Presentation (CTI)

OBJECTIVES: To examine key factors associated with BT and FT designations, likelihood of priority review, and impact on the time required for FDA review of original NDAs/BLAs.
METHODS: All 579 original NDAs/BLAs to FDA’s CDER from 2013-2025 were reviewed. Review times associated with and without BT and FT designations were compared. Relative risks (RRs) were calculated for comparisons of interest. Indications were categorized according to the International Classification of Diseases 11.
RESULTS: Across a mean (range) of 45 (22-59) annual approvals, 28% (11-42%) had BT, 37% (31-45%) had FT, and 12% (7-20%) received both (BT+FT) designations. Orphan indications were significantly more likely than non-orphan indications to receive BT (RR=1.9), FT (RR=1.5), or BT+FT designations (RR=2.5). Neoplasms (BT and FT) and infectious and parasitic diseases (FT only) were significantly more likely to receive designation than other indications. Conversely, BT or FT were significantly less likely to be designated for indications relating to nervous system or skin, respectively, compared to other indications. NDAs/BLAs with BT or FT designations had a significantly higher likelihood of receiving priority review (BT, RR=1.9; FT, RR=1.8; BT+FT, RR=3.0). BT and FT were also associated with small but significantly higher likelihoods of first-cycle approval. Across NDAs/BLAs with first-cycle approval, BT or FT designation did not substantially reduce the mean FDA review period (BT, 7.4 months; FT, 8.1 months; BT+FT, 7.7 months) compared to priority review with no expedited pathways (8.1 months). Conversely, the mean review time for non-priority (i.e., standard) NDAs/BLAs was 14.7 months.
CONCLUSIONS: Approximately half of all NDAs/BLAs have BT and/or FT designations. For original NDAs/BLAs, BT and FT appear to have a limited direct effect on FDA review time. However, BT or FT may indirectly reduce FDA review time by substantially increasing the likelihood of priority review, reducing FDA review by 6-7 months.