PP09-69: Analysis of Expedited Approval Pathways for Approved New Molecular Entities in Oncology
University of North Carolina United States
The objective of this study is to review new molecular entity (NME) approvals in oncology in the United States (US) and European Union (EU) and to analyze trends in use of expedited approval pathways in both regions.
We searched the US Food and Drug Administration and European Medicines Agency public databases from 2012-2019 to identify NME approvals in oncology. We then analyzed trends in sponsors’ use of expedited approval pathways.
This retrospective study evaluated oncology NME approvals in both the US (n=91) and EU (n=87). Most US approvals were classified as small molecules (60, 66%), utilized the priority review pathway (71, 78%), and gained orphan designation (64, 70%). The US average review time without using an expedited pathway was 345.1 days compared to 208.4 days using at least one pathway. For the US from 2012-2019, a negative trend was observed for fast track, and a positive trend was observed for accelerated approval (AA), priority review (PR), and breakthrough therapy designation (BTD). The top 4 approvals by cancer indication in the US used the following pathways: priority review for breast cancer (7/10), priority review for non-small cell lung cancer (NSCLC; 7/8), fast track for melanoma (4/8), and priority review and accelerated approval for multiple myeloma (MM; 5/7 for both).
Most EU approvals were classified as small molecules (55, 63%). EU approvals were often granted orphan designation (41, 47%) at some point in the development process. The commonly used expedited pathways were Accelerated Assessment (Aa; 21, 24%) and Conditional Marketing Authorization (CMA)/Approval Under Exceptional Circumstances (AUEC; 21, 24%). The EU average review time without using an expedited pathway was 490 days compared to 415 days using at least one pathway. The review times for the NMEs utilizing the PRIME scheme (n=2) were 293 and 390 days with both NMEs using PRIME in conjunction with Aa. There were no trends observed in the use of expedited pathways in the EU for the years reviewed. The top 4 approvals by cancer indication in the EU used the following pathways: CMA/AUEC for NSCLC (5/13), Aa for melanoma (2/9), Aa for MM (4/6), and none used for breast cancer (0/6).
Expedited pathways have reduced average review times for oncology NMEs in the US market, but these reductions were not seen to the same extent in the EU market. Average review times in the EU were also consistently longer compared to the US. The use of expedited pathways was more common in the US compared to the EU; this may have contributed to greater reductions in review times. In both markets, orphan designation was pursued frequently. The impact of novel pathways such as PRIME cannot yet be evaluated and warrants further analysis.