Table 3. Number and Characteristics of Aggregated Pivotal Efficacy Trials Providing the Basis for Original and Supplemental Indication Approvals by the US Food and Drug Administration Between 2017 and 2019, Stratified by Supplemental or Original Indication, Therapeutic Agent, and Indication Characteristics.
| Variable | Trials, No. | Pivotal efficacy trials, median (IQR), No. | Patients in aggregated pivotal efficacy trials, median (IQR), No. | |
|---|---|---|---|---|
| Overall | Intervention group | |||
| Supplemental or original indication | ||||
| Original | 109 | 1 (1-2) | 326 (161.5-676) | 256.3 (120.1-375.4) |
| Supplemental | 146 | 1 (1-1) | 445 (211.8-799.3) | 256.5 (144.8-447.3) |
| P value | <.001 | .05 | .12 | |
| Therapeutic agent | ||||
| Pharmacologic sNDA | 99 | 1 (1-1) | 514 (269-834) | 281.5 (155-451) |
| Biologic sBLA | 47 | 1 (1-1) | 306 (157-713) | 222 (105-340) |
| P value | .26 | .05 | .16 | |
| Therapeutic area | ||||
| Oncology sNDA or sBLA | 78 | 1 (1-1) | 505 (300.8-873) | 286 (200-471.5) |
| Nononcology sNDA or sBLA | 68 | 1 (1-1) | 334.3 (153.3-749.8) | 215.5 (99-421) |
| P value | .13 | .06 | .04 | |
Abbreviations: IQR, interquartile range; sBLA, supplemental biologics license applications; sNDA, supplemental new drug application.