Impact of Regulatory Measures on the Approval Timelines of Advanced Therapy Medicinal Products by the European Medicines Agency

Simonita Alaburde¹Justinas Ivaska^1,2Greta Kaspute¹Tatjana Ivaskiene^1*

• ¹State Research Institute Center for Innovative Medicine, Vilnius, Lithuania
• ²Faculty of Medicine, Clinic of Ear, Nose, and Eye Diseases, Vilnius University, Vilnius, Lithuania

The study examines the impact of regulatory tools, including PRIority MEdicines (PRIME) scheme, on the marketing authorization (MA) timeline of advanced therapy medicinal products (ATMPs) approved by the European Medicines Agency (EMA). A retrospective analysis of EMA-approved ATMPs was conducted using publicly available European public assessment reports. Timelines from submission to approval, regulatory pathways, scientific advice frequency, and use of supportive mechanisms such as PRIME and orphan designation were analyzed. The findings indicate that ATMPs benefiting from the PRIME scheme achieved MA approximately one year faster than those outside the scheme, as developers could respond to regulatory requirements more efficiently. This underscores the scheme’s role in expediting patient access to innovative treatments. The study highlights the value of early and frequent engagement with regulatory authorities, emphasizing the need for tailored regulatory frameworks to facilitate smoother approval processes. These insights are critical for manufacturers navigating regulatory pathways, enabling better planning and optimization of approval strategies. By demonstrating the tangible benefits of PRIME, the research supports its continued implementation in accelerating access for patients with critical medical needs.