(AI) Comparison of FDA and EMA Regulatory Routes and Approval Timing for Personalized Medicines

Publication Number: 669

Background: One of every four drugs approved by the FDA in recent years was a personalized medicine tailored to patients based on their predicted response. Recently, we found that 95% of the personalized medicines approved by the FDA between 2017 and 2019 was granted at least one regulatory tool to accelerate market access. The EMA has similar regulatory tools available. However, FDA and EMA approval and use of regulatory tools might differ based on strategy and tool eligibility requirements.

Objectives: This study investigated the difference in FDA and EMA approval and use of regulatory tools for personalized medicines approved by the FDA between 2017 and 2020.

Methods: Approved new personalized medicines were identified through review of FDA Progress and Outlook Reports. EMA approvals for these drugs were identified on the EMA website. Regulatory tools were identified in FDA’s Novel Drug Therapy Approval reports and EMA’s Human Medicines Highlights reports. Data were analyzed descriptively in terms of the time between FDA and EMA approval, and the number, percentage, median and range of regulatory tools used.

Results: Of the 76 new personalized medicines approved by the FDA between 2017 and 2020, 60 (79%) were also approved by the EMA. EMA approval was before FDA approval for 8 medicines (13%; median 107, range 4-3262 days before) and after FDA approval for 52 medicines (87%; median 267, range 8-579 days after). Of the 60 medicines receiving FDA and EMA approval, 54 medicines (90%) used at least one FDA expedited review regulatory tool (median 2, range 0-4 tools) and 32 (53%) at least one EMA expedited review regulatory tool (median 1, range 0-3 tools). Of the 12 medicines approved under the FDA accelerated pathway, 10 (83%) underwent an EMA accelerated assessment or received conditional authorization or approval under exceptional circumstances. In contrast, of the 50 personalized medicines receiving an FDA priority review designation, only 7 received an EMA PRIME designation (14%). Also, of the 38 personalized medicines receiving an FDA breakthrough designation, only 5 (13%) were considered an advanced therapy medicinal product by the EMA. Thirty medicines (50%) received both FDA and EMA orphan designations and 13 medicines (22%) only an FDA orphan designation.

Conclusions: A majority of the personalized medicines approved by the FDA between 2017 and 2020 have received EMA approval, mostly after FDA approval. Expedited review and orphan designations were more often granted by the FDA than the EMA for the same drug. The results of this study indicate a trend towards seeking FDA guidance before EMA guidance during drug development.