Presentation (CTI)

OBJECTIVES: This study aimed to assess how recent trends in medicines development are reflected in the approvals by the European Medicines Agency (EMA). Emphasis was given to evolution of regulatory pathways and the emergence of new mechanisms of action.
METHODS: Data on new medicines were collected from the EMA’s Annual Reports. Medicines approved as “new active substance” (NAS) were included. The number of new medicines in anatomic-therapeutic-chemical (ATC) classification system belonging to fourth-level class X (“other”) was used as a proxy for new mechanism of action. The analysis compared subperiods 2015-2019 and 2020-2024.
RESULTS: A total of 338 NASs met the inclusion criteria (147 in 2015-2019 vs. 191 in 2020-2024). The dominance of ATC main group L (antineoplastic and immunomodulating agents) continued to grow, increasing from 38% to 43% of approvals. This trend was largely driven by subgroup L04 (immunosuppressants), which increased from 7% to 12%. The proportion of NASs with a new mechanism of action increased from 23% (2015-2019) to 32% (2020-2024). Within this subset, the share of medicines belonging to group L increased from 26% to 37%, and the proportion of subgroup L01FX (other monoclonal antibodies and antibody drug conjugates) increased from 9% to 18%. A notable shift in regulatory strategy was observed. The use of accelerated assessment decreased from 12% (2015-2019) to 1% (2020-2024). In contrast, the role of other regulatory pathways increased. These included conditional marketing authorisations (3% vs. 16%), approval under exceptional circumstances (4% vs. 7%), and PRIME designations (1% vs. 15%).
CONCLUSIONS: Antineoplastics and immunomodulating agents have increased their share both overall and among innovative therapies, highlighting their growing importance in medicines development. The transition from accelerated assessment to more sophisticated regulatory pathways signals a broader shift in how innovation is managed within the regulatory system.