Investigating the Landscape of Personalized Medicine Approvals by the FDA

This study characterizes the Food and Drug Administration (FDA) approved personalized medicines in the segment of New Molecular Entity (NME) and original biologic license applications (BLA) from 2015 to 2024. It focuses on approval trends of personalized medicine, their pathway utilization, orphan drug designations, and distribution across different therapeutic areas.

Data on the number of approvals, review classifications, and therapeutic areas are sourced from the Center for Drug Evaluation and Research (CDER) NME and from original BLA drug approvals. Year-wise approvals of NME and BLA belonging to personalized medicine were analysed for orphan drug designations. Further, orphan drugs among NME and BLA approvals were analysed with respect to their regulatory review type and therapeutic area. Chi-square tests were performed to identify the significant personalized medicine and its dependence on the review pathway & orphan drug designation.

During the duration of 2015–2024, a total of 465 CDER NME and original BLA were approved, of which 156 were personalized medicines that contributes approximately one-third (33.5%) of the approvals. The majority of the personalized medicines approved (107) got orphan drug designation. It was interesting to note that 92 (86%) of these orphan drug designated NMEs and BLAs were approved via the priority review pathway. When therapeutics are considered, oncology is the largest therapeutic area within the orphan-designated cohort (47 of 107 approvals), along with other indications such as amyloidosis, cystic fibrosis, and neurological disorders. Oncology accounted for 23 of 49 non-orphan personalized medicines.

Personalized medicines were significantly more associated with FDA expedited regulatory pathways than the overall CDER approval portfolio. Both priority review and Orphan Drug Designation were more frequently adopted for the approval of personalized medicines. Most approvals of NME/BLA remained concentrated in the oncology therapeutics, which may have important implications for future regulatory incentive policies.