Table 5. Number of Pivotal Efficacy Trials and Number of Treated Patients in Aggregated Pivotal Trials Supporting US Food and Drug Administration Indication Approvals of New Drugs and Biologics From 1995 to 1997, 2005 to 2007, and 2015 to 2017, Overall and Stratified by Special Regulatory Program Use and Orphan Designation.
| Characteristic | Trials, No. | Pivotal efficacy trials, median (IQR), No. | Treated patients, median (IQR), aggregated No. | |
|---|---|---|---|---|
| Overall | Intervention | |||
| Overall | ||||
| 1995-1997 | 124 | 2.0 (2.0-3.0) | 774 (464-1314) | 490 (236-738) |
| 2005-2007 | 63 | 2.0 (1.0-2.8) | 699 (218-1380) | 416 (191-808) |
| 2015-2017 | 106 | 1.0 (1.0-3.0) | 816 (199-2112) | 523 (145-1303) |
| 3-Way P value | NA | .001 | .89 | .80 |
| 2-Way P valuea | NA | .001 | .83 | .73 |
| Special regulatory program | ||||
| Any | ||||
| 1995-1997 | 32 | 2.0 (1.4-3.0) | 618 (223-945) | 384 (147-568) |
| 2005-2007 | 36 | 2.0 (1.0-2.0) | 534 (166-979) | 340 (119-544) |
| 2015-2017 | 63 | 1.0 (1.0-2.0) | 404 (137-1076) | 261 (101-710) |
| 3-Way P value | NA | .002 | .40 | .54 |
| 2-Way P valuea | NA | .001 | .31 | .47 |
| None | ||||
| 1995-1997 | 92 | 2.0 (2.0-3.0) | 805 (531-1403) | 514 (293-970) |
| 2005-2007 | 27 | 2.0 (1.0-3.8) | 1167 (419-1835) | 680 (244-1374) |
| 2015-2017 | 43 | 2.0 (1.2-3.0) | 1740 (1047-2853) | 1050 (565-1768) |
| 3-Way P value | NA | .41 | .001 | .001 |
| 2-Way P valuea | NA | .43 | .001 | .001 |
| Orphan designation | ||||
| Yes | ||||
| 1995-1997 | 11 | 2.0 (1.2-2.0) | 260 (212-893) | 153 (137-444) |
| 2005-2007 | 17 | 1.0 (1.0-1.3) | 164 (73-422) | 83 (42-273) |
| 2015-2017 | 38 | 1.0 (1.0-1.1) | 196 (90-495) | 134 (79-326) |
| 3-Way P value | NA | .02 | .42 | .72 |
| 2-Way P valuea | NA | .003 | .08 | .25 |
| No | ||||
| 1995-1997 | 113 | 2.0 (2.0-3.0) | 811 (526-1369) | 510 (283-791) |
| 2005-2007 | 46 | 2.0 (1.0-3.0) | 1027 (474-1584) | 577 (267-955) |
| 2015-2017 | 68 | 2.0 (1.0-3.0) | 1646 (768-2668) | 1019 (428-1568) |
| 3-Way P value | NA | .11 | .001 | .001 |
| 2-Way P valuea | NA | .14 | .001 | .001 |
Abbreviations: IQR, interquartile range; NA, not applicable.
a
Two-way P value was calculated for differences between 1995 to 1997 and 2015 to 2017 periods.