Human medicines

In 2023, EMA recommended 77 medicines for marketing authorisation. Of these, 39 had a new active substance which had never previously been authorised in the EU.

The CHMP adopted negative opinions for three medicines in 2023: 

• Albrioza, for the treatment of amyotrophic lateral sclerosis, a rare neurological disease affecting nerve cells in the brain and spinal cord that control voluntary muscle movement;
• Lagevrio, for the treatment of COVID-19 in adults; and
• Sohonos, to treat fibrodysplasia ossificans progressiva, a rare genetic disease that causes extra bone to form in places outside the skeleton, such as in joints, muscles, tendons and ligaments, leading to progressively decreasing mobility and other severe impairments. 

The applications for 18 medicines were withdrawn by the applicants prior to the CHMP adopting an opinion, in most cases because the data included in the application were insufficient to support a marketing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 67 % of the medicines granted a positive opinion by the CHMP in 2023 had received scientific advice during the development phase of their medicine. The figure rises to 88 % for medicines with a new active substance.

Average assessment time

EMA has a maximum of 210 active 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 counting of the days 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 start of the evaluation process to the adoption of a decision by the European Commission, was an average of 465 days in 2023, similar to 2022 (461 days). The overall total time for medicines that had received scientific advice was 430 days. 

For medicines evaluated under accelerated assessment, the total time from start of assessment until granting of authorisation was reduced by more than 8 months (from 465 to 215 days), potentially facilitating the subsequent decision-making steps at a national level and, ultimately, patient access.

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 1.9 million ADR reports were submitted to EudraVigilance in 2023, representing a substantial decrease (34 %) compared with 2022. 

Over 60 % of all reports in EudraVigilance originated outside the EEA. 

The share of reports submitted by European patients and consumers in 2023 also decreased considerably compared to 2021 and 2022, 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 2023, 1,364 potential signals were reviewed by EMA, a decrease of 15 % compared to 2022. Approximately 74 % of these signals originated from monitoring the EudraVigilance database, highlighting its central role for safety monitoring. The PRAC assessed 71 signals and, of these, EMA validated 39. The number of signals validated by Member States and assessed by the PRAC increased to 32, from 25. In addition to signal detection activities and assessments at PRAC level, experts from the NCAs, in collaboration with EMA, provided a major contribution to the development of signal detection methods and continuous process improvement.