Table. Characteristics of New Therapeutic Agents Granted Accelerated Approval and FDA-Required Postapproval Confirmatory Trials, 2009-2018.
| Characteristic | No. (%) |
|---|---|
| Therapeutic agent characteristics | 41 (100) |
| Class | |
| Drug | 30 (73) |
| Biologic | 11 (27) |
| Therapeutic area | |
| Cancer and hematology | 33 (80) |
| Other | 8 (20) |
| Priority review | |
| Yes | 37 (90) |
| No | 4 (10) |
| Fast track | |
| Yes | 24 (59) |
| No | 17 (41) |
| Breakthrough therapy | |
| Yes | 21 (51) |
| No | 12 (29) |
| NAa | 8 (20) |
| Orphan drug designation | |
| Yes | 38 (93) |
| No | 3 (7) |
| FDA-required postapproval confirmatory trial characteristics | 50 (100) |
| Type of postapproval confirmatory trial | |
| New clinical trial | 27 (54) |
| Complete or submit results | 20 (40) |
| Otherb | 3 (6) |
| Trial focus | |
| Efficacy | 32 (64) |
| Efficacy and safety | 18 (36) |
| Randomization | |
| Yes | 43 (86) |
| No | 1 (2) |
| NA (single group assignment) | 6 (12) |
| Allocation concealment | |
| Double or higher | 18 (36) |
| Single | 0 |
| None | 29 (58) |
| Unclearc | 3 (6) |
| No. of study patients, median (IQR) | 424 (295-545) |
Abbreviations: FDA, US Food and Drug Administration; NA, not applicable.
a
Therapeutic agents approved before the origination of the breakthrough therapy designation on July 9, 2012.
b
Includes postmarketing requirements for longer-duration trial follow-up, revisions of ongoing trials, and submission of results from new and completed trials.
c
All 3 trials with unclear allocation concealment were randomized trials.