Table 5.
Proportion of novel therapeutic indications approved by the Food and Drug Administration between 2005 and 2012 on the basis of at least one trial that met the criteria below, stratified by therapeutic and indication characteristics.
| Therapeutic/Indication Characteristic | Indications, No. | ≥ 2 Pivotal
Trials No. % (95% CI)* |
Trial Duration† No. % (95% CI) |
Comparator No. % (95% CI) |
Trial Endpoint No. % (95% CI) |
|||
|---|---|---|---|---|---|---|---|---|
| ≥ 6 months | ≥ 12 months | Active | Placebo | Clinical Outcome | Clinical Scale | |||
| All Indications | 201 | 127 63.2% (56.5%–69.9%) |
68 33.8% (27.2%–40.4%) |
17 8.5% (4.6%–12.3%) |
79 39.3% (32.5%–46.1%) |
119 59.2% (52.4%–66.0%) |
73 36.3% (29.6%–43.0%) |
39 19.4% (13.9%–24.9%) |
| Therapeutic Area | ||||||||
| Cancer | 41 | 8 19.5% (6.8%–32.1%) |
16 39.0% 23.4%–54.6%) |
2 4.9% (0.0%–11.8%) |
10 24.4% (10.7%–38.1%) |
15 36.6% (21.2%–52.0%) |
9 22.0% (8.7%–35.2%) |
0 0.0% (0.0%–0.0%) |
| Infectious Disease | 27 | 21 77.8% (61.0%–94.5%) |
5 18.5% (2.9%–34.1%) |
1 3.7% (0.0%–11.3%) |
21 77.8% (61.1%–94.5%) |
7 25.9% (8.3%–43.6%) |
13 48.1% (28.0%–68.3%) |
0 0.0% (0.0%–0.0%) |
| CV/DM/Lipids | 23 | 16 69.6% (49.2%–90.0%) |
12 52.2% (30.0%–74.3%) |
4 17.4% (0.0%–34.2%) |
13 56.5% (34.6%–78.4%) |
16 69.6% (49.2%–89.9%) |
8 34.8% (13.7%–55.8%) |
0 0.0% (0.0%–0.0%) |
| Neurology | 17 | 15 88.2% (71.1%–100.0%) |
4 23.5% (1.0%–46.0%) |
2 11.8% (0.0%–28.8%) |
5 29.4% (5.3%–53.6%) |
15 88.2% (71.1%–100.0%) |
11 64.7% (39.4%–90.0%) |
7 41.2% (15.1%–67.2%) |
| Dermatology | 15 | 11 73.3% (48.0%–98.6%) |
2 13.3% (0.0%–32.8%) |
0 0% (0.0%–0.0%) |
3 20.0% (0.0%–42.9%) |
11 73.3% (48.0%–98.7%) |
8 53.3% (24.7%–81.9%) |
5 33.3% (6.3%–60.3%) |
| Autoimmune/Musculoskeletal | 13 | 11 84.6% (61.9%–100.0%) |
6 46.2% 14.8%–77.5%) |
1 7.7% (0.0%–24.5%) |
6 46.2% (14.8%–77.5%) |
11 84.6% (61.9%–100.0%) |
1 7.7% (0.0%–24.5%) |
10 76.9% (50.4%–100.0%) |
| Psychiatry | 10 | 10 100.0% (100.0%–100.0%) |
0 0% (0.0%–0.0%) |
0 0% (0.0%–0.0%) |
8 80.0% (49.8–100.0%) |
7 70.0% (35.4%–100.0%) |
2 20.0% (0.0%–50.2%) |
8 80.0% (49.8%–100.0%) |
| Other | 55 | 35 63.6% (50.5%–76.8%) |
23 41.8% (28.4%–55.3%) |
7 12.7% (3.6%–21.8%) |
13 23.6% (12.0%–35.2%) |
37 67.3% (54.5%–80.0%) |
21 38.2% (24.9%–51.4%) |
9 16.4% (6.3%–26.5%) |
| P value | < 0.001 | 0.01 | 0.36 | < 0.001 | < 0.001 | 0.008 | < 0.001 | |
| Expected Length of Treatment | ||||||||
| Acute | 36 | 28 77.8% (63.5%–92.0%) |
1 2.8% (0.0%–8.4%) |
0 0% (0.0%–0.0%) |
20 55.6% (38.5%–72.6%) |
17 47.2% (30.0%–64.4%) |
22 61.1% (44.4%–77.8%) |
3 8.3% (0.0%–17.8%) |
| Intermediate | 57 | 21 36.8% (23.9%–49.8%) |
19 33.3% (20.7%–46.0%) |
4 7.0% (0.0%–13.8%) |
17 29.8% (17.6%–42.1%) |
25 43.9% (30.6%–57.1%) |
14 24.6% (13.0%–36.1%) |
10 17.5% (7.4%–27.7%) |
| Chronic | 108 | 78 72.2% (63.6%–80.8%) |
48 44.4% (34.9%–54.0%) |
13 12.0% (5.8%–18.3%) |
42 38.9% (29.5%–48.2%) |
77 71.3% (62.6%–80.0%) |
37 34.3% (25.2%–43.4%) |
26 24.1% (15.9%–32.3%) |
| P value | < 0.001 | < 0.001 | 0.07 | 0.05 | 0.001 | 0.001 | 0.11 | |
| Novel Therapeutic Type | ||||||||
| Pharmacologic | 164 | 110 67.1% (59.8%–74.3%) |
52 31.7% (24.5%–38.9%) |
15 9.1% (4.7%–13.6%) |
71 43.3% (35.6%–51.0%) |
92 56.1% (48.4%–63.8%) |
66 40.2% (32.7%–47.8%) |
23 14.0% (8.7%–19.4%) |
| Biologic | 37 | 17 45.9% (29.1%–62.8%) |
16 43.2% (26.5%–60.0%) |
2 5.4% (0.0%–13.0%) |
8 21.6% (7.7%–35.5%) |
27 73.0% (58.0%–88.0%) |
7 18.9% (5.7%–32.2%) |
16 43.2% (26.5%–60.0%) |
| P value | 0.02 | 0.18 | 0.46 | 0.01 | 0.06 | 0.01 | < 0.001 | |
| Orphan Status | ||||||||
| Yes | 29 | 13 44.8% (25.6%–64.0%) |
12 41.4% (22.3%–60.4%) |
5 17.2% (2.6%–31.9%) |
8 27.6% (10.3%–44.9%) |
11 37.9% (19.1%–56.7%) |
8 27.6% (10.3%–44.9%) |
2 6.9% (0.0%–16.7%) |
| No | 172 | 114 66.3% (59.1%–73.4%) |
56 32.6% (25.5%–39.6%) |
12 7.0% (3.1%–10.8%) |
71 41.3% (33.8%–48.7%) |
108 62.8% (55.5%–70.9%) |
65 37.8% (30.5%–45.1%) |
37 21.5% (15.3%–27.7%) |
| P value | 0.03 | 0.35 | 0.07 | 0.16 | 0.01 | 0.29 | 0.07 | |
| Accelerated Approval | ||||||||
| Yes | 23 | 13 56.5% (34.6%–78.4%) |
8 34.8% (13.7%–55.8%) |
2 8.7% (0.0%–21.2%) |
4 17.4% (0.0%–34.2%) |
7 30.4% (10.0%–50.8%) |
1 4.3% (0.0%–13.4%) |
0 0.0% (0.0%–0.0%) |
| No | 178 | 114 64.0% (56.9%–71.2%) |
60 33.7% (26.7%–40.7%) |
15 8.4% (4.3%–12.5%) |
75 42.1% (34.8%–49.5%) |
112 62.9% (55.8%–70.0%) |
72 40.4% (33.2%–47.7%) |
39 21.9% (15.8%–28.0%) |
| P value | 0.48 | 0.92 | 0.97 | 0.02 | 0.003 | 0.001 | 0.001 | |
Notes: CI=Confidence Interval; CV/DM/Lipids=Cardiovascular disease, diabetes mellitus, and hyperlipidemia.
*
Two or more pivotal trials served as the basis of FDA approval
†
Excludes “single administration” trials, in which the investigational drug was given only once.