Ground-breaking Approvals and Medicines Innovation
The European Medicines Agency (EMA) marked a historic year, recommending 114 new medicines for marketing authorisation—its highest total in over a decade. Among those, 46 featured a completely new active substance, reflecting significant innovation and a commitment to addressing urgent public health needs. Noteworthy advancements included the approval of the first medicine for early Alzheimer’s disease, novel forms of adrenaline for allergic reactions, targeted therapies for rare conditions such as von Hippel-Lindau tumors, and new antibiotics for severe infections. EMA also recommended several new vaccines, such as those for Chikungunya and respiratory syncytial virus (RSV), and expanded adolescent access to mpox vaccination. Oncology stood as the most active area, with 28 oncology drug approvals and an equal number of biosimilar recommendations, which enhance treatment access for major diseases like cancer, osteoporosis, and autoimmune conditions.
Regulatory Reforms, Guidance, and Trial Infrastructure
EMA’s regulatory landscape underwent significant transformation in 2024, introducing measures to accelerate medicine access and make trials more efficient. The Agency implemented a streamlined application process, reducing assessment timelines for medicines and introducing new, harmonised fee structures. Recent regulation (EU) 2024/568 drove fee increases for regulatory activities—including clinical trial assessments—but also provided reductions or exemptions for small and mid-sized enterprises (SMEs) and products targeting unmet medical needs or rare diseases. This move aims for greater predictability and transparency in regulatory planning, especially for companies with multi-type product portfolios. The revised approach encourages developers to optimise their submissions while balancing rising costs with newly available incentives.
EMA continued to shape trial design and oversight through extensive updated guidance. Its Committee for Medicinal Products for Human Use (CHMP) remains central in assessing clinical trial data, relying on rigorous scientific criteria for quality, safety, and efficacy. The Clinical Trials Regulation and the Accelerating Clinical Trials in the EU (ACT EU) initiative are reshaping clinical research, supporting adoption of complex and adaptive trial designs, real-world data use, greater transparency, and efficient multinational trials. Additionally, EMA strengthened collaboration with EU national agencies to support public health emergency research and rapid setup of large, harmonised studies—lessons learned from prior emergencies are now driving new standards for setup and data sharing.
2024 also saw the adoption of pivotal guidelines for advanced therapy medicinal products (ATMPs) such as gene and cell therapies, clarifying requirements for exploratory and confirmatory trials and increasingly aligning with international standards. EMA’s focus on quality, non-clinical, and clinical requirements supports innovation while ensuring robust data and patient safety.
Emphasis on Safety, Transparency, and Diversity
EU-wide pharmacovigilance measures remained a top priority, with EMA and its Pharmacovigilance Risk Assessment Committee (PRAC) actively monitoring product safety and issuing timely updates to labelling and patient information. The regulatory environment also supported a strong push for clinical trial transparency, data publication, and inclusion of diverse populations—directing sponsors to innovate and broaden access in line with Europe’s public health objectives.
Looking Forward: Harmonisation and Opportunity
EMA’s ongoing reforms signal a dynamic future for clinical research in Europe, combining speed, scientific rigor, and patient focus. The Agency’s expanding mandate includes new legislation, digitalisation initiatives, and continued advocacy for adaptive trials, biosimilars, and advanced therapies. As the regulatory environment evolves, sponsors and developers will find both new challenges and unprecedented opportunities—especially those who prioritise submission efficiency, quality management, and patient safety.
