CHAPTER 2: Key figures in 2024
Human medicines
In 2024, EMA's work across the medicines' lifecycle helped to guide innovative treatments to market that strengthen public health. The Agency supports developers at every stage of medicine development, helping to boost innovation and research by offering expertise before, during and after marketing authorisation.
Supporting research and development
Scientific advice
EMA provides guidance and support to medicine developers. This includes scientific and regulatory information on how to design and run clinical trials, compliance standards and obligations and incentives for developers of specialised medicines.
During a medicine's development, a developer can request guidance and direction from EMA on the best methods and study designs to generate robust information on how well a medicine works and how safe it is. This is known as scientific advice. Scientific advice is one of the Agency's key instruments for supporting the development of high-quality, effective and safe medicines, for the benefit of patients. Early dialogue and scientific advice lead to better development plans, promote the collection of high-quality data and, most importantly, help to ensure that patients only take part in those clinical trials that are likely to be robust enough to generate data that are relevant to support the evaluation of a marketing authorisation application or extension of indication.
In 2024, EMA received a total of 635 requests for scientific advice. Among these, ten were for COVID-19 medicines or vaccines. Protocol assistance is the special form of scientific advice for developers of designated orphan medicines for rare diseases. The requests for protocol assistance increased by 10 %, from 119 requests in 2023 to 131 in 2024.
PRIority Medicines (PRIME) programme
PRIME aims to support and optimise medicine development so that patients who have no or only unsatisfactory treatments for their disease have access to new medicines that have the potential to make a difference and enable them to live healthier lives. In 2024, EMA received 58 PRIME eligibility requests, 12 % more than in 2023, and adopted 56 recommendations, 17 % more than in 2023. The Agency received 42 requests for scientific advice for PRIME products, a higher number than in 2023, when 38 requests were received. PRIME is meant for the most promising medicines and EMA focuses its attention on medicines that have the potential to bring a major therapeutic advantage. That is why only a limited number of applications are accepted into the scheme. The acceptance rate in 2024 was 27 %, or 15 out of 56 requests. Six PRIME-designated medicines were recommended for approval (Beqvez, Fabhalta, Ixchiq, Seladelpar Gilead, Voydeya and Winrevair).
Recommendations for marketing authorisation
Applications for initial evaluation
EMA's committee for human medicines, the CHMP, carries out robust scientific evaluations of medicines and issues recommendations for the European Commission, which ultimately decides whether or not to authorise a medicine for marketing throughout the EU.
Activities in the initial evaluation of marketing authorisation applications for new medicines, which have never been authorised before, range from the pre-submission discussion with future applicants, through to the evaluation by the CHMP and the granting of the marketing authorisation by the European Commission.
A total of 121 applications were received in 2024.
Outcome of initial evaluation1
| Therapeutic area/ Product name | New active substance | PRIME | Orphan | ATMP | Biosimiliar | Generic | Accelerated assesment | Conditional approval | Exceptional circumstances |
|---|---|---|---|---|---|---|---|---|---|
| Cancer | |||||||||
| Apexelsin | |||||||||
| Augtyro | |||||||||
| Avzivi | |||||||||
| Axitinib Accord | |||||||||
| Balversa | |||||||||
| Cejemly | |||||||||
| Dasatnib Accord Healthcare | |||||||||
| Elahere | |||||||||
| Enzalutamide Viatris | |||||||||
| Eribulin Baxter | |||||||||
| Fruzaqla | |||||||||
| Hetronifly | |||||||||
| Ituxredi | |||||||||
| Korjuny | |||||||||
| Lazcluze | |||||||||
| Loqtorzi | |||||||||
| Nilotinib Accord | |||||||||
| Ordspono | |||||||||
| Pomalidomide Accord | |||||||||
| Pomalidomide Krka | |||||||||
| Pomalidomide Teva | |||||||||
| Pomalidomide Zentiva | |||||||||
| Tizvenis2 | |||||||||
| Truqap | |||||||||
| Tuznue | |||||||||
| Vyloy | |||||||||
| Welireg | |||||||||
| Zynyz | |||||||||
| Cardiovascular | |||||||||
| Beyonttra | |||||||||
| Jeraygo | |||||||||
| Neoatricon | |||||||||
| Winrevair | |||||||||
| Yuvanci | |||||||||
| Dermatology | |||||||||
| Anzupgo | |||||||||
| Nemluvio | |||||||||
| Diagnostic agents | |||||||||
| GalliaPharm | |||||||||
| Siiltibcy | |||||||||
| Tauvid | |||||||||
| Theralugand | |||||||||
| Endocrinology | |||||||||
| Awiqli | |||||||||
| Emcitate | |||||||||
| Jubbonti | |||||||||
| Obodence | |||||||||
| Osenvelt | |||||||||
| Stoboclo | |||||||||
| Wyost | |||||||||
| Xbryk | |||||||||
| Zegalogue | |||||||||
| Gastroenterology/Hepatology | |||||||||
| Iqirvo | |||||||||
| Kayfanda | |||||||||
| Seladelpar Gilead | |||||||||
| Haematology/Haemostaseology | |||||||||
| Adzynma | |||||||||
| Alhemo | |||||||||
| Altuvoct | |||||||||
| Beqvez | |||||||||
| Eltrombopag Viatris | |||||||||
| Fabhalta | |||||||||
| Hympavzi | |||||||||
| Piasky | |||||||||
| Rytelo | |||||||||
| Ryzneuta | |||||||||
| Voydeya | |||||||||
| Zefylti | |||||||||
| Immunology/Rheumatology/Transplantation | |||||||||
| Absimky | |||||||||
| Andembry | |||||||||
| Apremilast Accord | |||||||||
| Avtozma | |||||||||
| Eksunbi | |||||||||
| Fymskina | |||||||||
| Imuldosa | |||||||||
| Otulfi | |||||||||
| Pyzchiva | |||||||||
| Steqeyma | |||||||||
| Tofidence | |||||||||
| Wezenla | |||||||||
| Yesintek | |||||||||
| Infections | |||||||||
| Akantior | |||||||||
| Emblaveo | |||||||||
| Exblifep | |||||||||
| Kavigale | |||||||||
| Metabolism | |||||||||
| Agilus | |||||||||
| Neurology | |||||||||
| Buprenorphine Neuraxpharm | |||||||||
| Dimethyl fumarate Accord | |||||||||
| Dimethyl fumarate Mylan | |||||||||
| Dimethyl fumarate Neuraxpharm | |||||||||
| Leqembi | |||||||||
| Qalsody | |||||||||
| Wainzua | |||||||||
| Ophthalmology | |||||||||
| Afqlir | |||||||||
| Ahzantive | |||||||||
| Baiama | |||||||||
| Eydenzelt | |||||||||
| Lytenava | |||||||||
| Opuviz | |||||||||
| Ranibizumab Midas | |||||||||
| Vevizye | |||||||||
| Pneumology/Allergology | |||||||||
| Eurneffy | |||||||||
| Gohibic | |||||||||
| Nintedanib Accord | |||||||||
| Omlyclo | |||||||||
| Psychiatry | |||||||||
| Niapelf | |||||||||
| Paxneury | |||||||||
| Tuzulby | |||||||||
| Uro-nephrology | |||||||||
| Filspari | |||||||||
| Obgemsa | |||||||||
| Vaccines | |||||||||
| Celldemic | |||||||||
| Fluad | |||||||||
| Flucelvax | |||||||||
| Fluenz | |||||||||
| Incellipan | |||||||||
| Ixchiq | |||||||||
| Kostaive | |||||||||
| mResvia | |||||||||
| Penbraya |
In 2024, EMA recommended 114 medicines for marketing authorisation. Of these, 46 had a new active substance that had never previously been authorised in the EU.
The CHMP adopted negative opinions for five medicines in 2024:
- Cinainu, for the treatment of moderate-to-severe alopecia areata, a disease causing hair loss of the scalp or other parts of the body;
- Kizfizo, for the treatment of neuroblastoma, a rare cancer that forms from immature nerve cells;
- Masitinib AB Science, for the treatment of amyotrophic lateral sclerosis, a rare disease of the nervous system leading to loss of muscle function and paralysis;
- Nezglyal, for the treatment of paediatric and adult male patients aged two years and older with cerebral adrenoleukodystrophy, a genetic condition that damages the membrane that covers nerve cells in the brain and spinal cord;
- Syfovre, for the treatment of geographic atrophy secondary to age-related macular degeneration, a progressive retinal macular disease causing gradual vision impairment mainly in elderly people.
The applications for eight medicines were withdrawn by the applicants prior to the CHMP adopting an opinion, in most cases because the data included in the application were insufficient to support a marketing authorisation.
Applicants for 60 % of the medicines granted a positive opinion by the CHMP in 2024 had received scientific advice during the development phase of their medicine. The figure rises to 79 % for medicines with a new active substance.
Average assessment time
EMA has a maximum of 210 days to carry out its assessment. Within this time frame, the CHMP must issue a scientific opinion on whether the medicine under evaluation should be authorised. During the assessment, concerns with the application may be identified, requiring further information or clarification from the company. In this case, the clock is stopped to give the company time to reply to the Agency. Once the reply is received, the countdown continues.
Once the reply is received, the countdown continues. Once issued, the CHMP opinion is transmitted to the European Commission, which has the ultimate authority to grant a marketing authorisation and will take a decision within 67 days of receipt of the CHMP opinion.
The overall total time required for the centralised procedure, from the start of the evaluation process to the adoption of a decision by the European Commission, was an average of 434 days in 2024, lower than in 2023 (465 days). The overall total time for medicines that had received scientific advice was 412 days.
For medicines evaluated under accelerated assessment, the total time from start of assessment until granting of authorisation was reduced by more than eight months (from 434 to 216 days).
Post-authorisation activities
In 2024, the CHMP gave 90 positive recommendations for an extension of the therapeutic indication of already authorised medicines. These included 39 medicines in the oncology area.
The product information for 401 authorised medicines was updated as new safety data were made available and assessed by EMA.
Safety monitoring of medicines
EMA and EU Member States are responsible for coordinating the EU's safety monitoring of medicines, also known as pharmacovigilance. Regulatory authorities constantly monitor the safety of medicines and can take action if there is plausible evidence that a medicine's safety profile or benefit-risk balance has changed since it was authorised. EMA's safety committee, the PRAC, plays a key role in overseeing the safety of medicines in the EU, covering all aspects of safety monitoring and risk management.
The Agency's main responsibilities in relation to the safety monitoring of medicines include coordination of the European pharmacovigilance system, setting standards and guidelines for pharmacovigilance, providing information on the safe and effective use of medicines, detecting new safety issues for centrally authorised products (CAPs), managing assessment procedures, e.g. for periodic safety update reports (PSURs), and the operation and maintenance of the EudraVigilance system.
EudraVigilance
Both EMA and the NCAs are legally required to continuously monitor the adverse drug reaction (ADR) data reported to EudraVigilance to determine whether new or changed risks have been identified, and whether these risks have an impact on a medicine's overall benefit-risk balance.
Over 1.7 million ADR reports were submitted to EudraVigilance in 2024, representing a slight decrease (8 %) compared with 2023.
63 % of all reports in EudraVigilance originated outside the EEA.
The share of reports submitted by European patients and consumers in 2024 also decreased considerably compared to 2022 and 2023, and is more in line with pre-pandemic figures.
The considerably higher rates of ADR reports, including from patient reporting, during the pandemic were a result of the mass vaccination campaigns and the heightened awareness of the importance of reporting any suspected side effects.
Signal detection
A safety signal is information on a new or known adverse event that is potentially caused by a medicine and warrants further investigation. Signals are generated from several sources, such as spontaneous reports of suspected adverse reactions, clinical studies and the scientific literature. The evaluation of a safety signal is a routine pharmacovigilance activity to establish whether there is a causal relationship between a medicine and a reported adverse event.
In cases where a causal relationship is confirmed or considered likely, regulatory action may be necessary. This mainly comprises changes in the information on medicines available for patients (in the package leaflet) and prescribers (in the summary of product characteristics).
In 2024, 1,254 potential signals were reviewed by EMA, a decrease of 8 % compared to 2023. Approximately 74 % of these signals originated from monitoring the EudraVigilance database, highlighting its central role for safety monitoring. The PRAC assessed 71 signals. Thirty-nine of these were validated by EMA and 32 by Member States. In addition to signal detection activities and assessments at PRAC level, experts from the NCAs, in collaboration with EMA, provided major contributions to the development of signal detection methods and continuous process improvement.
Outcome of signal assessment
1,254 potential signals were identified by EMA and underwent an initial confirmatory and prioritisation review;
71 confirmed signals were prioritised and assessed by the PRAC:
- Of these 71 signals, 39 were detected and validated by EMA;
- 32 were detected and validated by EU Member States;
Out of 71 confirmed and validated signals
- 32 signals led to a product information update;
- 14 signals led to a recommendation for routine pharmacovigilance; and
- 25 signals were undergoing review by the PRAC at the end of 2024 as further data were required.
Periodic safety update reports (PSURs)
Marketing authorisation holders are required to submit a report on the evaluation of a medicine's benefit-risk balance to the regulatory authorities at regular, predefined intervals following the authorisation of a medicine. These reports summarise data on the benefits and risks of a medicine and take into consideration all studies carried out with it, both in authorised and unauthorised indications.
The Agency is responsible for procedures supporting the analysis of these reports for both CAPs and for nationally authorised medicines (NAPs) that are authorised in more than one Member State. These reports are called PSURs. When the assessment procedure involves more than one medicinal product with the same active substance, the procedures are referred to as periodic safety update single assessments or PSUSAs.
In 2024, the PRAC started the assessment of 910 PSURs and PSUR single assessment procedures (PSUSAs), of which 26 % represent single assessments of active substances only contained in NAPs. The PRAC issued 913 recommendations based on the assessment of PSURs and PSUSAs, of which 27 % consisted of single assessments of active substances only contained in NAPs. 19 % of assessments led to changes in the product information to optimise the safe and effective use of medicines by patients and healthcare professionals.
PSURs and PSUSAs finalised
| 2020 | 2021 | 2022 | 2023 | 2024 | |
|---|---|---|---|---|---|
| PSURs - standalone (CAPs only) finalised | 516 | 575 | 542 | 584 | 627 |
| PSURs – single assessment finalised | 258 | 336 | 318 | 275 | 283 |
| PSURs – single assessment (CAPs with NAPs) finalised | 49 | 49 | 46 | 38 | 49 |
| PSURs – single assessment (NAPs only) finalised | 209 | 287 | 272 | 237 | 234 |
| Total outcomes | 774 | 911 | 860 | 859 | 910 |
PRAC outcomes of PSURs and PSUSAs
| 2020 | 2021 | 2022 | 2023 | 2024 | |
|---|---|---|---|---|---|
| Maintenance | 630 | 748 | 720 | 718 | 735 |
| NAPs only | 161 | 226 | 216 | 196 | 176 |
| CAPs/NAPs and CAPs only | 469 | 522 | 504 | 522 | 559 |
| CHMP variation | 144 | 163 | 140 | 128 | 178 |
| NAPs only | 49 | 61 | 56 | 43 | 68 |
| CAPs/NAPs and CAPs only | 209 | 102 | 84 | 85 | 110 |
| Total outcomes | 774 | 911 | 860 | 846 | 913 |