Introduction by Emer Cooke

EMA also adopted two positive opinions for medicines for use in countries outside the EU: a new treatment option for the estimated 50 million young children with schistosomiasis, and a medicine used to treat sleeping sickness caused by trypanosoma rhodesiense.

COVID lessons: preparedness

Our work in the field of human medicines was shaped by the long tail of the COVID-19 pandemic in 2023. We continued to carefully monitor and evaluate the safety, efficacy and quality of treatments and vaccines against the changing nature of the virus.

We have embedded its important lessons in our work and mission: you can now read the main COVID-19 learnings report, which was published by EMA and the Heads of Medicines Agencies (HMA) in 2023. Several of its recommendations have already been implemented as part of EMA’s extended mandate, with the Agency assuming an enhanced role on preparedness to be more proactive on public health threats.

Veterinary medicines highlights

In 2023, we recommended 14 veterinary medicines for marketing authorisation. Of these, nine had a new active substance, which had not previously been authorised in the EU – a threefold increase compared to 2022. Nine were vaccines, including six new biotechnological vaccines (compared to one in 2022).

We have come a long way in our fight against antimicrobial resistance (AMR) in the veterinary area. We brought the European Surveillance of Veterinary Antimicrobial Consumption (ESVAC) project to a successful conclusion in 2023. The final report, published in November 2023, shows that the sales of antibiotics for animal use more than halved (53 %) between 2011 and 2022, according to data from 25 participating countries. The ESVAC concept has now been integrated into EU legislation, making the collection of data on sales and use of veterinary antimicrobials mandatory for all EU countries.

The fight against AMR continues, e.g. by looking at alternatives to antibiotics. The Committee for Veterinary Medicinal Products (CVMP) adopted a guideline to encourage the development of new phage therapy products to treat bacterial infections. It is another fundamental step towards the promotion of animal and human health as envisaged by the One Health approach.

Medicines shortages in the EU

Throughout the year, we made significant progress in managing the ongoing challenge of shortages throughout the EU. We saw the need for a coordinated approach between Member States during the winter season 2022/2023, with shortages of antibiotics and other critical medicines.

The changes we made to achieve a more proactive approach to this systemic challenge are now bearing fruit. In addition to an extensive supply and demand exercise on key antibiotics, we launched a series of co-ordinated actions across the EU throughout 2023. The Medicines Shortages Steering Group (MSSG) created a solidarity mechanism which allows Member States to support each other in the face of a critical medicine shortage. The group also published a toolkit with comprehensive recommendations for Member States to tackle shortages in their territory.

In December 2023, the EU regulatory network published the first version of the Union list of critical medicines to support efforts to ensure supply security and prevent shortages of more than 200 substances.

In addition, there were also significant communication efforts to promote best practices by industry, healthcare professionals and patients to contribute to the prevention of shortages.

The results of these actions are noticeable. In January 2023, around 17 Member States reported critical shortages of antibiotics, down to seven Member States in January 2024. In most cases these shortages affect individual products only. We will continue to monitor and act where the need arises in the EU.

Strategic focus areas in 2023

In this report, we focus on three strategic areas in 2023: cancer medicines, data-driven medicine regulation and transparency and communication. In 2023, we stepped up our support for the EU Beating Cancer Plan with the launch of a new initiative, which has become known as the ‘Cancer Medicines Pathfinder.’

This initiative explores how EMA can enable high-quality, robust and rapid assessment of key medicines overall, applying the learnings from the COVID-19 pandemic. Cancer was selected as a pathfinder for this initiative because it is a therapeutic area with a high rate of innovation and scientific progress, but also a high unmet medical need.

In 2023, we made significant progress in creating and using better data to translate innovation into medicines that reach patients. The work of the Big Data Steering Group explores the best ways to integrate data analysis into its assessment processes to improve our decision making. Two years into its operation, the Data Analysis and Real World Interrogation Network EU (DARWIN EU) now boasts a network of 20 data partners from 12 EU Member States that together provide access to data from 130 million patients.

We also launched a draft Reflection Paper on Artificial Intelligence, which sets out our thinking around this huge technological shift in our societies. Furthermore, we continue to strengthen the EU as a destination for clinical trials. The Accelerating Clinical Trials in the EU initiative (ACT EU) is now in full swing. We have initiated a new multi-stakeholder platform to step up the dialogue with all key stakeholders in EU clinical trials.

Finally, we outline the role of trust, transparency and communication in this report. We always aim to be as open as possible about the data that informs the decisions EMA makes. This is why I am so pleased that EMA has resumed its clinical data publication initiative in 2023 beyond COVID-19 medicines, after several years of suspension linked to business continuity measures.

Collaboration beyond the EU

The COVID-19 pandemic has clearly demonstrated that public health can only be thought of in global terms. International collaboration is integral to medicines regulation, because we can better solve challenges by working together.

In 2023, we celebrated the 10th anniversary of the International Coalition of Medicines Regulatory Authorities (ICMRA), a network that was conceived to help tackle new and emerging human medicine, regulatory and safety challenges at the global level. ICMRA, which brings together the heads of 38 regulators, with the WHO as an observer, proved its value many times over during the COVID-19 pandemic.

Last year, EMA received a grant of ten million euros from the European Commission to support regulatory systems at national and regional level in Africa, and the setting up of the African Medicines Agency (AMA), in collaboration with African, European and international actors. This is an exciting collaboration. We will support the AMA, its technical committees, and regulators by sharing our experience in pooling resources and coordinating work to regulate medicines.

In 2023, we also expanded the scope of the OPEN initiative from COVID-19 vaccines and treatments to a wider range of medicines. OPEN was one of our success stories of the pandemic. It was established as a pilot in December 2020 to increase international collaboration and share scientific expertise on the evaluation of COVID-19 vaccines and therapeutics. Its expansion will facilitate patient access globally to medicines with the potential to address AMR, respiratory syncytial virus (RSV) infections, or newly diagnosed myelodysplastic syndromes (and other hereditary diseases).

Opportunity in transformation

Alongside all of these developments, in 2023 we welcomed the EU Pharma Review. This is a once-in-a-generation opportunity to transform and improve the way we do things to benefit the patients we serve. We are committed to using the momentum this creates to help ensure the EU regulatory framework is fit for new, innovative medicines, support better access to medicines for patients and help address major public health threats like AMR.

The future for medicine regulation in the EU is exciting, but we must stay in front of change and always be prepared for new challenges – not as they emerge, but long before that. We are continuously looking at how we can ensure that we have the right skills and expertise to deal with rapid scientific advancements within the network.

Finally, I would like to thank my many colleagues – EMA staff, experts from the Member States and partners in the EU institutions and sister agencies – for their work and dedication to the network. Behind all of the numbers in this annual report are people; and behind them is a network which has come out of one of the greatest healthcare challenges of our lifetime more proactive, resilient and better prepared.

I invite you to delve into these pages and look forward to your continued collaboration across our network to support the EU’s public health goals in 2024. Let the achievements documented here be a reminder of the real value of our scientific work. EMA is always committed to its mission to promote public health, drive scientific innovation and safeguard the well-being of patients across Europe. Thank you all for your continued support.