Table 2:
Number of label updates and issues added to labels for original therapeutic biologics, by specific regulatory pathways
| Regulatory Pathway | N | Whole Study Period | 3.5 years of Follow-up | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Total number of label updates | Total number of safety issues | Total number of label updates | Total number of safety issues | |||||||||||
| mean±SD | median | Wilcoxon rank-sum P value | mean±SD | median | Wilcoxon rank-sum P value | mean±SD | median | Wilcoxon rank-sum P value | mean±SD | median | Wilcoxon rank-sum P value | |||
| Fast-track designation | Yes | 30 | 2.6±2.3 | 2 | 0.06 | 15.8±20.6 | 7 | 0.16 | 1.1±1.4 | 1 | 0.09 | 5.9±9.9 | 2.5 | 0.19 |
| No | 31 | 4.4±3.7 | 3 | 20.3±18.9 | 16 | 1.8±1.8 | 1 | 11.5±15.1 | 4 | |||||
| Priority review designation | Yes | 42 | 2.9±2.5 | 2 | 0.047 | 16.5±20.1 | 8 | 0.14 | 1.3±1.8 | 1 | 0.09 | 7.6±12.9 | 3 | 0.14 |
| No | 19 | 4.9±4.2 | 3 | 21.5±18.7 | 18 | 1.8±1.4 | 1 | 11.2±13.4 | 7 | |||||
| Accelerated approval using a surrogate endpoint | Yes | 9 | 6.0±2.6 | 7 | 0.005 | 40.4±25.2 | 33 | 0.002 | 3.1±2.7 | 2 | 0.02 | 24.6±19.0 | 20 | 0.003 |
| No | 52 | 3.1±3.1 | 2 | 14.2±15.9 | 7 | 1.2±1.2 | 1 | 6.0±9.5 | 2 | |||||
| One or more expedited program | Yes | 43 | 2.9±2.4 | 2 | 0.052 | 16.3±19.9 | 7 | 0.12 | 1.3±1.7 | 1 | 0.08 | 7.5±12.7 | 3 | 0.10 |
| No | 18 | 5.0±4.3 | 3 | 22.3±18.9 | 19.5 | 1.8±1.4 | 1.5 | 11.7±13.5 | 8 | |||||
| First-in-class | Yes | 34 | 3.4±2.6 | 3 | 0.71 | 19.1±20.2 | 15.5 | 0.57 | 1.4±1.6 | 1 | 0.7 | 9.4±13.8 | 3.5 | 0.79 |
| No | 27 | 3.7±3.8 | 2 | 16.8±19.3 | 8 | 1.6±1.7 | 1 | 7.8±12.1 | 3 | |||||
| Orphan drug | Yes | 29 | 2.7±2.3 | 2 | 0.10 | 17.4±20.9 | 10 | 0.55 | 1.4±2.0 | 1 | 0.16 | 8.3±13.7 | 1 | 0.35 |
| No | 32 | 4.2±3.7 | 3 | 18.7±18.9 | 11.5 | 1.6±1.3 | 1 | 9.1±12.6 | 4 | |||||