Table 4. Proportion of Indication-Level Approvals of Novel Therapeutic Agents by the US Food and Drug Administration on the Basis of at Least 1 Trial That Met the Criteria Below, Stratified by Supplemental or Original Indication, Therapeutic Agent, and Indication Characteristics.
| Variable | Trials, No. | Percentage (95% CI) | ||||||
|---|---|---|---|---|---|---|---|---|
| ≥2 Pivotal trials | ≥1 Randomized trial | ≥1 Double-blind trial | Trial duration | ≥1 Trial | ||||
| ≥6 mo | ≥12 mo | With active or placebo comparator | With clinical outcome as primary efficacy end point | |||||
| Supplemental or original indication | ||||||||
| Original | 109 | 44.0 (33.7-42.6) | 80.0 (71.4-86.5) | 63.8 (54.3-72.4) | 51.5 (43.3-62.6) | 27.6 (17.9-35.0) | 78.8 (70.0-85.6) | 53.9 (46.2-65.0) |
| Supplemental | 146 | 15.8, (10.7-22.5) | 76.0 (68.5-82.2) | 58.9 (50.8-66.6) | 68.5 (60.6-75.5) | 54.8 (46.7-62.6) | 78.9 (71.4-84.6) | 45.2 (37.4-53.3) |
| P value | <.001 | .54 | .51 | .01 | <.001 | .87 | .18 | |
| Therapeutic area | ||||||||
| Pharmacologic sNDA | 99 | 18.2 (11.8-26.9) | 81.8 (73.1-88.2) | 65.7 (55.9-74.3) | 66.7 (56.9-75.2) | 54.6 (44.8-64.0) | 84.8 (76.5-90.6) | 51.5 (41.8-61.1) |
| Biologic sBLA | 47 | 10.6 (4.6-22.6) | 63.8 (49.5-76.0) | 44.7 (31.4-58.8) | 72.3 (58.2-83.1) | 55.3 (41.2-68.6) | 66.0 (51.7-77.8) | 31.9 (20.4-46.2) |
| P value | .24 | .02 | .02 | .49 | .93 | .009 | .03 | |
| Therapeutic area | ||||||||
| Oncology sNDA or sBLA | 78 | 11.5 (6.2-20.5) | 68.8 (57.9-77.8) | 37.5 (27.7-48.5) | 93.6 (85.9-97.2) | 76.9 (66.4-84.9) | 71.9 (61-80.6) | 32.1 (22.7-43.0) |
| Nononcology sNDA or sBLA | 68 | 20.6 (12.7-31.6) | 84.8 (74.3-91.6) | 84.8 (74.3-91.6) | 39.7 (28.9-51.6) | 29.4 (19.9-41.1) | 86.8 (76.7-92.9) | 60.3 (48.4-71.1) |
| P value | .10 | .04 | <.001 | <.001 | <.001 | .03 | <.001 | |
Abbreviations: sBLA, supplemental biologics license application; sNDA, supplemental new drug application.