regulatory Network (EMRN) harness AI opportunities in the regulatory and medical field. We have several ongoing AI initiatives under this work plan that are already changing the way scientists work.

In March, for example, we launched Scientific Explorer, an AI tool to help EU regulators access information from thousands of scientific advice procedures to simplify their work, support their decision-making and save them time. In the field of rare diseases, CollaboRARE was launched in April. This is a co-creation between EMA and EURORDIS to develop and pilot an AI-based patient-validated methodology to capture and use patient experience data (PED) for orphan medicines. The validation by patient organisations of the PED gathered by AI will help further integrate the patient perspective into medicines development and evaluation.

As regulators learn more about how best to realise the potential of AI, we need to also understand and educate ourselves about how to manage possible risks, and keep training and preparing our staff for the future. As more use cases are implemented, we need to continue to ask the right questions and to challenge and course correct where necessary.

Clinical evidenceand real-world evidence

High-quality clinical evidence is at the heart of every well-informed decision on medicines. We know that our biggest opportunity for medicines development in Europe lies in cultivating a robust, modern clinical trials ecosystem. Through our Accelerating Clinical Trials in the EU initiative, or ACT EU, we continued to bring all the ecosystem's stakeholders together – patients, healthcare professionals, industry, regulators, academia and Member States.

The year 2024 was the last of the three-year transition period for the Clinical Trials Regulation. I appreciated all the efforts by NCAs and EMA that went into ensuring that clinical trials taking place in the EU were transitioned into the Clinical Trials Information System (CTIS), which support the flow of information between clinical trial sponsors, EU Member States, European Economic Area (EEA) countries and the European Commission platform. Work on the simplification and modernisation of CTIS can now continue as a priority.

CTIS will also become a World Health Organisation (WHO) Primary Registry of the WHO International Clinical Trials Registry Platform (ICTRP), a major step forward from its previous status as data provider. This new status increases the recognition of CTIS for data sharing, reliability and promoting transparency.

While clinical trials remain our first source for evidence generation, we continue to develop real-world evidence (RWE) capabilities to fill knowledge gaps and complement the picture. In 2024, we continued to generate evidence based on knowledge and expertise, embracing diverse data and methods, and exploring, enabling and validating new approaches. We also made further progress to integrate RWE into our decision-making so that it brings value to public health in the EU. The Data Analysis and Real World Interrogation Network (DARWIN EU) became fully operational as the main pathway to generate RWE; the network now has access to 160 million patients' health data in 16 European countries and is generating studies that can tell us how medicines perform in the real world.

In 2024, these included research on the prevalence of rare blood cancers in Europe, the prescription of antibiotics and a study examining the characteristics of patients who have been prescribed GLP-1 receptor agonists, used for diabetes and weight management, and how these have changed over the past ten years.

Antimicrobial resistance: addressing an invisible threat

WHO launched a public health campaign in 2024 on the theme that antimicrobial resistance (AMR) is invisible, but its victims are not. This campaign highlights the silent crisis that AMR has become, causing 35,000 deaths annually in Europe and imposing significant costs on all systems. Coordinated action is essential to combat this health crisis.

The International Coalition of Medicines Regulatory Authorities (ICMRA) provides a unique platform for international regulators and other key stakeholders to develop strategic responses to antimicrobial resistance. At the 11th ICMRA Summit in November 2024, EMA co-chaired a crucial panel discussion on this topic where international regulators shared feedback and insights. This led to the publication of a statement reiterating the critical importance of preserving the effectiveness of antimicrobials and the crucial role regulators play in addressing the challenge collaboratively.

On the veterinary side we have a remarkable success story to tell. One of our past projects, the European Surveillance of Veterinary Antimicrobial Consumption (ESVAC) project, has significantly reduced sales of veterinary antibiotics in the EU. ESVAC was a voluntary project – a concerted effort by farmers, veterinarians and national authorities – that led to sales of veterinary antibiotics in Europe dropping by more than 50 % over 12 years. This demonstrates that we can reduce AMR through a combination of monitoring, collecting data and communication. These principles are now embedded in EU legislation. The collection of data on sales and use of veterinary antimicrobials is now mandatory for all Member States.

Collaboration acrossthe network

In 2024 we made a huge difference across the board to patients and animals in the EU, and we look ahead to 2025, our 30th anniversary, with renewed determination, enthusiasm and passion.

Through 30 years of operations our Agency has impacted millions of lives of Europeans for the better. This year, the Agency will maintain its focus on accelerating and optimising the assessment of key medicines, working collaboratively on the joint EU medicines agencies network strategy to improve both accessibility and availability of medicines, as well as working on future-proofing our activities in preparation for the new pharmaceutical legislation.

It will be another exciting year for medicines regulation. I am enormously grateful for all the wonderful stakeholders and partners whose support and collaboration inspires us and drives our network forwards in these challenging times.